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Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 1 of 400
Generic Name Trade Name Designation Date Designation
1
n/a 1/6/20162
ascorbic acid n/a 5/11/2009 Murigenetics SAS3
budesonide n/a 12/20/20064
n/a 1/13/2016 Treatment of cystic fibrosis5
n/a 9/11/2014 Treatment of empyema (pleural)6
n/a 10/12/2011 Treatment of Fragile X syndrome7
n/a 8/22/2016 SCYNEXIS, Inc.8
n/a 6/5/2013 Treatment of pancreatic cancer EMD Serono9
n/a 3/9/2012 Treatment of soft tissue sarcoma
Row Num
Contact Company/Sponsor
1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol;1H-1-(3-[18F]fluoro-2-hydroxypropyl)-2-nitroimidazole
As a diagnostic for clinical management of soft tissue sarcoma, including rhabdomyosarcoma.
Advanced Imaging Projects, LLC
Treatment of Charcot-Marie-Tooth disease type 1A.
Treatment of patients with eosinophilic esophagitis
Shire ViroPharma Incorporated
S-nitrosoglutathione reductase inhibitor
Nivalis Therapeutics, Inc.
single chain urokinase plasminogen activator
Lung Therapeutics, Inc.
(-)-(3aR,4S,7aR)-4-Hydroxy-4-m-tolylethynyl-octahydro-indole-1-carboxylic acid methyl ester
Novartis Pharmaceuticals Corp.
(1,3)-ß-D-glucan synthesis inhibitor
Treatment of invasive Aspergillus infections
(1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-broethyl) diamidophosphate
(1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate
Threshold Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 2 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
10
n/a 6/23/2015 Pfizer, Inc.11
n/a 2/17/2009 Pfizer, Inc.12
n/a 8/13/200413
n/a 1/29/200414
n/a 8/10/2004
(1OR)-7-amino-12-fluoro-2,10,16-trimethyl-15 oxo-10,15,16,17-tetrahydro-2H-8,4-(metheno)pyrazolo[4,3-h][2,5,11]benzoxadiazacyclotetradecine-3-carbonitrile
Treatment of anaplastic lymphoma kinase (ALK)-positive or ROS1-positive non-small cell lung cancer
(1R,3R,4R,5S)-3-O-[2-O-benzoyl-3-O-(sodium(2S)-3-cyclohexyl-propanoate-
Treatment of vaso-occlusive crisis in patients with sickle cell disease.
(1S)-1-(9-deazahypoxanthin-9-yl)-1,4-dideoxy-1,4-imino-D-ribitol-hydrochloride
Treatment of acute lymphoblastic leukemia
Mundipharma Research Limited
(1S)-1-(9-deazahypoxanthin-9-yl)-1,4-dideoxy-1,4-imino-D-ribitol-hydrochloride
Treatment of T-cell non-Hodgkin's lymphoma
Mundipharma Research Limited
(1S)-1-(9-deazahypoxanthin-9-yl)-1,4-dideoxy-1,4-imino-D-ribitol-hydrochloride
Treatment of chronic lymphocytic leukemia and related leukemias to include prolymphocytic leukemia, adult T-cell leukemia, and hairy cell leukemia
Mundipharma Research Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 3 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
15
n/a 9/15/2010 Treatment of infantile spasms.16
n/a 6/23/201417
n/a 5/18/2016 Treatment of Rett Syndrome.18
n/a 11/29/2016 Treatment of multiple myeloma19
n/a 10/14/2015 Treatment of systemic sclerosis.
(1S,3S)-3-amino-4-(difluoromethylene)cyclopentanecarboxylic acid hydrochloride, (1S,3S)-3-amino-4-difluoromethylenyl-1-cyclopentanoic acid hydrochloride
Catalyst Pharmaceuticals, Inc.
(2'R,3'S)-2'-hydroxy-N-carboxy-3'-amino-5'-methyl-hexanoic,N-tert-butyl ester, 13 ester 5B-20-epoxy-1B,2a,4a,7B,9a,10a,13a-heptahydroxy-4,10-diacetate-2-benzoate-(1"S)-7,9-acrolein acetal-11(15-1)-abeotaxane
Treatment of progressive supranuclear palsy
Cortice Biosciences, Inc.
(2,2-diphenyl-tetrahydro-furan-3-yl-methyl)-dimethylamine hydrochloride
Anavex Life Sciences Corporation
(2-((2-chlorophenyl)(phenyl)amino)-N-(7-(hydroxyamino)-7-oxoheptyl)pyrimidine-5-carboxamide)
Acetylon Pharmaceuticals, Inc.
(2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1h-pyrazolo [4,3-c] pyridine-3,6(2h,5h)-dione)
Genkyotex Innovation SAS
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 4 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
20
n/a 12/2/2010 QLT Inc.21
n/a 12/2/2010 QLT, Inc.22
n/a 10/31/2012 Albireo AB23
n/a 10/31/2012 Albireo AB
(2E, 4E, 6Z, 8E)-3,7-dimethyl-9-(2,6,6-trimethylcyclohex-1-en-l-yl) nona-2,4,6,8-tetraen-l-yl acetate
Treatment of retinitis pigmentosa
(2E,4E,6Z,8E)-3,7-dimethyl-9-(2,6,6-trimethylcyclohex-1-enyl)nona-2,4,6,8-tetraenyl acetate 9-cis-retinyl acetate (API)
Treatment of Leber congential amaurosis (LCA) due to inherited mutations in RPE65 (encoding the protein retinal pigment epithelial protein 65) or LRAT (encoding the enzyme lecithin:retinol acyltransferase)genes.
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid
Treatment of primary biliary cirrhosis
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid
Treatment of progressive familial intrahepatic cholestatis
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 5 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
24
Fk778 1/10/200525
n/a 3/18/201126
n/a 3/18/2011 Treatment of pancreatic cancer27
n/a 3/18/2011 Treatment of Glioma28
n/a 4/30/2014 Treatment of multiple myeloma VivoLux AB29
n/a 12/9/201530
n/a 9/11/2014 Treatment of Gaucher disease Genzyme
(2Z)-2-cyano-3-3hydroxy-N-[4-(trifluoromethly)phenyl]-2-hepten-6-ynamide
Prevention of acute rejection following kidney, heart, and liver transplantation
Fujisawa Healthcare, Inc.
(3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamide
Treatment of chronic myelogenous leukemia.
NATCO Pharma Limited
(3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamide
NATCO Pharma Limited
(3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamideNRC-AN-019
NATCO Pharma Limited
(3E,5E)-3,5-bis[(4-fluoro-3-nitrophenyl)methylidene]-1-(prop-2-enoyl)azepan-4-0ne
(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one
Treatment of Fragile X Syndrome.
Centre National de la Recherche Scientifique (CNRS)
(3S)-1-azabicyclo[2.2.2]oct-3-yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 6 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
31
n/a 8/26/2014 Treatment of Fabry's disease Genzyme Corporation32
n/a 7/5/2007 Treatment of tuberculosis33
n/a 8/19/200334
n/a 4/25/201635
n/a 9/18/2015 AbbVie, Inc.
(3S)-1-azabicylo[2.2.2]oct-3yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate
(3S)-3-(4-trifluoromethoxybenzyloxy)-6-nitro-2H-3,4-dihydroimidazo[2,1-b]oxazine
Global Alliance for TB Drug Development
(3S)-3-[(2S)-2-({N-[2-tert-butyl)phenyl]carbamoyl}carbonylamino) propanoylamino]-4-oxo-5-(2,3,5,6-tetrafluorophenoxy) pentanoci acid
Treatment of patients undergoing solid organ transplantation.
Pfizer Global Research and Development
(3S)-3-[({1-(2-Chlorobenzyl)-4-hydroxy-5-methyl-2-oxo-1,2-dihydropyridin-3yl}amino)carbonyl]amino-3-(4-methylphenyl)propanoic acid monosodium salt
For mobilization of hematopoietic stem cells
Aviara Pharmaceuticals, Inc/
(3S,4R)-3-Ethyl-4-(3H-imidazo[1,2-a]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)pyrrolidine-1-carboxamide (2R,3R)-2,3-dihydroxybutanedioate
Treatment of pediatric (aged 0 through 16 years) juvenile idiopathic arthritis (JIA) ILAR categories excluding systemic JIA
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 7 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
36
n/a 9/4/2013 Treatment of alagille syndrome37
n/a 9/4/201338
n/a 9/4/201339
n/a 7/24/2013
(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride
Shire Human Genetic Therapies, Inc.
(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride
Treatment of primary biliary cirrhosis
Shire Human Genetic Therapies, Inc.
(4R,5R)-1-[[4-[[4-[3,3-Dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-l-azoniabicyclo[2.2.2]octane Chloride
Treatment of primary sclerosing cholangitis
Shire Human Genetic Therapies, Inc.
(5R)-5-(4-{[2-fluorophenyl)methyl]oxy}phenyl)-L-prolinamide, hydrochloride
Treatment of trigeminal neuralgia
Convergence Pharmaceuticals Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 8 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
40
n/a 4/14/2016 Treatment of ovarian cancer41
n/a 6/29/2011 Treatment of soft tissue sarcoma CytRx Corporation42
n/a 2/21/201443
n/a 10/13/2015 Treatment of cystic fibrosis.44
n/a 6/10/2015 Treatment of systemic sclerosis45
Cofactor 8/13/2004
(5S,8S, 10aR)-N-benzhydryl-5-((S)-2-(methylamino)propanamido)-3-(3-methylbutanoyl)-6-oxodecahydropyrrolo[1,2-a][1,5] diazocine-8-carboxyamide
DebioPharm International SA
(6-maleimidocaproyl)hydrazone of doxorubicin
(6-[4-Deoxy-4-[(2E,4E)-tetradecadienoylglycyl] amino-L-glyceroB-L-manno-heptopyranosyl]amino-9H-purine)
Parenteral treatment of painful, chronic, chemotherapy-induced peripheral neuropathy that is refractory to conventional analgesics
DARA BioSciences, Inc.
(6aR, 10aR)-3-(1’,1’-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid
Corbus Pharmaceuticals, Inc.
(6aR, 10aR)-3-(1⿿,1⿿-dimethylheptyl)-ο8-tetrahydro-cannabinol-9-carboxylic acid
Corbus Pharmaceuticals, Inc.
(6R,S)5,10-methylene-tetrahydrofolic acid
For use in combination with 5-fluorouracil for the treatment of patients with pancreatic cancer
Adventrx Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 9 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
46
n/a 12/8/200447
n/a 4/21/2016 Neem Biotech Ltd.48
n/a 3/19/201549
n/a 3/9/2012 Atterocor, Inc.50
n/a 4/24/2014 Treatment of cystic fibrosis51
n/a 4/15/2011
(9-[N-(3-morpholinopropyl)-sulfonyl]-5,6-dihydro-5-oxo-11-H-indeno [1,2-c] isoquinoline methanesulfonic acid
Prevention of post-operative complications of aortic anuerysm surgical repair
Inotek Pharmaceuticals Corporation
(E, Z)-4,5,9-trithiadodeca-1, 6, 11-triene 9-oxide (Ajoene)
Treatment of Pseudomonas aeruginosa pulmonary infections in cystic fibrosis patients
(monomethoxypolyethylene glycol) recombinant adenosine deaminase
Treatment of adenosine deaminase deficiency in patients with severe combined immunodeficiency
Sigma-Tau Pharmaceuticals, Inc.
(N-[2,6-bis(1-methylethyl)-pheyl-N'-[[1-4-dimethyl-amino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt
Treatment of adrenocortical carcinoma
(R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide
Vertex Pharmaceuticals Inc.
(R)-1-phenylethyl-5-(4-biphenyl-4-cyclopropanecarboxylic acid)-3-methylisoxazole-4-yl carbamate sodium salt
Treatment of idiopathic pulmonary fibrosis
Bristol-Myers Squibb Company
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 10 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
52
n/a 5/12/2016 Trio Medicines Ltd.53
n/a 7/12/200754
n/a 12/19/2007 AbbVie, Inc55
n/a 11/19/2015 ArQule, Inc.
(R)-1-[1-(4-Acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea
Treatment of gastric neuroendocrine tumors (gastric NETs).
(R)-2-methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole
Treatment of pulmonary tuberculosis.
Otsuka Pharmaceutical Company, Ltd
(R)-4-(3-morpholin-4-yl-1-phenylsulfanylmethyl-propylamino)-N-(4-{4-[2-(4-chlorophenyl)-5,5-dimethylcyclohex-1-enylmethyl]-piperazin-1-yl}-benzoyl)-3-trifluoromethanesulfonylbenzenesulfonamide bis-hydrochloride
Treatment of small cell lung cancer.
(R)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride
Treatment of cholangiocarcinoma.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 11 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
56
n/a 10/3/200157
n/a 7/3/200358
n/a 3/17/2014 Pharnext SA59
n/a 11/5/2014 BerGenBio AS60
Lipotecan 10/6/2010
(R)-N-[2-(6-chloro-5-methoxy-1H-indol-3-yl)propyl]acetamide
Treatment of circadian rhythm sleep disorders in blind people with no light perception
Phase 2 Discovery, Inc.
(R)-N-[2-(6-Chloro-methoxy-1H-indol-3yl)propyl]acetamide
Treatment of neuroleptic-induced tardive dyskinesia in schizophrenia patients
Phase 2 Discovery, Inc.
(RS)-baclofen, naltrexone and D-sorbitol
Treatment of Charcot-Marie-Tooth disease type 1A
(S)-1-(6,7-Dihydro-5H-benzo[6,7]cyclohepta[1,2-c]pyridazin-3-yl)-N3-(7-(pyrrolidin-1-yl)-6,7,8,9-tetrahydro-5H-benzo[7]annulen-2-yl)-1H-1,2,4-triazole-3,5-diamine
Treatment of acute myeloid leukemia
(S)-10-[(dimethylamino)methyl]-4-ethyl-9hydroxy-4-O-[a-(2",4",5",7"-tetranitro-9"-fluorenylideneaminooxy)propionyl]- 1H-pyrano[3',4',6',7',] indolizino [1,2-B]-quinoline-3, 14-(4H, 12H)-dione, hydrochloride
Treatment of hepatocellular carcinoma
TLC Biopharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 12 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
61
n/a 1/26/2015 Treatment of pemphigus vulgaris Almirall S.A.62
n/a 11/17/2014 Pulmokine, Inc.63
n/a 8/1/201364
n/a 2/4/200965
n/a 6/24/2013
(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile
(S)-3-((3-(1-((6-((3,4-dimethoxyphenyl)pryazin-2-yl)amino)ethyl)phenyl)carbamoyl)-5-methylpridin-1-ium
Treatment of pulmonary arterial hypertension
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
Treatment of follicular lymphoma
Infinity Pharmaceuticals, Inc.
(S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiaxolecarboxylic acid
Treatment of chronic iron overload in patients with transfusion-dependent anemias
Ferrokin BioSciences, Inc.
(S)-4-(5-chloro-2-(isopropylamino)pyridin-4-yl)-N-(1-(3-chlorophenyl)-2-hydroxyethyl)-1H-pyrrole-2-carboxamide hydrochloride
Treatment of Stage IIb through Stage IV BRAF mutant melanoma
BioMed Valley Discoveries, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 13 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
66
n/a 10/22/2015 Acerta Pharma BV67
n/a 9/21/2015 Acerta Pharma BV68
n/a 11/17/2014 Khondrion BV69
n/a 4/29/2015 Treatment of Hodgkin lymphoma Incyte Corporation70
n/a 6/23/2016 BeiGene USA, Inc.
(S)-4-(8-amino-3-(1-but-2-ynoylpyrrolidin-2-yl)-imidazo[1,5-a]pyrazin-1-yl)-N-(pyridin-2-yl)-benzamide
Treatment of Waldenstrom macroglobulinemia.
(S)-4-(8-amino-3-(1-but-2-ynoylpyrrolidin-2-yl)-imidazo[1,5-a]pyrazine-1-yl)-N-(pyridine-2-yl)-benzamide
Treatment of mantle cell lymphoma.
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
Treatment of inherited mitochondrial respiratory chain diseases
(S)-7-(1-(9H-purin-6-ylamino)ethyl)-6-(3-fluorophenyl)-3-methyl-5H-thiazolo[3,2-a]pyrimidin-5-one
(S)-7-(1-acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydroprazolo[1,5-a]pyrimidine-3-carboxamide
Treatment of mantle cell lymphoma.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 14 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
71
n/a 6/29/2016 BeiGene USA, Inc.72
n/a 7/20/2016 BeiGene USA, Inc.73
n/a 10/6/201574
n/a 8/31/2015 Loxo Oncology, Inc.75
n/a 11/12/2014 Treatment of glioma76
n/a 3/28/2006 Auris Medical, Inc.
(S)-7-(1-acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydropyrazolo[1,5-a]pyrimidine-3-carboxamide
Treatment of Waldenstrom's macroglobulinemia
(S)-7-(1acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydropyrazolo[1,5-a]pyrimidine-3-carboxamide
Treatment of chronic lymphocytic leukemia.
(S)-8-{2-Amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester
Treatment of pulmonary arterial hypertension (PAH).
KAROS Pharmaceuticals, Inc.
(S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate
Treatment of soft tissue sarcoma.
(S)-perillyl alcohol temozolomide
NeOnc Technologies, Inc.
(UDU-stereoisomer of c-UJUun UNU-terminal UkUnhibitor)
Treatment of acute sensorineural hearing loss
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 15 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
77
n/a 5/14/201478
n/a 12/3/2014 Treatment of multiple myeloma79
n/a 11/19/2012 Treatment of erythromelalgia Teva Pharmaceuticals80
n/a 1/31/2012 Treatment of malignant gliomas81
1,2:5,6-dianhydrogalactitol n/a 3/10/2016 Treatment of medulloblastoma.82
1,2:5,6-dianhydrogalactitol n/a 4/19/2016 Treatment of ovarian cancer.83
n/a 5/12/1998 Treatment of Fabry's disease. Oxford GlycoSciences84
n/a 4/6/201685
Sapacitabine 6/24/2010 Cyclacel Limited
(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N-(pyrazin-2-yl)acrylohydrazide
Treatment of diffuse large B-cell lymphoma
Karyopharm Therapeutics, Inc.
1 8-(p[131I]-iodophenyl)octadecyl phosphocholine
Cellectar Biosciences, Inc.
1'-{[5-(trifluoromethyl)-2-furyl]methyl}spiro[furo[2,3-f][1,3]benzodioxole-7,3'-indol]-2'(1'H)-one
1,2:5,6-Dianhdrogalactitol, NSC-132313
DelMar Pharmaceuticals, Inc.
DelMar Pharmaceuticals, Inc.
DelMar Pharmaceuticals, Inc. (Clinical Operations)
1,5-(Butylimino)-1,5 dideoxy,D-glucitol
1-(2,8-Dimethyl-1-thia-3,8-diazaspiro[4.5]dec-3-yl)-3-(1H-indol-3-yl)propan-1-one
Treatment of frontotemporal dementia.
Anavex Life Sciences Corp.
1-(2-C-cyano-2-deoxy-B-D-arabino-pentafuranosyl)-N4-palmitoylcytosine
Treatment of acute myelogenous leukemia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 16 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
86
Sapacitabine 6/24/2010 Cyclacel Limited87
n/a 7/7/2016 Treatment of status epilepticus88
n/a 4/6/201689
n/a 9/1/2011 Eisai, Inc.90
n/a 7/8/2015 Treatment of multiple myeloma91
n/a 3/31/2015 Treatment of systemic sclerosis Inventiva Pharma92
n/a 11/12/2009
1-(2-C-cyano-2-deoxy-B-D-arabino-pentafuranosyl)-N4-palmitoylcytosine
Treatment of myelodysplastic syndrome
1-(2-chlorophenyl)-1-(S)-hydroxy-2-(S)-carbamoyloxy-propane
Bio-Pharm Solutions Co., Ltd.
1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol; 1H-1-(3-[18F]fluoro-2-hydroxypropyl0-2-nitroimidazole (18F-Misonidazole)
Diagnostic for management of glioma.
Advanced Imaging Projects, LLC
1-(3-chloro-5-{[4-(4-chloro-2-thienyl)-5-(4-cyclohexylpiperazin-l-yl)-1,3-thiazol-2-yl]carbamoyl}- 2-pyridyl) piperidine-4-caboxylic acid Monomaleate
Treatment of idiopathic thrombocytopenic purpura
1-(4-(benzylamino)-7,8-dihydro-5H-pyrano[4,3-d]pyrimidin-2-yl)-2-methyl-1H-indole-4-carboxamide
Cleave Biosciences, Inc.
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
1-Cyclopropyl-3-[3-(5-morpholin-4-ylmethyl-1H-benzoimidazol-2-yl)-1H-pyrazol-4-yl]-urea
Treatment of acute myeloid leukemia
Astex Therapeutics Ltd
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 17 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
93
n/a 1/9/2012 ViroDefense, Inc.94
n/a 1/23/201395
111Indium pentetreotide Somatother 6/10/199996
n/a 6/7/2011 Treatment of pancreatic cancer Oncos Therapeutics97
n/a 8/29/2016 Treatment of neuroblastoma98
n/a 3/28/201699
17 amino acid peptide n/a 1/29/2010100
n/a 6/1/2015 Lipocine, Inc.
1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy) methyl]benzene
Treatment of poliovirus infection.
10 synthetic peptides targeting 5 tumor asociated antigens
Treatment of non-small cell lung cancer in patients expressing HLA-A2
Orphan Synergy Europe Pharma (OSE Pharma)
Treatment of somatostatin receptor positive neuroendocrine tumors.
Louisiana State University Medical Center Foundation
12-A-p21 RAS(5-21). 12-C-p21 RAS(5-21). 12-D-p21 RAS(5-21). 12-Rp21 RAS(5-21). 12-S-p21 RAS(5-21). 12-V-p21 RAS(5-21). 13-D-p21 RAS(5-21)
131-I-8H9 monoclonal antibody
Memorial Sloan Kettering Cancer Center
131I-BC8 monoclonal antibody (Anti-CD45 murine monoclonal antibody radiolabeled with 131I
Treatment of Acute Myeloid Leukemia (AML)
Actinium Pharmaceuticals, Inc.
Prevention of ischemia reperfusion injury in the lung during lung tranplantation
Apeptico Forschung und Entwicklung GmbH
17-a-hydroxyprogesterone caporate (oral formulation)
Prevention of preterm birth in women with a singleton pregnancy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 18 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
101
n/a 9/3/2004102
n/a 4/16/2015 SolaranRx, Inc.103
177Lu-tetraxetan-tetulomab Betalutin 5/14/2014 Nordic Nanovector AS104
n/a 4/30/2014105
n/a 1/26/2015106
n/a 2/17/2016107
2 dimethylbutyrate n/a 6/18/2008 Treatment of beta thalassemia108
2'-deoxycytidine n/a 9/9/1996 Grant, Steven M.D.
17-allylamino-17-demethoxygeldanamycin (17-AGG)
Treatment of chronic myelogenous leukemia
Bristol-Myers Squibb Company
177-LU-DOTA-GlyGlyNle-CycMSHhex
Treatment of Stage IIB through IV malignant melanoma
Treatment of Follicular Lymphoma
18-(p-[124I]-iodophenyl)octadecyl phosphocholine
Diagnostic for the management of glioma
Cellectar Biosciences, Inc.
18Fluorine-N-3-Fluoropropyl-2beta-carbomethoxy-3beta-(4-iodophenyl) Nortropane
Diagnostic to be used in the management of multiple system atrophy (MSA)
Advanced Imaging Projects, LLC
1E,6E)-1,7-Bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione or [(1E, 4Z,6E)-4-(cyclobutylmethyl)-1,7-bis(3,4-dimethoxyphenyl)-5-hydroxyhepta-1,4,6-trien-3-one]
Treatment of spinal and bulbar muscular atrophy
Allianz Pharmascience Ltd.
HemaQuest Pharmaceuticals, Inc.
As a host-protective agent in the treatment of acute myelogenous leukemia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 19 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
109
n/a 5/10/2016 Treatment of acromegaly110
n/a 10/31/2012 LifeSplice Pharma LLC111
n/a 3/9/2012112
n/a 1/15/2015113
n/a 9/10/2015 InFlectis BioScience114
n/a 9/21/2010 GenKyoTex S.A.
2'-O-(2-methoxyethyl)phosphorothioate antisense oligonucleotide
Antisense Therapeutics Limited
2'-O-methyl phosphorothioate 5'-GCUAGGUUUACGGGACCUCU-3'
Treatment of amyotrophic lateral sclerosis
2,2'-{2-[1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid (ixazomib citrate)
Treatment of systemic light chain (AL) amyloidosis.
Millennium Pharmaceuticals, Inc.
2,5-dimethyl-3-[2-methyl-4-(methyloxy)phenyl]-N-[(1S)-1-(3-methyl-1,2,4-oxadiazol-5-
Treatment of congenital adrenal hyperplasia (CAH)
Neurocrine Biosciences, Inc.
2-(2-chlorobenzylidene)hydrazinecarboximidamide acetate
Treatment of Charcot-Marie Tooth disease.
2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione
Treatment of idiopathic pulmonary fibrosis.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 20 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
115
n/a 1/29/2010 EntreMed, Inc.116
n/a 7/1/2014117
n/a 12/22/2008 Treatment of multiple myeloma. EntreMed, Inc.118
n/a 3/18/2009 Treatment of ovarian carcinoma EntreMed, Inc.119
n/a 1/26/2015 Treatment of pancreatic cancer Incyte Corporation120
Atiprimod 12/2/2003
2-(2-phenylvinyl)-4-[-methylpiperazin-l-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt
Treatment of acute myeloid leukemia.
2-(2-phenylvinyl)-4-[4-methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt
Treatment of hepatocellular carcinoma
CASI Pharmaceuticals, Inc.
2-(2-phenylvinyl)-4-[4-methylpiperazin-l-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt
2-(2-phenylvinyl)-4-[4-methylpiperazin-l-yl)]-6-(5-methyl-2H-pyrazol-3-ylamino)-pyrimidine L(+) tartrate salt
2-(3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-l-yl)-1-(1-(3-fluoro-2-(trifluoromethyl)isonicotinoyl)piperidin-4-yl)azetidin-3-yl)acetonitrile adipate
2-(3-diethylaminopropyl)-8,8-dipropyl-2-azaspiro [4,5] decan dimaleate
Treatment of mulitple myeloma and associated bone resorption
Callisto Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 21 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
121
Atiprimod 9/18/2006 Treatment of carcinoid tumors122
n/a 6/1/2015123
n/a 5/13/2015 Treatment of cystic fibrosis Flatley Discovery Lab124
n/a 8/15/2003 Treatment of cystic fibrosis Inologic, Inc.125
2-0-desulfated heparin Aeropin 9/17/1993 Treatment of cystic fibrosis.126
n/a 10/6/2015127
n/a 10/26/2016
2-(3-Diethylaminopropyl)-8,8-dipropyl-2-azaspiro[4,5] decane dimaleate
Callisto Pharmaceuticals, Inc.
2-(5-fluoro-2-methyl-1H-indol-3-yl)-1H-imidazo[4,5-f][1,10] phenanthroline
Treatment of acute myeloid leukemia
Aptose Biosciences, Inc.
2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl)acetamide
2-0-Butyryl-1-0-octyl-myo-inositol 3,4,5,6-tetrakisphosphate
Kennedy & Hoidal, M.D.'s
2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy) phenyl] thio]phenyl]ethyl]1,3-propanediol hydrochloride
Prevention of graft versus host disease.
Novartis Pharmaceuticals Corporation
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate
Treatment of invasive aspergillosis
Amplyx Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 22 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
128
n/a 11/2/2016129
n/a 10/19/2016 Treatment of invasive candidiasis130
n/a 11/3/2016 Treatment of coccidioidomycosis131
n/a 5/18/2016132
n/a 2/17/2012133
n/a 8/3/2001 Treatment for malignant gliomas
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate
Treatment of invasive rare mold infections caused by scedosporium spp., fusarium spp., and mucorales fungi (including mucor spp., and rhizopus spp.)
Amplyx Pharmaceuticals, Inc.
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate
Amplyx Pharmaceuticals, Inc.
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate
Amplyx Pharmaceuticals, Inc.
2-amino-N-({methyl-[(6-trifluoromethoxy-benzothiazol-2-ylcarbamoyl)-methyl]-carbamoyl}-methyl)-acetamide monohydrochloride
Treatment of spinocerebellar ataxia.
Biohaven Pharmaceuticals, Inc.
2-Chloro-N6--(3-iodobenzyl)adenosine-5'-N-methyluronamide
Treatment of hepatocellular carcinoma
Can-Fite BioPharma Ltd.
2-chloroethyl-3-sarcosinamide-1-nitrosourea
Lawrence Panasci, MD
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 23 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
134
Sarmustine 11/15/2001 Treatment for malignant glioma Pangene Corporation135
n/a 9/18/2015 Treatment of Ebola virus disease Gilead Sciences, Inc.136
n/a 12/29/2015137
Kleptose 2/18/2013 Vtesse, Inc.138
2-iminobiotin n/a 2/24/2009 Treatment of perinatal asphyxia. Neurophyxia B.V.139
2-methoxyestradiol Pulmolar 4/11/2005140
n/a 4/9/2015 Treatment of anal cancer Eli Lilly and Company
2-chloroethyl-3-sarcosinamide-1-nitrosourea
2-Ethylbutyl (2S)-2-{[(S)-{[(2R,3S,4R,5R)-5-(4-aminopyrrolo[2,1-f][1,2,4]triazin-7-yl)-5-cyano-3,4-dihydroxytetrahydrofuran-2-yl]methoxy}(phenoxy)phosphoryl]amino}propanoate
2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl) methoxy)benzaldehyde
Treatment of sickle cell disease (SCD).
Global Blood Therapeutics, Inc.
2-hydroxypropyl-B-cyclodextrin
Treatment of Niemann Pick disease, type C.
Treatment of pulmonary arterial hypertension
PR Pharmaceuticals, Inc.
2-Pyrazinecarbonitrile, 5-[[5-2-(3-aminopropoxy)-6-methoxyphenyl]-1H-pyrazol-3-yl]amino] monomesylate monohydrate
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 24 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
141
Prevonco (Tm) 8/27/2008 BioQuant, Inc.142
n/a 9/29/2015143
n/a 7/16/2014 OncoEthix SA144
n/a 12/18/2007 AbbVie, Inc.145
n/a 6/2/2014 Pfizer Inc.146
n/a 5/14/2013 Sanofi U. S., Inc.
2-[(3-methyl-4-(2,2,2-trifluoroethoxy) pyridin-2-yl)methylsulfinyl] -1H-benzoimidazole
Treatment of hepatocellular carcinoma.
2-[(4S)-6-(4-chlorophenyl)-1,7,8-trimethylthiophenol[3,2-f]1,2,4-triazolo[4,3-a]1,4-diazepin-4-yl]-N-[3-(4-methylpiperazinyl)propyl]acetamide
Treatment of nuclear protein in testis (NUT) midline carcinoma.
Genentech, Inc (a Roche Group Member)
2-[(6S)-4-(4-chlorophenyl)-2,3,9-trimethyl-6H-thienol[3,2,-f]-[1,2,4]triazolo[4,3-a][1,4]diazepin-6-yl]-N-(4-hydroxyphenyl)-acetamide dihydrate
Treatment of acute myeloid leukemia
2-[(R)-2-methylpyrrolidin-2-yl]-1H-benzimadazole-4-carboxamide
Treatment of malignant melanoma stages IIb through IV.
2-[4-(1-Methyl-4-pyridin-4-yl-lH-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid
Treatment of Huntington's disease.
2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indian-2-carboxylic acid
Treatment of patients with systemic sclerosis
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 25 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
147
n/a 4/15/2015148
n/a 11/7/2016149
n/a 3/18/2015150
n/a 1/19/2010151
n/a 6/16/2015 Treatment of ovarian cancer. Rgenix, Inc.
2-[4-[[(2-R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate
Treatment of patients with Frederickson Type I or V hyperlipoproteinemia
CymaBay Therapeutics, Inc.
2-[4-[[(2R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate
Treatment of primary biliary cholangitis
CymaBay Therapeutics
2-[4-[[(2R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate
Treatment of homozygous familial hypercholesterolemia (HoFH)
CymaBay Therapeutics, Inc.
2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate
Treatment of acute myeloid leukemia
AstraZeneca Pharmaceuticals LP
2-{3-[(3R)-3-({[2-chloro-3-(trifluoromethyl)phenyl]methyl}(2,2-diphenylethyl)amino)butoxy]phenyl}acetic acid
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 26 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
152
n/a 6/16/2015 Rgenix, Inc.153
n/a 6/23/2015 Rgenix, Inc.154
n/a 8/28/2014155
n/a 12/10/2004 Treatment of stomach cancer Rexahn Corporation156
n/a 12/1/2004 Rexahn Corporation157
n/a 12/8/2004 Treatment of pancreatic cancer Rexahn Corporation158
n/a 12/1/2004 Treatment of ovarian cancer Rexahn Corporation159
n/a 12/8/2004 Treatment of glioblastoma Rexahn Corporation160
225Ac-lintuzumab n/a 11/25/2014
2-{3-[(3R)-3-({[2-chloro-3-(trifluoromethyl)phenyl]methyl}(2,2-diphenylethyl)amino)butoxy]phenyl}acetic acid
Treatment of glioblastoma multiforme.
2-{3-[(3R)-3-({[2-chloro-3-(trifluoromethyl)phenyl]methyl}(2,2-diphenylethyl)amino)butoxy]phenyl}acetic acid
Treatment of malignant melanoma stages IIB to IV.
2-{[(1r,4r)-4-(((4-chlorophenyl)(phenyl)carbamoyloxy)methyl)cyclohexy]methoxy} acetic acid
Treatment of pulmonary arterial hypertension
Arena Pharmaceuticals, Inc.
20-mer complementary to Akt mRNA
20-mer oligonucleotide complementary to Akt mRNA
Treatment of renal cell carcinoma
20-mer oligonucleotide complementary to Akt mRNA
20-mer oligonucleotide complementary to Akt mRNA
20-mer oligonucleotide complementary to Akt mRNA
Treatment of acute myelogenous leukemia
Actinium Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 27 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
16124,25 dihydroxycholecalciferol n/a 2/27/1987 Lemmon Company
1622S,4R ketoconazole n/a 3/9/2012
163n/a 4/30/2010
164
3,4-diaminopyridine n/a 12/18/1990165
3,5,3'-triiodothyroacetate n/a 9/20/2000166
3,5-diiodothyropropionic acid n/a 5/14/2013167
n/a 9/11/1998 Treatment of Kaposi's sarcoma. Sugen, Inc.168
n/a 3/23/2000 Sugen, Inc.169
n/a 7/14/2015
Treatment of uremic osteodystrophy.
Treatment of endogenous Cushing's syndrome
Cortendo AB (HQ address)
3,4 diaminopyridine and choline bitartrate
Treatment of Lambert-Eaton myasthenic syndrome
MS Therapeutics Limited
Treatment of Lambert-Eaton myasthenic syndrome.
Jacobus Pharmaceutical Company
Treatment of well-differentiated papillary, follicular or combined papillary/follicular carcinomas of the thyroid gland.
Elliot Danforth, Jr., M.D.
Treatment of Allan-Herndon-Dudley syndrome
Zarion Pharmaceuticals P/L
3-(3,5-dimethyl-1H-2ylmethylene)-1,3-dihydro-indol-2-one
3-(3,5-Dimethyl-1H-2ylmethylene)-1,3-dihydro-indol-2-one
Treatment of von Hippel-Lindau disease.
3-(4-(1,5-Napthrydine)-Imidazole[1,2-a]pyridine-7-(1-phenyl-4-(1-(4-methyl piperazine)
Treatment of fibrodysplasia ossificans progressiva.
La Jolla Pharmaceutical Company, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 28 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
170
n/a 7/14/2015171
n/a 9/19/2016 Celgene Corporation172
n/a 3/29/2016 Treatment of glioblastoma Actuate Therapeutics173
n/a 3/2/2010 Treatment of cystic fibrosis174
3-bromopyruvate n/a 3/5/2013175
3-bromopyruvate n/a 4/29/2014 Treatment of pancreatic cancer PreScience Labs, LLC176
n/a 10/25/2013 Treatment of Rett syndrome Neurolixis, Inc.177
n/a 7/28/2008 Seaside Therapeutics
3-(4-(8-fluoroquinoline)-Imidazole[1,2-a]pyridine-7-(1-phenyl-4-(1-(4-methyl piperazine)
Treatment of fibrodysplasia ossificans progressiva.
La Jolla Pharmaceutical Company, Inc.
3-(5-amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride
Treatment of diffuse large B-cell lymphoma
3-(5-Fluoro-benzofuran-3-yl)-4-(5-methyl-5H-[1,3]dioxolo[4,5-f]indol-7-yl)-pyrrole-2,5-dione
3-(6-(1-(2,2-difluorobenzo[d] [1,3]dioxyl-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
Vertex Pharmaceuticals Inc.
Treatment of liver and intrahepatic bile duct cancer
Primocure Pharma, Inc.
3-chloro-4-fluorophenyl-[4-fluoro-4-[[(5-methylpyrimidin-2-ylmethyl)amino]methyl]piperidin-1yl]methanone
3-fluoro-5-[5-(2-menthyl-thiazol-4-ylethylnyl)-pyridin-2-yl]-benzonitrile dihydrochloride
Treatment of behavioral abnormalities associated with fragile X syndrome.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 29 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
178n/a 2/11/2015
179
n/a 12/23/2014180
n/a 11/9/2015 Treatment of Proteus Syndrome. ArQule, Inc.181
10/30/2008182
10/30/2008183
10/23/2007184
n/a 4/18/2011185
n/a 9/6/2016 Ovid Therapeutics
3-pentylbenzenacetic acid sodium salt
Treatment of idiopathic pulmonary fibrosis.
ProMetic Life Sciences, Inc.
3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid
Prevention of acute attacks of angioedema in individuals with hereditary angioedema
BioCryst Pharmaceuticals, Inc.
3-{3-[4-(1-aminocyclobutyl)phenyl]-5-phenyl-3H-imidazo[4,5-b]pyridin-2-yl}cpyridin-2-amine bis-mesylate
4, 5 dibromorhodamine methyl ester
Theralux Photodynamic Therapy
Treatment of diffuse large B-cell lymphoma
Kiadis Pharma Canada, Inc.
4, 5 dibromorhodamine methyl ester
Theralux Photodynamic Therapy
Treatment of follicular lymphoma
Kiadis Pharma Canada, Inc.
4,5 dibromorhodamine methyl ester
Theralux Photodynamic Therapy
For immune reconstitution and prevention of graft versus host disease following allogeneic hematopoietic stem cell transplantation.
Kiadis Pharma Netherlands B.V.
4,5,6,7-tetrachloro-2',4',5',7'-tetraiodofluorescein disodium salt
Treatment of hepatocellular carcinoma.
Provectus Pharmaceuticals, Inc.
4,5,6,7-tetrahydroisoxazolo(5,4-c)pyridin-3-ol
Treatment of Angelman syndrome.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 30 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
186
n/a 10/11/2006187
n/a 12/12/2005188
n/a 3/24/2015 Clanotech AB189
n/a 3/27/2014 AbbVie190
n/a 4/15/2013191
n/a 5/4/2015 Gilead Sciences, Inc.
4-(2-fluorophenyl)-6-methyl-2-(1-piperzinyl)
Treatment of Chronic Functional Vomiting to include functional vomiting and cyclic vomiting syndrome.
Dynogen Pharmaceuticals, Inc.
4-(3-Methanesulfonyl-phenyl)-1-propylpiperidine HCl
Treatment of Huntington's disease.
Teva Branded Pharmaceutical Products R&D
4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid
Prevention of scarring post ab externo glaucoma surgery
4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide
Treatment of diffuse large B-cell lymphoma.
4-(6-(4-(piperazin-1-yl)phenyl_pyrazolo[1,5-a]pyrimidin-3-yl)quinoline hydrochloride
Treatment of fibrodysplasia ossificans progressiva
La Jolla Pharmaceutical Company, Inc.
4-(pyrimidin-2-ylmethyl)-7-[4-(trifluoromethoxy)phenyl]-3,4-dihydro-1,4-benzoxazepin-5(2H)-one
Treatment of congenital long QT syndrome
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 31 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
192
n/a 9/23/2014 Treatment of pancreatic cancer.193
n/a 8/22/2007194
4-Aminopyridine n/a 12/14/2005195
4-aminosalicylic acid 12/13/1989 Beeken, Warren M.D.196
4-aminosalicylic acid Paser Granules 4/26/2006197
n/a 7/20/2006198
n/a 9/29/2015
4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl) cyclopent-2-en-1-yl] pyrimidin-2-one
Rexahn Pharmaceuticals, Inc.
4-Amino-1-[5-O-[(2R, 4S)-2-oxido-4-(4-pyridinyl)-1, 3, 2-dioxaphosphorinan-2-yl]-b-D-arabinofuranosyl]-2(1H)-pyrimidinone
Treatment of hepatocellular carcinoma.
Ligand Pharmaceuticals, Inc.
Treatment chronic functional motor and sensory deficits from Guillain-Barre syndrome
Acorda Therapeutics, Inc.
Pamisyl (P-D), Rezipas (Squibb)
Treatment of mild to moderate ulcerative colitis in patients intolerant to sulfasalazine.
Treatment of acute flares in pediatric patients with ileo-cecal Crohn's disease
Jacobus Pharmaceutical Co., Inc.
4-cyano-N-[2-(1-cyclohexen-1-yl)-4-[1-[dimethylamino)acetyl]-4-piperidinyl]phenyl]-1H-imidazole-2-carboxamide monohydrochloride
Treatment of acute myeloid leukemia
Johnson & Johnson Pharmaceutical Research & Dev,
4-Hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl
Treatment of cerebral cavernous malformation.
REcursion Pharmaceuticals, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 32 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
199
n/a 11/2/2010 Treatment of pancreatic cancer.200
n/a 2/8/2016 Shire HGT, Inc.201
n/a 11/18/2015 ARMGO Pharma, Inc.202
4-[131I]iodo-L-phenylalanine n/a 1/4/2011 Treatment of glioma.203
n/a 7/30/2008 SuperGen, Inc.204
n/a 10/4/2011205
n/a 9/3/2009
4-pyridinylmethyl 3(4-chlorophenyl)adamantine carboxamide
Apogee Biotechnology Corporation
4-[(2E)-1-Oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide
Treatment of retinitis pigmentosa
4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate
Treatment of patients with Duchenne Muscular Dystrophy.
Therapeia GmbH & Co KG
4-[1]benzofuro[3,2-d]pyrimidin-4-yl-N-(1,3-benzodioxol-5-ylmethyl)piperazine-1-carbothioamide
Treatment, in combination with radiotherapy and temozolomide chemotherapy, of patients with glioblastoma multiforme.
4-[2-(3-Propyl-[1,2,4]0xadiazol-5-yl)-vinyl]-benzene-1,2-diol
Treatment of chronic myeloid leukemia
Piramal Enterprises Limited
4-[4-[[[[3-(1,1-dimethylethyl)-1-(6-quinolinyl)-1H-pyrazol-5-yl]amino]carbonyl]-amino]-3-fluorophenoxyl]-N-methyl-2-pyridinecarboxamide, p-toluenesulfonate salt
Treatment of Philadelphia chromosome positive chronic myeloid leukemia
Deciphera Pharmaceuticals, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 33 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
206
n/a 9/23/2011 Celgene Corporation207
n/a 5/19/2014 Roche Genentech208
n/a 3/18/2013 Novo Nordisk, Inc.209
n/a 3/31/2015210
5'-GCCATGGTTTTTTCTCAGG-3' n/a 10/31/2012211
n/a 6/15/2007
4-[[9-(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol
Treatment of idiopathic pulmonary fibrosis
4-{[(2R,3S,4R,5S)-4-(4-Chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid
Treatment of Acute Myeloid Leukemia
40K PEGylated recombinant factor IX
Routine prophylactic administration for prevention of bleeding in patients with hemophilia B (Christmas disease)
5'-CTATCTGUC*G1TTCTCTGU-3'.17 Na+
Treatment of diffuse large B-cell lymphoma.
Idera Pharmaceuticals, Inc.
Prophylaxis for patients following documented or suspected exposure to ebolavirus
Sarepta Therapeutics, Inc.
5(S)-(2'-hydroxy ethoxy)-20(S)-Camptothecin
Treatment of osteosarcoma (bone cancer)
Dr. Reddy's Laboratories, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 34 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
212
n/a 8/20/2003 Treatment of pancreatic cancer Now Pharm AG213
5,6-dihydro-5-azacytidine n/a 5/11/1992 ILEX Oncology, Inc.214
n/a 9/4/2014215
n/a 6/18/2007216
n/a 4/18/2011 Mithridion, Inc.217
n/a 11/21/2016218
n/a 1/15/2015 Gilead Sciences, Inc.219
n/a 11/22/2011
5,5',5"-[Phosphinothioylidyne-tris(imino-2,1-ethanediyl)]tris[5-methylchelidoninium]trihydroide hexahydrochloride
Treatment of malignant mesothelioma.
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride
Treatment of Huntington's disease
Prana Biotechnology Limited
5,7-dihydroxy-3-(4-hydroxyphenyl)-chromen-4-one
Prevention of acute radiation syndrome
Humanetics Corporation
5-(3-ethyl-1,2,4-oxadiazol-5-yl)-1,4,5,6-tetrahydropyrimidine hydrochloride
Treatment of progressive supranuclear palsy.
5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)-pyridin-2-amine
Treatment of primary central nervous system lymphoma.
PIQUR Therapeutics AG
5-(4-Cyclopropyl-1H-imidazol-l-yl)-2-fluoro-N-(6-(4-isopropyl-4H-1,2,4-triazol-3-yl)pyridin-2-yl)-4-methylbenzamide
Treatment pulmonary arterial hypertension
5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzp[d]oxazole
Treatment of Duchenne Muscular Dystrophy
Summit Corporation plc
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 35 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
220
5-aminolevulinic acid Gliolan 1/15/2013221
5-aza-2'-deoxycytidine n/a 8/3/1987 Treatment of acute leukemia. SuperGen, Inc.222
n/a 5/26/2006 Treatment of sickle cell disease Baxalta US, Inc.223
n/a 12/31/2015 Treatment of soft tissue sarcoma224
n/a 5/26/2006 Treatment of malginant glioma Hana Biosciences, Inc.225
Resveratrol 3/13/2008 Treatment of MELAS syndrome226
n/a 8/25/2009 CureSMA227
n/a 10/31/2012228
n/a 11/3/2016 Treatment of Rett syndrome
Visualization of malignant tissue during surgery for malignant glioma (WHO garde III and IV)
NX Development Corporation
5-hydroxymethyl-2-furfuraldehyde
5-imino-13-deoxydoxorubicin HCl
Gem Pharmaceuticals, LLC
5-iodo-2-pytimidinone-2'-deoxyribose
5-[(E)-2-(4-hydroxyphenyl)-ethenyl] benzene-1,3 diol
Sirtris Pharmaceuticals, Inc.
5-[1-(2,6-Dichlorobenzyl)-piperidin-4-yl]methoxyquinazoline-2,4-diamine
Treatment of spinal muscular atrophy
5ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬"-GAATATTAACAIACTGACAAGTC-3ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬"
Prophylaxis following documented or suspected exposure to marburg virus
Sarepta Therapeutics, Inc.
6'-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)- paromamine sulfate
Eloxx Pharmaceuticals, Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 36 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
229
n/a 2/6/2006 Treatment of pancreatic cancer230
n/a 8/22/2011231
n/a 9/26/2013232
n/a 9/4/2014 Treatment of sickle cell disease Pfizer, Inc.233
n/a 4/14/2014 Gilead Sciences, Inc.234
n/a 11/9/2016235
n/a 10/18/2016
6,8-bis-benzylsulfanyl-octanoic acid
Cornerstone Pharmaceuticals, Inc.
6,8-bis-benzylsulfanyl-octanoic acid
Treatment of acute myeloid leukemia.
Cornerstone Pharmaceuticals, Inc.
6,8-bis-benzylsulfanyl-octanoic acid
Treatment of myelodysplastic syndrome
Cornerstone Pharmaceuticals, Inc.
6-((3S,4S)-4-Methyl-1-pyrimidin-2-ylmethyl-pyrrolidin-3-yl-1-(tetrahydro-pyran-4-yl)-1,5-dihydro-pryazolo[3,4-d]pyrimidin-4-one
6-(1H-Indazol-6-yl)-N-(4-morpholinophenyl)imidazo-[1,2-alpha]pyrazin-8-amine bis-methanesulfonate
Treatment of chronic lymphocytic leukemia
6-(4-(4-(2,3-dichlorophenyl)-piperazin-1-yl)-butoxy)-2H-benzo[b][1,4]oxazin-3(4H)-one hydrochloride
Treatment of pulmonary arterial hypertension (WHO Group 1)
Reviva Pharmaceuticals, Inc.
6-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)- paromamine sulfate
Treatment of mucopolysaccharidosis type 1 (MPS I)
Eloxx Pharmaceuticals, Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 37 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
236
n/a 4/9/2008237
n/a 10/15/2013238
n/a 6/18/2015 Sixera Pharma AB239
n/a 9/7/2016240
6-mercaptopurine oral liquid n/a 12/7/2009 Orbona Pharma Ltd241
n/a 5/11/2016242
n/a 6/12/2014 Treatment of multiple myeloma243
n/a 2/11/2016
6-alpha-ethylchenodeoxycholic acid
Treatment of primary sclerosing cholangitis
Intercept Pharmaceuticals, Inc.
6-amino-5-chloro-N-[1R)-1-[5-[[[5-hloro-4-(trifluoromethyl)-2pyridinyl]amino]carbonyl]-2-thiazoyl]ethyl]-4-pyrimdinecarboxamide
Treatment of stage IIb-IV melanoma
Millennium Pharmaceuticals, Inc.
6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-bensoxazin-4-one
Treatment of Netherton syndrome
6-fluoro-(18F)-L-3,4-dihydroxyphenylalanine
A diagnostic for management of patients with Lesch-Nyhan syndrome
Advanced Imaging Projects, LLC
Treatment of acute lymphoblastic leukemia in the pediatric population
6-methoxy-3-(3',4',5'-trimethoxybenzoyl) indole
Treatment of cholangiocarcinoma
SynCore Biotechnology Co. Ltd.
6-[4-Deoxy-4-[(2E,4E)-tetradecadienoylglycyl]amino-L-glycero-B-L-manno-heptopyranosyl]amino-9H-purine
DARA BioSciences, Inc.
6-{4-[l-(propan-2-yl)piperidin-4-yl]-1,4-diazepan-1-yl}-N-(pyridin-4-yl)pyridine-2-carboxamide
Treatment of glioblastoma multiforme (GBM).
Upsher-Smith Laboratories, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 38 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
244
n/a 4/27/2016 MyoKardia, Inc.245
n/a 9/24/2014246
n/a 6/15/2011 Treatment of gliomas. Intsel Chimos SA247
n/a 6/23/2016 Treatment of glioblastoma. Oncoceutics, Inc.248
n/a 4/13/2016249
n/a 1/21/2015250
8-methoxsalen Uvadex 6/22/1993 Therakos, Inc.251
n/a 9/3/2009 Treatment of ovarian cancer.
6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione
Treatment of symptomatic obstructive hypertrophic cardiomyopathy
68G-OPS202, small somatostatin analog labeled with 68Gallium
Management of gastroenteropancreatic neuroendocrine tumors.
Ipsen Biosciences, Inc.
7-B-Hydroxy Cholesteryl-3-B-Oleate-Ester
7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one*2HCl
7-ethyl-10-hydroxycamptothecin (EHC)
Treatment of small cell lung cancer.
Nippon Kayaku Co., Ltd.
8-(1-Hydroxy-ethyl)-2-methoxy-3-(4-methoxy-benzyloxy)-benzo[c]chromen-6-one
Treatment of glioblastoma multiforme
Diffusion Pharmaceuticals, Inc.
For use in conjunction with the UVAR photopheresis to treat diffuse systemic sclerosis.
8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naph-thyrin-3(2H)-one mono-hydrochloride
Merck Sharp & Dohme Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 39 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
252
n/a 8/29/2016253
n/a 5/23/2016 Treatment of Friedreich's Ataxia Retroitope, Inc.254
9-nitro-20-(S)-camptothecin Camvirex 5/15/2001255
9-nitro-20-(S)-camptothecin n/a 9/16/1996 Treatment of pancreatic cancer. SuperGen, Inc.256
90Y-DOTA-tyr3-Octreotide n/a 1/20/2016257
90Y-hPAMA4 Pan-Cide 1/29/2004 Treatment of pancreatic cancer Immunomedics, Inc.258
n/a 4/29/2014 Advaxis, Inc.259
A-dmDT390-bisFv(UCHT1) Resimmune 10/9/2014 Angimmune, LLC260
a-Galactosidase A 1/21/2003 Treatment of Fabry's disease iBio, Inc.261
A10 & AS2-1 Antineoplaston n/a 9/3/2004
9,10-dimethoxy-5,6-dihydro-[1,3] dioxolo[4,5-g]isoquinolino[3,2-alpha]isoquinolin-7-ium(R)-4-((3R,5S,7S,8R,9S,10S,13R,14S,17R)-3,7-dihydroxy-10,13-dimethyhexadecahydro-1H-cyclopenta[alpha]
Treatment of primary sclerosing cholangitis
HighTide Biopharma Pty. Ltd.
9-cis, 12-cis-11,11-d2-linoleic acid ethyl ester
Treatment of pediatric HIV infection/AIDS
NovoMed Pharmaceuticals, Inc.
Treatment of neuroendocrine tumors
M. Sue O'Dorisio, MD, PhD
a live attenuated bioengineered Listeria monocytogenes cancer immunotherapy
Treatment of Stage II to IV invasive cervical carcinoma
Treatment of cutaneous T-cell lymphoma
Plant-Produced Human A-Glactosidase A
Treatment for patients with brain stem glioma
Burzynski Research Institute, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 40 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
262
n/a 9/12/2013263
AAV-G6Pase vector n/a 3/11/2013264
abatacept Orencia 5/30/2013 Orban Biotech LLC265
ABCA4 DNA nanoparticles n/a 10/28/2015266
n/a 4/18/2011 Cortice Biosciences267
n/a 10/7/2011 Treatment of gliomas Cortice Biosciences268
Abetimus n/a 7/28/2000 Treatment of lupus nephritis.269
n/a 4/20/2014270
acadesine n/a 5/4/2011 Treatment of multiple myeloma
aadeno-associated viral vector, serotype 2, containing the human choroideremia gene encoding human Rab escort protein 1
Treatment of choroideremia due to mutations in the human choroideremia gene (CHM)
Sparks Therapeutics, Inc.
Treatment of glycogen storage disease type Ia
GlyGenix Therapeutics, Inc.
Treatment of type 1 diabetes mellitus patients with residual beta cell function
Treatment of Stargardt Macular Degeneration
Copernicus Therapeutics, Inc.
abeotaxane inhibitor of microtubules
Treatment of pediatric neuroblastoma.
abeotaxane inhibitor of microtubules
La Jolla Pharmaceutical Co.
Ac-Ile-Cys-Val-Trp(1-Me)-Gln-Asp-Trp-Gly-Ala-His-Arg-Cys-Thr-AEEA-Lys-PEG 40KDa-Lys-AEEA-Thr-Cys-Arg-His-ALa-Gly-Trp-Asp-Gln-(1-Me)Trp-Val-Cys-Ile-Ac
Treatment of paroxysmal nocturnal hemoglobinuria
Apellis Pharmaceuticals, Inc.
Advancell-Advanced In Vitro Cell Technologies S.A.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 41 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
271
acadesine n/a 3/3/2011272
acamprosate n/a 3/25/2013 Treatment of fragile X syndrome273
aceneuramic acid n/a 9/23/2011274
n/a 11/24/2015 Genus Oncology, LLC275
acetyl-l-carnitine n/a 7/24/2012 Treatment of Fragile X syndrome276
acetyl-l-carnitine (ALC) n/a 3/18/2011 Treatment of Rett syndrome277
Acetylcysteine 8/13/1987278
ActRIIB-IgG1) n/a 8/16/2010
Treatment of chronic lymphocytic leukemia
Avanced In Vitro Cell Technologies, S.L.
Confluence Pharmaceuticals, LLC
Treatment of hereditary inclusion body myopathy
Ultragenyx Pharmaceutical, Inc.
acetyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-cysteinyl-D-glutaminyl-D-cystenyl-D-arginyl-D-arginyl-D-lysyl-D-asparaginylmaide disulfide
Treatment of acute myeloid leukemia.
Sigma-Tau Pharmaceuticals, Inc.
Sigma-Tau Pharmaceuticals, Inc.
Mucomyst/Mucomyst 10 Iv
Intravenous treatment of patients presenting with moderate to severe acetaminophen overdose.
Bristol-Myers Squibb Company
Treatment of Duchenne muscular dystrophy
Acceleron Pharma, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 42 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
279
acyclovir n/a 5/3/2016 Treatment of herpetic keratitis.280
acyclovir n/a 12/13/2010 Fera Pharmaceuticals281
n/a 3/17/2014 Treatment of ovarian cancer Oncos Therapeutics282
adalimumab Humira 5/11/2011 AbbVie, Inc.283
adalimumab n/a 7/10/2014 Treatment of Behcet's disease Mucora284
n/a 9/29/2015 REGENXBIO, Inc.285
n/a 3/17/2011 Fondazione Telethon286
n/a 5/6/2013 Lysogene
Cumulus Pharmaceuticals LLC
Treatment of acute herpetic keratitis caused by Herpes Simplex Virus type 1 and 2
Ad5/3-D24-granulocyte-macrophage colony stimulating factor (GMCSF)-encoding oncolytic adenovirus
Treatment of pediatric patients with ulcerative colitis
aden-associated virus vector serotype 9 expressing human a-L-iduronidase
Treatment of mucopolysaccharidosis Type I (MSP I).
adeno associated viral vector containing human ARSB gene
Treatment of mucopolysaccharidosis type VI (MPS VI; Maroteaux-Lamy syndrome)
adeno associated viral vector serotype rh.10 carrying the human SGSH and SUMF1 cDNAs
Treatment of mucopolysaccharidosis type IIIA (Sanfilippo type A syndrome)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 43 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
287
n/a 3/14/2014 Treatment of hemophilia B Baxalta US, Inc.288
n/a 5/21/2007 uniQure B.V.289
n/a 11/18/2015 LYSOGENE290
n/a 3/16/2016291
n/a 9/21/2015 Treatment of hemophilia B.
adeno associated virus with modified transthyretin and sequence encoding factor IX variant gene
Adeno-associated vector expressing the human lipoprotein lipase protein
Treatment of lipoprotein lipase deficiency
adeno-associated viral (AAV) vector serotype rh. 10 encoding the human N-sulfoglycosamine sulphohydrolase cDNA (SGSH)
Treatment of mucopolysaccharidosis type IIIA or Sanfilippo type A syndrome
adeno-associated viral serotype 2 vector under the regulatory control of a CMV promoter encoding the human tripeptidyl peptidase-1 (hTPP1) cDNA
Treatment of CLN2 disease (neuronal ceroid lipofuscinosis (NCL)) caused by TPP1 deficiency.
Spark Therapeutics, Inc.
adeno-associated viral vector composed of a bioengineered AAV capsid and a codon-optimized expression cassette encoding a high-specific activity variant of human coagulation factor IX
Spark Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 44 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
292
n/a 12/13/2012 treatment of retinitis pigmentosa293
n/a 9/15/2010 uniQure B.V.294
Coagulin-B 6/13/2001 Avigen, Inc.295
Coagulin-B 6/13/2001 Avigen, Inc.296
n/a 11/20/2013 Gen Sight Biologics297
n/a 8/31/2016 Solid GT, LLC
adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing DNA encoding a rhodopsin gene
Genable Technologies Limited
adeno-associated viral vector containing modified U11 snRNA
Treatment of Duchenne muscular dystrophy.
Adeno-associated viral vector containing the gene for human coagulation factor IX
Intrahepatic treatment of patients with moderate to severe hemophilia
Adeno-associated viral vector containing the gene for human coagulation factor IX
Intramuscular treatment of patients with moderate to severe hemophilia
adeno-associated viral vector containing the human NADH Dehydrogenase 4 Gene
Treatment of Leber Hereditary Optic Neuropathy
Adeno-associated viral vector differentially expressing microdystrophin 5 gene via the muscle-specific promoter CK8.
Treatment of Duchenne muscular dystrophy.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 45 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
298
n/a 3/20/2007 Treatment of Pompe disease299
n/a 5/21/2007300
n/a 1/31/2012301
n/a 12/31/2015302
n/a 11/5/2014 Treatment of choroideremia NightstaRx Ltd.303
n/a 12/27/2012304
Calmarythm 10/2/2014
Adeno-associated viral vector expressing human acid alpha glucosidase gene
Audentes Therapeutics, Inc.
adeno-associated viral vector expressing human retinoschisin-1 gene
Treatment of X-linked juvenile retinoschisis (XLRS).
Applied Genetic Technologies Corporation
Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor
Treatment of homozygous familial hypercholesterolemia
ReGenX Biosciences LLC
adeno-associated viral vector expressing the human vascular endothelial growth factor gene
Treatment of critical limb ischemia in no-option patients where other procedures, grafts, or angioplasty are not indicated
Integene International Holding, LLC
adeno-associated viral vector serotype 2 containing the human Rab escort protein 1 gene
adeno-associated viral vector serotype 9 containing human N-acetylglucosaminidase alpha gene
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Laboratorios del Dr. Esteve, S.A.
adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene
Treatment of catecholaminergic polymorphic ventricular tachycardia
Audentes Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 46 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
305
n/a 3/18/2015306
n/a 9/19/2016 Milo Biotechnology307
n/a 9/1/2016 Treatment of hemophilia B308
n/a 9/30/2014 AveXis, Inc.309
n/a 7/16/2015310
n/a 8/25/2015 Treatment of hemophilia B.311
n/a 11/19/2012 Milo Biotechnology
adeno-associated viral vector type 2 expressing human recombinant retinal pigment epithelial 65KDa protein gene
Treatment of inherited retinal dystrophy due to biallelic RPE65 gene mutations
Spark Therapeutics, Inc.
Adeno-associated virus delivered transgene of follistatin
Treatment of inclusion body myositis
Adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX)
Sangamo BioSciences, Inc.
adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene
Treatment of spinal muscular atrophy
adeno-associated virus serotype 9 vector containing human Iduronate-2-sulfatase transgene
Treatment of mucopolysaccharidosis type II (Hunter syndrome).
Laboratorios del Dr. Esteve, S.A.
Adeno-associated virus serotype rh10 vector encoding the human factor IX gene
Dimension Therapeutics
adeno-associated virus transgene of follistatin
Treatment of Duchennes and Becker's muscular dystrophy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 47 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
312
n/a 11/16/2015313
n/a 2/29/2016 Treatment of Hemophilia A.314
n/a 6/1/2011315
n/a 9/4/2014316
n/a 3/3/2011 Eli Lilly and Company
adeno-associated virus type 8 delivering a vector genome with human retinoschisin promoter (RS/IRBP) and the human retinoschisin cDNA (hRS)
Treatment of X-linked retinoschisis
Paul A. Sievig, MD, PhD, National Eye Institute, NIH
adeno-associated virus vector encoding the SQ variant of human blood clotting factor VIII
BioMarin Pharmaceutical, Inc.
adeno-associated virus vector serotype 9 expressing human sulfamidase
Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A Syndrome).
Laboratorios del Dr. Esteve, S.A.
Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter
For the treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)
Columbia University Medical Center
Adenosine triphosphate type 1 competitive inhibitor of JAK2 V617F tyrosine kinase
Treatment of myeloproliferative disorders (polycythemia vera, essential throbocythemia, and myelofibrosis)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 48 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
317
n/a 6/17/2005 Advantagene, Inc.318
n/a 6/8/2015319
n/a 7/23/2015 Treatment of malignant glioma.320
n/a 3/10/2016 MeiraGTx Limited321
n/a 11/29/2016 MeiraGTx Limited322
n/a 3/10/2016 MeiraGTx Limited323
n/a 4/2/2012 Treatment of malignant glioma Vascular Biogenics Ltd324
n/a 10/1/2014 Treatment of glioma DNAtrix, Inc.
Adenoviral vector expressing Herpes simplex virus thymidine kinase gene
Treatment of malignant brain tumors
adenoviral vector expressing the E.coli purine nucleoside phosphorylase gene and fludarabine
Intratumoral treatment of anatomically accessible oral and pharyngeal cancers (lip, tongue, gum, floor of mouth, salivary gland, and other oral cavity)
PNP Therapeutics, Inc.
Adenoviral vector-RheoSwitch Therapeutic System-human interleukin 12 + veledimex
Ziopharm Oncology, Inc.
adenovirus associated viral vector serotype 5 containing the human RPE65 gene
Treatment of Leber congenital amaurosis
adenovirus associated viral vector serotype 5 containing the RPGR gene
Treatment of retinitis pigmentosa
adenovirus associated viral vector serotype 8 containing the human CNGB3 gene
Treatment of achromatopsia due to mutations in the CNGB3 gene.
adenovirus containing a human FAS-c gene
adenovirus delta 24-RGD, an oncolytic adenovirus genetically modified for treatment of glioma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 49 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
325n/a 4/19/2016 Memgen LLC
326
n/a 9/4/2014 Etubics Corporation327
n/a 7/14/2015 Treatment of mesothelioma MTG Biotherapeutics328
n/a 7/5/2016 HORAMA, SAS329
Miniadfviii 12/15/1999 Treatment of hemophilia A.330
Multistem 7/6/2012 Athersys, Inc.331
n/a 5/27/2015 Treatment of aplastic anemia HemoGenyx LLC332
ADXS11-001 n/a 8/12/2013 Advaxis, Inc.333
aerosolized beractant n/a 3/11/2013334
n/a 4/30/2010 Treatment of ovarian cancer Aeterna Zentaris
Adenovirus encoding the recombinant CD40 ligand
Treatment of stage IIb through IV melanoma.
Adenovirus serotype-5 (Ad5) vector that contains a modified non-oncogenic fused early 6 (E6) and early 7 (E7) gene of the human papillomavirus (HPV); (Ad5 [E1-, E2b-]-E6/E7)
Treatment of human papillomavirus (HPV)-associated head and neck squamous cell carcinoma (HNSCC)
adenovirus vaccine encoding reduced expression in immortalized cell protein
adenovirus-associated viral vector serotype 5 containing the human pde6B gene
Treatment of retinitis pigmentosa due to pde6B gene mutations
Adenovirus-based vector Factor VIII complementary DNA to somatic cells
GenStar Therapeutics Corporation
adult adherent bone marrow-derived multipotent stem cells
Treatment of MPS-1, including Hurler syndrome
adult hemogenic endothelial cells
Treatment of HPV-positive associated anal cancer
Treatment of respiratory distress syndrome
Beena G. Sood, MD, MS
AEZS-108 (LHRH-agonist linked to doxorubicin)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 50 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
335afamelanotide n/a 7/17/2008 Clinuvel Inc.
336 afamelanotide n/a 12/11/2009 Treatment of solar urticaria Clinuvel, Inc.337
afamelanotide n/a 5/14/2014 Clinuvel, Inc.338
afamelanotide n/a 2/4/2016 Clinuvel Inc.339
afatinib Gilotrif(R) 6/4/2014340
aganirsen n/a 11/7/2016341
Albuterol n/a 3/12/2002 MotoGen, Inc.342
Aldesleukin Proleukin 11/24/1998343
Aldesleukin Proleukin 3/22/1989344 aldoxorubicin n/a 9/24/2014 Treatment of ovarian cancer CytRx Corporation345
aldoxorubicin n/a 9/24/2014 CytRx Corporation346
aldoxorubicin n/a 9/24/2014 CytRx Corporation
Treatment of erythropoietic porphyrias
Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)
Treatment of cutaneous variants of porphyria (which includes treatment and prevention of cutaneous manifestations of disease)
Treatment of malignant brain and central nervous system tumors
Boehringer Ingelheim Pharmaceuticals, Inc.
Prevention of corneal graft rejection.
Gene Signal International SA
Prevention of paralysis due to spinal cord injury
For the treatment non-Hodgkin's lymphoma.
Prometheus Laboratories, Inc.
Treatment of primary immunodeficiency disease associated with T-cell defects.
Prometheus Laboratories, Inc.
Treatment of glioblastoma multiforme
Treatment of small cell lung cancer
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 51 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
347
Alendronate disodium Fosamax 2/13/2001348
Alfentanil n/a 7/8/2005 Cinergen, LLC349
Alfentanil n/a 8/9/2005 Cinergen, LLC350
alfimeprase n/a 9/20/2012351
algenpantucel-L 10/21/2010 Treatment of pancreatic cancer.352
Alglucerase injection Ceredase 7/21/1995 Genzyme Corporation353
alicaforsen n/a 6/24/2008 Treatment of pouchitis.354
alisertib n/a 7/12/2013355
Alitretinoin Panretin 4/10/1992356
Retriacyl 5/21/2014 Natac Pharma, S.L.
Treatment of the bone manifestations of Gaucher disease
Richard J. Wenstrup, M.D.
Management of postherpetic neuralgia
Treatment of painful HIV-associated neuropathy
Treatment of ST-elevation myocardial infarcted patients who are undergoing primary percutaneous coronary intervention
Niche Therapeutics, LLC
Hyperacute(R)-Pancreatic Cance
NewLink Genetics Corporation
Replacement therapy in patients with Type II and III Gaucher's disease.
Atlantic Healthcare Limited
Treatment of small cell lung cancer
Millennium Pharmaceuticals, Inc.
Treatment of acute promyelocytic leukemia
Ligand Pharmaceuticals, Inc.
all-cis-docosa-4,7,10,13,16,19-hexaenoic acid
Treatment of retinitis pigmentosa
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 52 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
357
allantoin Alwextin 11/21/2002 Scioderm, Inc.358
n/a 4/21/2015 Capricor, Inc.359
n/a 12/2/2010360
n/a 2/4/2016361
n/a 2/18/2013 Treatment of Aplastic Anemia362
n/a 12/29/2015363
n/a 8/22/2011
Treatment of skin blistering and erosions associated with inherited epidermolysis bullosa
allogeneic cardiosphere-derived cells
Treatment of Duchenne Muscular Dystrophy
allogeneic cord blood cells, ex vivo modulated with 16,16 dimethyl prostaglandin E2 (dmPGE2)
Enhancement of stem cell engraftment through ex vivo treatment of human allogeneic hematopoietic stem cells (treatment of neuropenia, thrombocytopenia, lymphopenia, and anemia).
Fate Therapeutics, Inc.
allogeneic Epstein-Barr virus cytotoxic T lymphocytes
Treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disorders.
ATARA Biotherapeutics, Inc.
allogeneic ex-vivo expanded placental adherent stromal cells
Pluristem Therapeutics, Inc.
allogeneic ex-vivo expanded placental adherent stromal cells
For the treatment of severe preeclampsia
Pluristem Therapeutics, Inc
allogeneic ex-vivo expanded placental adherent stromal cells
Treatment of thromboangiitis obliterans (Buerger's disease)
Pluristem Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 53 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
364
n/a 4/20/2016 IPD-Therapeutics BV365
Atir 2/2/2010366
Vascugel (R) 4/3/2009 Shire367
Motorgraft (Tm) 1/17/2014 Caladrius Biosciences368
Cytoimplant 6/13/1997 Treatment of pancreatic cancer
allogeneic ex-vivo generated CD56+CD3-natural killer cells from CD34+ umbilical cord blood progenitor cells
Treatment of acute myeloid leukemia
allogeneic hematopoietic stem cell
Prevention (reduction) of transplant related mortality (TRM, which is caused by graft versus host disease and/or infections) following mismatched (haploidentical) allogeneic hematopoietic stem cell transplantation.
Kiadis Pharma Netherlands B.V.
Allogeneic human aortic endothelial cells cultured in a porcine gelatin matrix
Prevention of arteriovenous fistula or arteriovenous graft failure in patients with end satge renal disease receiving hemodialysis or preparing for hemodialysis
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Treatment of Amyotrophic Lateral Sclerosis
Allogeneic peripheral blood mononuclear cells sensitized against patient alloantigens by mixed lymphocyte culture
Applied Immunotherapeutics, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 54 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
369
n/a 9/1/2004 Neurotech USA, Inc.370
n/a 1/28/2005 MolMed S.p.A.371
n/a 7/24/2012 Neurotech USA, Inc.372
allopregnanolone n/a 7/12/2013373
allopregnanolone n/a 4/20/2014 Treatment of status epilepticus Sage Therapeutics374
n/a 3/24/2015375
alpha melanotropin n/a 9/2/2010
Allogeneic retinal epithelial cells transfected with plasmid vector expressing ciliary neurotrophic growth factor
Treatment of retinitis pigmentosa
Allogeneic T-cells cultured with anti-CD3 and IL-2; transduced with retroviral vector (SFCMM-3), expressing herpes simplex 1 virus-thymidine kinase (HSV-TK) and truncated low affinity nerve growth factor receptor; selected with anti-low affinity nerve gro
Immunotherapy for acceleration of T-cell reconstitution in patients undergoing allogeneic hematopoietic stem cell transplantation
allogenic retinal epithelial cells transfected with plasmid vector expressing ciliary neurotrophic growth factor
Treatment of macular telangiectasia type 2 (MacTel)
Treatment of Neimann-Pick disease, type C
La Jolla Pharmaceutical Company, Inc.
allosteric activator of the red blood cell-specific form of pyruvate kinase
Treatment of pyruvate kinase deficiency
Agios Pharmaceuticals, Inc.
Treatment of chronic beryllium disease
mondoBIOTECH Laboratories AG
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 55 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
376Glassia 10/23/2014 Kamada Ltd.
377Glassia 7/28/2011 Kamada, Ltd.
378
Alpha-galactosidase A Cc-Galactosidase 6/17/1991 David Calhoun, Ph.D.379
Alpha-galactosidase A Fabrase 7/20/1990 Treatment of Fabry's disease.380
Alpha-galactosidase A Replagal 6/22/1998381
n/a 8/19/1997382
Alpha-tocopherol quinone n/a 3/28/2006383
alpha-tocotrienol quinone n/a 10/21/2010384
n/a 1/29/2010385 Alpha1-antitrypsin(human) n/a 4/3/2008 Treatment of bronchiectasis Kamada Ltd.386
n/a 2/20/2009 Treatment of cystic fibrosis CSL Behring LLC387
n/a 4/6/2012 Treatment of cystic fibrosis
alpha-1 proteinase inhibitor (human)
Treatment of graft versus host disease
alpha-1 proteinase inhibitor (human)
Treatment of patients with recent onset (
Treatment of alpha-galactosidase A deficiency (Fabry's disease).
Desnick, Robert J. M.D.
Long-term enzyme replacement therapy for the treatment of Fabry disease
Shire Human Genetic Therapies, Inc.
Alpha-melanocyte stimulating hormone
Prevention and treatment of intrinsic acute renal failure due to ischemia.
National Institute of Diabetes, and Digestive and Kidney Diseases
Treatment of inherited mitochondrial respiratory chain diseases
Penwest Pharmaceuticals Company
Treatment of inherited mitochondrial respiratory chain diseases.
Edison pharmaceuticals, Inc.
alpha1 proteinase inhibitor (human)
Treatment of emphysema secondary to congenital alpha1-antitrypsin deficiency.
Grifols Therapeutics, Inc.
alpha1-proteinase inhibitor (human)
alpha1-proteinase inhibitor (human)
Grifols Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 56 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
388
Prolastin(R)-C 3/3/2015389
n/a 12/22/2004 Kamada Ltd.390
Arc-Api 9/1/2004 Treatment of cystic fibrosis Kamada Ltd.391
alteplase Activase 1/27/2003 Daniel F. Hanley, MD392
altiratinib n/a 8/19/2014393
alvocidib n/a 4/21/2014394
Alvocidib n/a 4/13/2007395
Amantadine HCl n/a 7/20/2015396
Amantadine hydrochloride Nurelin 4/9/2015397 amatuximab n/a 9/28/2012 Treatment of mesothelioma Morphotek, Inc.398
n/a 8/22/2016 Gilead Sciences, Inc.
alpha1-proteinase inhibitor (human)
Treatment of Type 1 diabetes mellitus patients with residual beta-cell function.
Grifols Therapeutics, Inc.
Alpha1-Proteinase Inhibitor (Human)
Inhalation therapy for the treatment of congential deficiency of alpha1-proteinase inhibitor.
Alpha1-Proteinase Inhibitor (Human)
Treatment of intraventricular hemorrhage associated with intracerebral hemorrhage
Treatment of glioblastoma multiforme.
Deciphera Pharmaceuticals, LLC
Treatment of acute myeloid leukemia
Tolero Pharmaceuticals, Inc.
Teatment of B-cell chronic lymphocytic leukemia (B-CLL) or prolymphocytic leukemia arising from CLL.
Tolero Pharmaceuticals, Inc.
Treatment of levodopa-induced dyskinesia
Osmotica Pharmaceutical Corporation
Treatment of levodopa-induced dyskinesia
Adamas Pharmaceuticals, Inc.
ambrisentan/tadalafil fixed combination
Treatment of pulmonary arterial hypertension
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 57 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
399ambroxol n/a 6/29/2011 Treatment of Gaucher disease Belrose Pharma, Inc.
400amifampridine phosphate n/a 3/3/2015
401
amifampridine phosphate n/a 11/12/2009402
amifampridine phosphate n/a 8/31/2016 Treatment of myasthenia gravis403
Amifostine Ethyol 11/24/1998 Clinigen Group plc404
Amifostine Ethyol 10/4/1999 Clinigen Group plc405
Amifostine Ethyol 5/30/1990 Clinigen Group plc406
Amifostine Ethyol 5/30/1990 Clinigen Group plc407
amikacin sulfate n/a 1/5/2015 PlumeStars s.r.l.408
Aminocaproic acid Caprogel 1/6/1995
Treatment of congenital myasthenic syndromes
Catalyst Pharmaceuticals
Treatment of Lambert-Eaton Myasthenic Syndrome
Catalyst Pharmaceuticals, Inc.
Catalyst Pharmaceuticals, Inc.
For the reduction of the incidence and severity of toxicities associated with cisplatin administration.
Treatment of myelodysplastic syndromes.
For use as a chemoprotective agent for cyclophosphamide in the treatment of advanced ovarian carcinoma.
For use as a chemoprotective agent for cisplatin in the treatment of metastatic melanoma.
Management of cystic fibrosis patients with Pseudomonas aeruginosa lung infections
For the topical treatment of traumatic hyphema of the eye.
Eastern Virginia Medical School
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 58 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
409
Levulan 3/20/2007410
Aminosidine Paromomycin 9/9/1994411
Aminosidine Gabbromicina 11/15/1993412
Aminosidine Gabbromicina 5/14/1993 Treatment of tuberculosis.413
aminosidine Paromomycin 3/29/2005414
Amiodarone Amio-Aqueous 8/17/1993415
amitriptyline and ketamine Amiket(Tm) 1/19/2010416
n/a 5/5/2008417
Coprexa 1/31/1994 Treatment of Wilson's disease.418
amphotericin B n/a 9/15/2010 iCo Therapeutics Inc.
aminolevulinic acid hydrochloride
Treatment of esophageal dysplasia
DUSA Pharmaceuticals, Inc.
Treatment of visceral leishmaniasis (kala-azar).
Kanyok, Thomas P. Pharm.D.
Treatment of Mycobacterium avium complex.
Kanyok, Thomas P. Pharm.D.
Kanyok, Thomas P. Pharm.D.
Treatment of visceral leishmaniasis
The Institute for One World Health
Treatment of incessant ventricular tachycardia.
Academic Pharmaceuticals, Inc.
Treatment of postherpetic neuralgia
Immune Pharmaceuicals, Inc.
ammonium tetrathiomolybdate
Treatment of idiopathic pulmonary fibrosis
Pipex Pharmaceuticals, Inc.
Ammonium tetrathiomolybdate
Pipex Pharmaceuticals, Inc.
Treatment of visceral leishmaniasis.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 59 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
419
n/a 12/15/2005420
Cyanide Antidote Kit 12/18/2006 Treatment of cyanide poisoning Akorn, Inc.421
amylopectin n/a 3/24/2015422
n/a 12/22/2011 Treatment of hemophilia B uniQure, B.V.423
n/a 7/7/2015 Treatment of Ewing sarcoma424
Anagrelide Agrylin 7/14/1986425
Anagrelide Agrylin 6/11/1985 Treatment of polycythemia vera.426
Ananain, comosain Vianain 1/21/1992 Genzyme Corporation
Amphotericin B inhalation powder
Prevention of pulmonary fungal infections in patients at risk for aspergillosis due to immunosuppressive therapy including those receiving organ or stem cell transplants, or treated with chemotherapy or radiation for hematologic malignancies
Novartis Pharmaceuticals Corporation
Amyl nitrite, sodium nitrite, sodium thiosulfate
Treatment of glycogen storage disease types Ia and Ib
Michael K. Davis, MD, MBA
an adeno-associated viral vector containing a codon-optimised human factor IX gene (AAV5-hFIXco)
ANA-conjugated dactunomycin nanoemulsion
NanoSmart Pharmaceuticals, Inc.
Treatment of thrombocytosis in chronic myelogenous leukemia.
Roberts Pharmaceutical Corp.
Roberts Pharmaceutical Corp.
For the enzymatic debridement of severe burns.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 60 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
427
Anatibant n/a 4/15/2005 Xytis, Inc.428
Ancrod Viprinex 10/20/1989429
andexanet alfa n/a 2/23/2015430
anfibatide n/a 3/10/2016431
Angiotensin (1-7) n/a 2/8/2016 Tarix Orphan, LLC432
Angiotensin (1-7) n/a 9/1/2016 Tarix Orphan, LLC
Treatment of patients having experienced a severe traumatic brain injury (Glasgow Coma Scale 3 to 8) in order to decrease early mortality and improve long-term functional and neurological outcome
To establish and maintain anticoagulation in heparin-intolerant patients undergoing cardiopulmonary bypass.
Knoll Pharmaceutical Company
For reversing the anticoagulant effect of direct or indirect factor Xa inhibitors in patients experiencing a serious uncontrolled bleeding event or who require urgent or emergent surgery
Portola Pharmaceuticals
Treatment of thrombotic thrombocytopenic purpura.
Lee's Pharmaceutical (Hong Kong) Limited
Treatment of LAMA2-related muscular dystrophy
Treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 61 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
433Angiotensin (1-7) n/a 11/26/2013 US Biotest, Inc.
434
angiotensin (1-7) n/a 8/30/2013 US Biotest, Inc.435
angiotensin (1-7)[A(1-7)] n/a 7/25/2013 US Biotest, Inc.436
Angiotensin 1-7 n/a 2/16/2000437
angiotensin 1-7 n/a 1/29/2010 Treatment of sarcoma. W. Jeffrey Petty, MD438
Angiotensin 1-7 Marstem 8/3/2001439
angiotensin 1-7 n/a 6/17/2010 US Biotest, Inc.
Treatment of limb-girdle muscular dystrophy
Treatment of patients requiring stem cell transplantation to accelerate the mobilization of hematopoietic stem cells (CD34+) from the bone marrow to the peripheral blood when combined with a granulocyte colony-stimulating factor
Treatment of Duchenne muscular dystrophy
Treatment of neutropenia associated with autologous bone marrow transplantation.
Maret Pharmaceuticals
Treatment of myelodysplastic syndrome
Maret Pharmaceutical Corporation
To accelerate engraftment of hematopoietic cells (treatment of neutropenia, thrombocytopenia, lymphoma, and aneima)in hematopoietic stem cell transplants.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 62 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
440Angiotensin-(1-7) n/a 9/13/2011 US Biotest, Inc.
441anisina n/a 7/14/2015 Treatment of neuroblastoma. Novogen Ltd
442anlotinib hydrochloride n/a 12/31/2015 Treatment of ovarian cancer
443n/a 11/17/2014 ChemoCentryx, Inc.
444
n/a 6/2/2014 ChemoCentryx, Inc.445
n/a 12/23/2014446
n/a 10/20/2008447
n/a 8/7/2014 Treatment of glioblastoma OncoSynergy, Inc.448
n/a 1/13/2015 Treatment of ovarian cancer OncoSynergy, Inc.449
n/a 9/13/2013 Xenikos BV
Treatment of pulmonary arterial hypertension.
Advenchen Laboratories, LLC
antagonist of the complement 5a receptor
Treatment of atypical hemolytic uremic syndrome
antagonist of the complement 5a receptor
Treatment of anti-neutrophil cytoplasmic autoantibodies associated vasculitides (granulomatosis with polyangiitis or Wegener's granulomatosis), microscopic polyangiitis, and Churg-Strauss syndrome.
antagonist of the endosomal Toll-like receptors (TLRs) 7, 8, and 9
Treatment of Waldenstrom's macroglobulinemia
Idera Pharmaceuticals, Inc.
anti human Nogo-A human monoclonal antibody
Treatment of acute spinal cord injury
Novartis Pharmaceuticals Corporation
Anti-Beta1 integrin monoclonal antibody
anti-Beta1 integrin monoclonal antibody
anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein and anti-CD7 mAb (WT1)-ricin A chain fusion protein
Treatment of graft versus host disease
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 63 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
450
n/a 12/2/2008 Treatment of Hodgkin lymphoma Xencor, Inc.451
n/a 5/13/2015452
anti-HER2 bispecific antibody n/a 5/18/2016 Treatment of ovarian cancer Zymeworks Inc.453
n/a 7/6/2016 Treatment of ovarian cancer Zymeworks Inc.454
n/a 11/18/2003455
n/a 3/9/2012 Treament of ovarian cancer456
n/a 9/23/2013 Treatment of cystic fibrosis457
n/a 8/29/2016 Treatment of pancreatic cancer458
n/a 4/3/2009
anti-CD30 Fc engineered humanized monoclonal antibody
Anti-eTau Humanized IgG4 Monoclonal Antibody
Treatment of Progressive Supranuclear Palsy
Britol-Myers Squibb Company
anti-HER2 bispecific antibody drug conjugate
anti-interferon-gamma Fab from goats
For the treatment of immunologic corneal allograft rejection
Advanced Biotherapy, Inc.
anti-Lewis Y humanized monoclonal antibody
Recepta Biopharma S.A.
anti-sense oligonucleotide consisting of 2ÿƿÿ⿿ÿ⿿ÿ¢ÿƿÿ¢ÿ¢â⿿¬ÿ¡ÿ⿿ÿ¬ÿƿÿ¢ÿ¢â⿿¬ÿ¾ÿ⿿ÿ¢0-Me RNA with a phosphorothioate backbone
ProQR Therapeutics III B.V.
anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody
Singh Biotechnology, LLC
Anti-T cell receptor murine monoclonal antibody
Prophylaxis of acute rejection of solid organ transplantation
Tolera Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 64 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
459
n/a 9/12/2008460
Atg-Fresenius(R) 3/26/2010461
n/a 1/12/2011462
n/a 6/7/2010463
Neuradiab 10/4/2005464
Thymoglobulin 5/25/2010 Genzyme Corporation465
Anti-thymocyte serum 6/2/1993466
n/a 11/17/2014467
Neovastat (Ae-941) 10/16/2002 AEterna Zentaris, Inc.
anti-T-lymphocyte immune globulin, rabbit
Prophylaxis of acute allograft rejection in adult recipients in solid organ transplantation
Neovii Biotech NA, Inc.
anti-T-lymphocyte immune globulin, rabbit
Prevention of graft versus host disease (GVHD)
Neovii Biotech NA, Inc.
anti-TCR murine monoclonal antibody (MAB, type IgM)
Treatment of pediatric multiple sclerosis in patients less than or equal to 16 years of age
Tolera Therapeutics, Inc.
anti-TCR murine monoclonal antibody (MAb, type IgM)
Treatment of patients 16 years of age and younger with immune-mediated T1DM and preserved pancreatic beta cell function
Tolera Therapeutics, Inc.
Anti-tenascin 81C6 monoclonal antibody labeled w/ I 131
Treatment of primary malignant brain tumors
Bradmer Pharmaceuticals, Inc.
anti-thymocyte globulin [rabbit]
Prophylaxis of acute organ rejection in patients receiving renal transplants
Nashville Rabbit Anti-Thymocyte Serum
Treatment of allograft rejection, including solid organ (kidney, liver, heart, lung, and pancreas) and bone marrow transplantation.
Applied Medical Research
anti-tumor necrosis factor (TNF) polyclonal antibody (bovine)
Treatment of pediatric ulcerative colitis (0 through 16 years of age)
Avaxia Biologics, Incorporated
antiangiogenic components extracted from marine cartilage
Treatment of renal cell carcinoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 65 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
468
Antiepilepsirine n/a 3/23/1989 Children's Hospital469
n/a 7/5/2012 Treatment of mesothelioma470
n/a 11/21/2008 Treatment of gliomas471
n/a 2/3/2015 Treatment of Ewing's sarcoma.472
n/a 4/15/2005473
Gti-2040 3/12/2003
Treatment of drug resistant generalized tonic-clonic epilepsy in children and adults.
antimesothelin-ADC (antibody drug conjugate)
Bayer HealthCare Pharmaceuticals, Inc.
antineoplaston A10, antineoplaston AS2-1
Burzynski Research Institute, Inc.
antinuclear antibody conjugated liposomal doxorubicin
NanoSmart Pharmaceuticals, Inc.
Antisense 20-mer oligonucleotide complementary to R2 component of ribonucleotide reductase mRNA
Treatment of acute myeloid leukemia
Lorus Therapeutics, Inc.
antisense 20-mer phosphorothioate oligonucleotide [complementary to the coding region of R2 component of the human ribonucleotide reductase mRNA]
Treatment for renal cell carcinoma
Lorus Therapeutics, Inc
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 66 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
474
n/a 5/25/2016475
Nexagon 4/27/2009476
n/a 9/19/2016 Biogen477
n/a 7/24/2012478
n/a 12/29/2015479
n/a 6/16/2016 Wave LIfe Sciences
antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA
Treatment of Leber's congenital amaurosis.
ProQR Therapeutics IV B.V.
Antisense oligonucleotide directed against connexin43
Treatment of persistent corneal epithelial defects
CoDa Therapeutics, Inc.
antisense oligonucleotide inhibitor of the expression of superoxide dismutase 1 gene
Treatment of amyotrophic lateral sclerosis
antisense oligonucleotide targeted to human transthyretin (TTR) mRNA
Treatment of familial amyloid polyneuropathy
Isis Pharmaceuticals, Inc.
Antisense oligonucleotide targeted to the huntingtin protein
Treatment of Huntington's disease
Isis Pharmaceuticals, Inc.
antisense oligonucleotide targeting the U isoform of SNP rs362307
Treatment of Huntington's disease (prodromal stage Huntington's disease and diagnosed Huntington's disease)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 67 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
480
Antithrombin III human Antithrombin Iii Human 1/2/1986481
n/a 2/12/1991482
antroquinonol Hocena(R) 4/30/2015483
antroquinonol n/a 7/23/2015484
antroquinonol n/a 1/21/2015 Treatment of pancreatic cancer485
aphidicolin n/a 4/20/2016 Biospherics.net LLC486
n/a 12/3/2012
Preventing or arresting episodes of thrombosis in patients with congenital AT-III deficiency and/or to prevent the occurrence of thrombosis in patients with AT-III deficiency who have undergone trauma or who are about to undergo surgery or parturition.
American National Red Cross
Antivenom (crotalidae) purified (avian)
Treatment of envenomation by poisonous snakes belonging to the Crotalidae family.
Ophidian Pharmaceuticals, Inc.
Treatment of acute myeloid leukemia
Golden Biotechnology Corporation
Treatment of hepatocellular carcinoma
Golden Biotechnology Corporation
Golden Biotechnology Corp.
Treatment of Ebola virus infection
apolipoprotein E mimetic peptide
Treatment of homozygous familial hypercholesterolemia
LipimetiX Development, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 68 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
487
Apomorphine n/a 7/17/1995488
Apomorphine hydrochloride n/a 5/23/2006489
apremilast n/a 1/17/2013 Treatment of Behcet's disease Celgene Corporation490
n/a 9/12/2008 Treatment of malaria491
Arenegyr n/a 8/22/2008 MolMed S.p.A.492
Arginine butyrate n/a 4/7/1992493
arimoclomol n/a 3/29/2005 CytRx Corporation494
arimoclomol citrate n/a 1/13/2015 Orphazyme ApS495
arsenic Trisenox 11/2/2001496
arsenic trioxide Trisenox 10/18/2001
Treatment of the on-off fluctuations associated with late-stage Parkinson's disease.
Pentech Pharmaceuticals, Inc.
For the treatment of patients in a vegetative state or minimally conscious state for up to 12 months following a severe traumatic brain injury (traumatic or spontaneous)
NeuroHealing Pharmaceuticals, Inc.
AQ-13 (4-aminoquinoline analog)
Immtech Pharmaceuticals, Inc.
Treatment of malignant pleural mesothelioma
Treatment of beta-hemoglobinopathies and beta-thalassemia.
Perrine, Susan P., M.D.
Treatment of amyotrophic lateral sclerosis
Treatment of Neimann-Pick disease, type C
Treatment of acute myelocytic leukemia subtypes M0, M1, M2, M4, M5, M6 and M7
Teva Branded Pharmaceutical Products R&D, Inc.
Treatment of chronic myeloid leukemia
Teva Branded Pharmaceutical Products R*D, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 69 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
497
arsenic trioxide Trisenox 6/13/2003 Treatment of liver cancer498
Arsenic trioxide Trisenox 3/4/2005 Treatment of malignant glioma499
arsenic trioxide Trisenox 7/17/2000500
Arsenic trioxide Trisenox 4/28/2000 Treatment of multiple myeloma.501
Arsenic trioxide Trisenox 5/13/2003502
arsenic trioxide capsule (oral) n/a 11/2/2015 Orsenix Holdings BV503
Artesunate n/a 7/19/1999 Treatment of malaria.504
Artesunate n/a 3/28/2006 Immediate treatment of malaria505
arylsulfatase A (rhASA) n/a 2/27/2008506
Ascorbic acid Ascor L 500 8/31/2007 Treatment of scurvy
Teva Branded Pharmaceutical Products R&D, Inc.
Teva Branded Pharmaceutical Products R&D, Inc.
Treatment of myelodysplastic syndrome.
Teva Branded Pharmaceutical Products R&D, Inc.
TEVA Branded Pharmaceutical Products R & D, Inc.
Treatment of chronic lymphocytic leukemia
Teva Branded Pharmaceutical Products R&D, Inc.
Treatment of acute promyelocytic leukemia.
World Health Organization
US Army Medical Materiel Development Activity
Treatment of metachromatic leukodystrophy
Shire Human Genetic Therapies, Inc.
McGuff Pharmaceuticals Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 70 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
507
astuprotimut-R n/a 5/29/2009508
ataluren n/a 1/10/2005 PTC Therapeutics, Inc.509
ataluren n/a 3/10/2008 PTC Therapeutics, Inc.510
ataluren Translarna 12/10/2014 PTC Therapeutics, Inc.511
ataluren n/a 9/1/2004 PTC Therapeutics, Inc.512
ataluren n/a 9/1/2015 Treatment of aniridia PTC Therapeutics, Inc.513
Atezolizumab n/a 10/13/2016 Genentech, Inc.514
Atrasentan n/a 9/29/2016 AbbVie Inc.515
n/a 4/27/2007 TVAX Biomedical, LLC
Treatment of MAGE-A3 positive non-small cell lung cancer
GlaxoSmithKline Biologicals, S.A.
Treatment of Muscular Dystrophy resulting from premature stop mutations in the dystrohin gene
Treatment of spinal muscular atrophy
Treatment of mucopolysaccharidosis type I
For use in the treatment of cystic fibrosis resulting fron a nonsense (premature stopcodon) mutation in the cystic bibrosis transmembrane conductance regulatory gene
Treatment of small cell lung cancer (SCLC)
Treatment of pediatric nephrotic syndrome
Attenuated autologous cancer cells and granulocyte macrophage colony stimulating factor in combination with activated autologous blood derived T-cells
Treatment of primary central nervous system malignancies
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 71 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
516
auranofin Ridaura 4/30/2010 Treatment of amebiasis517
n/a 10/22/2014 Mayo Clinic518
n/a 5/4/2016519
n/a 10/21/2014520
n/a 11/13/2014521
n/a 1/15/2015
Ctr for Discovery & Innovation in Parasitic Diseas
autologous adipose derived mesenchymal stromal cells
Treatment of amyotrophic lateral sclerosis
autologous adipose tissue derived mesenchymal stem cells
Treatment of Buerger's disease (thromboangiitis obliterans).
Biostar Stem Cell Research Institute
autologous bone marrow CD34+ cells transduced ex vivo with a self activating HIV-1 -based lentiviral vector, EFS-ADA
Treatment of adenosine deaminase deficient severe combined immunodeficiency
Donald B. Kohn, MD - Professor, UC-UCLA
Autologous CD3+ T cells transduced with retroviral vector containing a chimeric antigen receptor directed against CD19 (Autologous CD3+ T cells containing CD19 chimeric antigen receptor)
Treatment of acute lymphoblastic leukemia (ALL)
Juno Therapeutics, Inc.
autologous CD34+ bone marrow derived stem cells transduced with a self-inactivating gammaretroviral vector encoding the human IL2RG (yc)
Treatment of severe combined immune deficiency-X1
Boston Children's Hospital
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 72 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
522
n/a 4/30/2010523
n/a 4/19/2012 bluebird bio, Inc.524
n/a 2/26/2014 bluebird bio Inc.525
n/a 3/18/2013 bluebird bio, Inc.526
n/a 10/4/2016
Autologous CD34+ cells transfected with lentiviral vector containing the human WAS cDNA (Telethon 003)
Treatment of Wiskott Aldrich syndrome
GlaxoSmithKline Intellectual Property Development
autologous CD34+ hematopoietic stem cells tranduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA
Treatment of andrenoleukodystrophy
autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human BA-T87Q-globin gene
For the treatment of Sickle Cell Disease
autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human BA-T87Q-globin gene
Treatment of B-thalassemia major and intermedia
autologous CD4+/CD8+ T cells expressing a CD19 chimeric antigen receptor (CAR) and a truncated epidermal growth factor receptor.
Treatment of chronic lymphocytic leukemia
Juno Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 73 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
527
n/a 9/7/2016528
n/a 4/27/2016529
n/a 11/6/2014530
n/a 3/28/2016 Adaptimmune, LLC531
n/a 11/19/2013 iREG Medical AB532
n/a 6/1/2007533
n/a 9/27/2016
autologous CD4+/CD8+ T cells expressing a CD19 chimeric antigen receptor and a truncated epidermal growth factor receptor
Treatment of acute lymphoblastic leukemia
Juno Therapeutics, Inc.
autologous CD4+/CD8+ T cells expressing a CD19 chimeric antigen receptor and truncated epidermal growth factor receptor
Treatment of diffuse large B-cell lymphoma
Juno Therapeutics, Inc.
autologous CD4+/CD8+ T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Treatment of acute lymphoblastic leukemia (inclusive of B-cell acute lymphoblastic leukemia)
Juno Therapeutics, Inc.
Autologous CD4+/CD8+ T cells transduced with lentiviral vector directed against the NY-ESO-1 protein
Treatment of soft tissue sarcoma.
autologous CD4+CD25hiFoxP3+regulatory T cells
Prevention of graft rejection following solid organ transplantation
Autologous cultured endothelial cells on a donor human corneal disk
Treatment of Fuch's dystrophy, pseudophakic bullous keratopathy, and bullous keratopathy
Cellular Bioengineering, Inc.
Autologous dendritic cell/tumor antigens
Treatment of glioblastoma multiforme (GBM)
China Medical University Hospital
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 74 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
534
n/a 5/6/2014 Amphera BV535
Dcvax-Brain 11/29/2002536
Cvac(Tm) 9/13/2010 Treatment of ovarian cancer. Prima Biomed Ltd.537
M-Vax 2/23/1999538
Permaderm 6/1/2012539
n/a 3/9/2015 Cell Medica, Inc.
autologous dendritic cells pulsed with allogeneic tumor cell lysate
Treatment of malignant mesothelioma
Autologous dendritic cells pulsed with autologous antigens from primay malignant brain tumor cells
Treatment of primary brain malignant cancer
Northwest Biotherapeutics, Inc.
Autologous dendritic cells pulsed with recombinant human fusion protein (mucin1- glutathione S transferase) coupled to oxidized polymannose
Autologous DNP-conjugated tumor vaccine
For adjuvant therapy in melanoma patients with surgically resectable lymph node metastasis (Stage III and limited Stage IV disease).
Avax Technologies, Inc.
Autologous Engineered Skin Substitute
Treatment of hospitalized patients with deep partial and full thickness burns requiring grafting.
Amarantus BioScience Holdings, Inc.
autologous Epstein-Barr virus specific T-cells
Treatment of Epstein-Barr virus positive non-Hodgkin lymphomas.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 75 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
540
Alecsat 9/13/2013 CytoVac A/S541
n/a 5/10/2016 Caladrius Biosciences542
n/a 6/10/2014543
n/a 9/3/2004544
4/3/2008545
Biovaxid 10/18/2011546
n/a 6/7/2011 Tengion, Inc.
autologous ex vivo expanded CD4+-enriched leukocytes treated with the de-methylating agent 5-aza-2â⿬⿢-deoxycytidine
Treatment of glioblastoma multiforme
autologous ex vivo expanded polyclonal CD4+CD25+CD1271o/-FOXP3+ Regulatory T cells
Treatment of type 1 diabetes mellitus with residual beta cell function
autologous genetically modified human dermal fibroblasts
Treatment of dystrophic epidermolysis bullosa.
Fibrocell Technologies, Inc.
Autologous incubated macrophage
Therapy to improve the motor and sensory neurological outcome in acute cases of spinal cord injury
Proneuron Biotechnologies, Inc.
autologous lymphocytes depleted ex vivo of immunoreactive T cells using 4,5
Theralux Photodynamic Therapy
Treatment of chronic graft versus host disease
Kiadis Pharma Canada, Inc.
Autologous lymphoma-derived immunoglobulin idiotype antigen vaccine conjugated to keyhole limpet hemocyanin
Treatment of Waldenstrom's macroglobulinemia
Biovest International, Inc.
autologous neo-uninary conduit
Treatment of bladder dysfunction requiring incontinent urinary diversion.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 76 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
547
Rhinocytes 2/1/2008 RhinoCyte, Inc.548
Rhinocytes 12/31/2008 RhinoCyte, Inc.549
n/a 7/14/2005 TissueTech, Inc.550
n/a 6/30/2016 Novadip Biosciences551
n/a 3/27/2014 Kite Pharma, Inc.552
Eccs-50 11/9/2016553
n/a 3/14/2014
Autologous olfactory neural progenitors
Treatment of spinal cord injury patients with ASIA Impairment grades A, B, or C
autologous olfactory neural progenitors
Treatment of amyotrophic lateral sclerosis
Autologous or allogeneic limbal epithelial stem cells expanded ex vivo on human amniotic membrane
Treatment of ocular surface diseases that are characterized by total limbal stem cell deficiency
autologous osteogenic cells cultured from adipose tissue derived stem cells embedded in extracellular matrix with demineralized bone matrix.
Treatment of congenital pseudarthrosis of the tibia
autologous peripheral blood T lymphocytes transduced with retroviral vector containing anti CD19 CD28/CD3 zeta chimeric antigen receptor
Treatment of diffuse large B-cell lymphoma.
Autologous stromal vascular cell fraction from adipose tissue (central facitily processed)
Treatment of impaired hand function due to systemic sclerosis
Cytori Therapuetics, Inc.
autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Treatment of chronic lymphocytic leukemia
Novartis Pharmaceuticals Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 77 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
554
n/a 1/31/2014555
n/a 4/20/2016 Kite Pharma, Inc.556
n/a 4/20/2016 Kite Pharma, Inc.557
n/a 4/28/2016 Kite Pharma, Inc.558
n/a 4/25/2016 Kite Pharma, Inc.559
n/a 4/20/2016 Kite Pharma, Inc.
Autologous T Cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
For the treatment of Acute Lymphoblastic Leukemia
Novartis Pharmaceuticals Corporation
autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor
Treatment of primary mediastinal B-cell lymphoma.
Autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor
Treatment of acute lymphoblastic leukemia. (ALL).
autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor
Treatment of mantle cell lymphoma.
autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor
Treatment of follicular lymphoma.
autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor
Treatment of chronic lymphocytic leukemia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 78 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
560
n/a 5/11/2016 Treatment of multiple myeloma bluebird bio, Inc.561
n/a 5/6/2015 Treatment of glioblastoma562
Oncophage 5/10/2002 Agenus, Inc.563
Oncophage 7/11/2002 Agenus, Inc.564
n/a 7/24/2012 Duke University565
n/a 8/26/2009 Glaxo Group Limited566
Autolymphocyte therapy n/a 7/12/1994 Cytogen Corporation567
avicin d n/a 3/24/2015 Treatment of multiple myeloma
autologous T-lymphocytes transduced with anti-BCMA02 CAR lentiviral vector encoding a chimeric antigen receptor (CAR) specific for the human B cell maturation antigen
autologous tumor cells fused with polyethylene glycol to human allogeneic FO-1, DNA plasmid transfected tumor cell line
Sebastiano Gattoni-Celli, MD
autologous tumor-derived gp96 heat shock protein-peptide complex
Treatment of renal cell carcinoma
autologous tumor-derived gp96 heat shock protein-peptide complex
Treatment of metastatic melanoma
autologous umbilical cord blood
Treatment of pediatric (0-16 yrs old inclusive) cerebral palsy due to acquired brain injury
autologus CD34+ cells transfected with retroviral vector containing adenosine deaminase gene
Treatment of severe combined immunodeficiency due to adenosine deaminase deficiency.
Treatment of renal cell carcinoma.
Avicin Therapeutics Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 79 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
568aviptadil n/a 2/22/2005
569Azacitidine Vidaza 6/18/2008 Celgene Corporation
570
Azathioprine Imuran 9/14/1999 Oral Solutions, Inc.571
Azurin-p28 n/a 12/2/2015 Treatment of glioma572
n/a 8/9/2006 OncoVac Corporation573
n/a 2/29/2016574
Bacitracin Altracin 3/13/1984 A. L. Laboratories, Inc.575
Baclofen Lioresal Intrathecal 9/26/1994 Medtronic, Inc.576
baclofen n/a 12/2/2003 Treatment of dystonia577
baclofen n/a 12/16/1991578
baclofen Gablofen 10/28/2015 Mallinckrodt, Inc.
Treatment of pulmonary arterial hypertension
Mondobiotech Laboratories AG
Treatment of acute myeloid leukemia
Treatment of oral manifestations of graft-versus-host disease.
CDG Therapeutics, Inc.
Bacillus Calmette-Guerin vaccine
Treatment of stage IIb through IV metastatic melanoma
Bacillus subtilis oxalate decarboxylase
Treatment of pediatric hyperoxaluria
Allena Pharmaceuticals
Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.
Treatment of spasticity associated with cerebral palsy.
Medtronic Neurological
Treatment of intractable spasticity due to multiple sclerosis or spinal cord injury.
Metacel Pharmaceuticals, LLC
Treatment of complex regional pain syndrome.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 80 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
579
n/a 9/26/2013580
bardoxolone n/a 3/30/2015581
Bardoxolone n/a 8/6/2008 Treatment of pancreatic cancer582
basimglurant n/a 3/9/2012 Treatment of Fragile X Syndrome583
beclomethasone n/a 3/4/2009584
n/a 11/19/2012 Soligenix, Inc585
Bec 8/28/2001 Soligenix, Inc.586
Orbec(R) 7/24/2009 Soligenix, Inc.587
n/a 12/18/2007 Soligenix, Inc.
bacterium Bacteroides thetaiotaomicron
Treatment of active Crohn's disease in the pediatric population
4D Pharma Research Ltd
Treatment of pulmonary arterial hypertension
Reata Pharmaceuticals, Inc.
Reata Pharmaceuticals, Inc.
Hoffmann-La Roche, Inc.
Treatment of pediatriac patients with ulcerative colitis
Aptalis Pharma US, Inc.
beclomethasone 17,21-dipropionate
Prevention of death following a potentially lethal dose of total body irradiation during or after a radiation disaster
beclomethasone 17,21-dipropionate
Prevention of gastrointestinal graft-versus-host disease
beclomethasone 17,21-dipropionate
Treatment of gastrointestinal symptoms with chronic graft versed host disease in patients undergoing allogenic hematopoietic cell transplantation
beclomethasone 17,21-diproprionate
Treatment of pediatric patients with Crohn disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 81 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
588
Beclomethasone dipropionate n/a 3/27/1998 Soligenix, Inc.589
beloranib n/a 1/15/2013 Zafgen, Inc.590
n/a 9/10/2013 Supratek Pharma, Inc.591
n/a 11/5/2015592
benznidazole n/a 4/14/2014 Treatment of Chagas disease Chemo Research, S.L.593
benzoate Naben 7/6/2012594
Total Block Vl Spf 75 8/13/2001595
Benzydamine hydrochloride Tantum 5/18/1998
For oral administration in the treatment of intestinal graft-versus-host disease.
Treatment of Prader-Willi syndrome
bendamustine hydrochloride with betadex sulfobutyl ether sodium
Treatment of chronic lymphocytic leukemia
Bendamustine oral doseage formulation
Treatment of chronic lymphocytic leukemia
Exinda Theapeutics LLC
Treatment of pediatric schizophrenia
SyneuRx International (Taiwan) Corp.
Benzophenone-3, octylmethoxycinnamate, avobenzone, titanium dioxide, zinc oxide
For the prevention of visible light induced skin photosensitivity as a result of porfimer sodium photodynamic therapy
Fallien Cosmeceuticals Ltd.
Prophylactic treatment of oral mucositis resulting from radiation therapy for head and neck cancer.
Angelini Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 82 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
596
Pre-Pen/Mdm 9/28/1987 AllerQuest LLC597
Beractant 12/20/1993 Ross Laboratories598
Beraprost n/a 4/29/1999 LungRx, Inc.599
beraprost sodium 314d n/a 12/22/2011 Lung Rx, Inc.600
Beta alethine Betathine 3/24/1997 Treatment of multiple myeloma.601
Beta alethine Betathine 3/24/1997602
Betahistine dihydrochloride n/a 11/8/2007
Benzylpenicillin, benzylpenicilloic, benzylpenilloic acid
Assessing the risk of administrating penicillin when it is the preferred drug of choice in adult patients who have previously received penicillin and have a history of clinical sensitivity.
Survanta Intratracheal Suspension
Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.
Treatment of pulmonary arterial hypertension associated with any New York Heart Association classification (Class I, II, III, or IV).
Treatment of pulmonary arterial hypertension
Dovetail Technologies, Inc.
Treatment of metastatic melanoma.
Dovetail Technologies, Inc.
Treatment of obesity associated with Prader Willi syndrome
Floyd R. Sallee, M.D., Ph.D.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 83 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
603betamethasone n/a 10/7/2015
604Betulinic acid n/a 8/9/2007
605bevacizumab n/a 10/21/2010
606bevacizumab Avastin 10/20/2004 Treatment of pancreatic cancer Genentech, Inc.
607
bevacizumab Avastin 11/20/2009 Genentech, Inc.608
bevacizumab Avastin 10/13/2009 Genentech, Inc.609 bevacizumab n/a 3/10/2016 Treatment of Coat's disease MicroSert, Ltd.610 bevacizumab Avastin 1/27/2016 Treatment of mesothelioma Genentech, Inc.611
bezafibrate Bezalip 7/24/2013612 BF2.649 (Pitolisant) n/a 5/17/2010 Treatment of narcolepsy Bioprojet Pharma613
Fang 10/22/2014 Treatment of Ewing's sarcoma Gradalis, Inc.614
Fang 2/17/2012 Gradalis, Inc.
Treatment of Ataxia Telangiectasia.
Grace Therapeutics, LLC
Topical treatment of metastatic melanoma
Advanced Life Sciences, Inc. (ALS)
Treatment of hereditary hemorrhagic telangiectasia
Terence M. Davidson, MD
In combination with a platinum and 5-FU or capecitabine for the treatment of stomach cancer.
Treatment of melanoma stages IIb through IV as part of a combination chemotherapy regimen
For therapeutic treatment of Barth syndrome
Barth Sydrome Foundation, Inc.
bi-shRNA furin and GMCSF Autologous Tumor Cell Vaccine
bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine
Treatment of stage IIB to IV melanoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 84 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
615
Fang 4/18/2011 Treatment of ovarian cancer Gradalis, Inc.616
bifidobacterium infantis 35624 n/a 3/24/2008617
n/a 1/16/2003618
Primeskin 9/6/2016619
Bindarit n/a 2/3/1998 Treatment of lupus nephritis.620
binimetinib n/a 11/19/2013 Array BioPharma, Inc.621
binimetinib n/a 7/31/2014 Treatment of ovarian cancer Array Biopharma, Inc.622
n/a 4/27/2006 TissueTech, Inc.
bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine
Treatment of pediatric ulcerative colitis
Alimentary Health Limited
bifidobacterium longum infantis 35624
Treatment of pediatric Crohn's disease
Alimentary Health Limited
bilayer, engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts
Treatment of partial deep dermal and full thickness burn wounds
University Children's Hospital Zurich
Angelini Pharmaceuticals, Inc.
Treatment Stage IIB-IV melanoma.
Bio-engineered oral mucosal tissue
For use as a graft for restoring a cornea-like epithelial phenotype to substitute for the normal corneal epithelium that is lost in patients due to total limbal stem cell deficiency
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 85 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
623
n/a 2/11/2002 Excorp Medical, Inc.624
n/a 3/2/2000 Treatment of sickle cell disease. ICAgen Inc.625
bisantrene HCl n/a 2/14/2014 Update Pharma, Inc.626
n/a 8/8/2013627
n/a 8/8/2013628
n/a 9/14/2011629
n/a 2/10/2015630
bivalirudin Angiomax 11/2/2005
Bioartificial liver system utilizing xenogenic hepatocytes in a hollow fiber bioreactor cartridge (BAL)
Treatment of patients with acute liver failure presenting with encephalopathy deteriorating beyond Parson's grade 2
Bis(4-fluorophenyl)phenylacetamide
Treatment of acute myeloid leukemia
bispecific antibody (monoclonal antibody)
Treatment of HER2-expressing advanced adenocarcinoma of the stomach and gastroesophageal junction
Merrimack Pharmaceuticals, inc.
bispecific antibody (monoclonmal antibody)
Treatment of HER2-expressing adenocarcinoma of the esophagus
Merrimack Pharmaceuticals, Inc.
bispecific antibody targeting interleukin 4 (IL-4) and interleukin 13 (IL-13)
Treatment of idiopathic pulmonary fibrosis.
Sanofi-Aventis US, Inc.
bivalent anti-human myostatin adnectin-IgG1
Treatment of duchenne muscular dystrophy
Bristol-Myers Squibb Co.
For use as an anticoagulant in patients with or at risk of heparin-induced thrombocytopenia/heparin-induced thrombocytopenia thrombosis syndrome
The Medicines Company
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 86 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
631Bleomycin n/a 12/20/2010 Treatment of pancreatic cancer. CIRJ Company Ltd.
632Bleomycin Blenoxane 2/9/1999 Treatment of pancreatic cancer. Genetronics, Inc.
633blinatumomab n/a 5/16/2008 Amgen, Inc.
634blinatumomab n/a 5/16/2008 Amgen, Inc.
635
blinatumomab n/a 2/6/2006 Amgen, Inc.636
Blinatumomab n/a 5/16/2008 Amgen, Inc.637
n/a 5/17/2010 t2cure GmbH638
bortezomib n/a 7/5/2016 BioXcel Corporation639
bortezomib Velcade 2/3/2015640
bortezomib Velcade 1/4/2011641
Botulinum toxin type A Botox 12/6/1991 Allergan, Inc.
Treatment for hairy cell leukemia.
Treatment of prolymphocytic leukemia
Treatment of indolent B-cell lymphoma, excluding CLL and NHL with CNS involvement
Treatment of chronic lymphocytic leukemia
Bone marrow-derived mononuclear cells
Treatment of thromboangiitis obliterans (Buerger's disease)
Treatment of neurofibromatosis type 2 (NF2)
Treatment of acute lymphoblastic leukemia
Millennium Pharmaceuticals, Inc.
Treatment of follicular non-Hodgkin lymphoma
Millennium Pharmaceuticals, Inc.
Treatment of dynamic muscle contracture in pediatric cerebral palsy patients.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 87 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
642Botulinum toxin type A Dysport 3/23/1989
643Botulinum toxin type A Dysport 8/12/1998
644
Botulinum toxin type A n/a 9/15/1992645
Botulinum toxin type F n/a 10/24/1991 Ipsen Limited646
Bovine colostrum n/a 11/19/1990647
Sporidin-G 3/1/1994 GalaGen, Inc.648
bovine lactoferrin n/a 2/23/2015649
bovine lactoferrin n/a 2/19/2015
Treatment of essential blepharospasm.
Porton International, Inc.
Treatment of spasmodic torticollis (cervical dystonia).
Ipsen Biopharm Limited
Treatment of synkinetic closure of the eyelid associated with VII cranial nerve aberrant regeneration.
Botulinum Toxin Research Associates, Inc.
Treatment of spasmodic torticollis (cervical dystonia).
Treatment of AIDS-related diarrhea.
Hastings, Donald DVM
Bovine immunoglobulin concentrate, Cryptosporidium parvum
Treatment and symptomatic relief of Cryptosporidium parvum infection of the gastrointestinal tract in immunocompromised patients.
Prevention of necrotizing enterocolitis in very low birth weight infants (birth weight less than or equal to 1500 grams)
Metrodora Therapeutics, LLC
Prevention of late-onset sepsis in very low birth weight infants
Metrodora Therapeutics, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 88 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
650
Immuno-C 9/30/1993651
Branched chain amino acids n/a 12/23/1988652
Brentuximab vedotin Adcetris 9/7/2016 Seattle Genetics, Inc.653
brentuximab vedotin Adcetris 9/13/2013 Seattle Genetics, Inc.654
brentuximab vedotin Adcetris(R) 1/31/2014 Seattle Genetics, Inc.655
brentuximab vedotin Adcetris 4/15/2013 Seattle Genetics, Inc.656
brentuximab vedotin Adcetris 7/7/2016 Seattle Genetics, Inc.657
brentuximab vedotin Adcetris 11/19/2012 Treatment of mycosis fungoides Seattle Genetics, Inc.658
brentuximab vedotin n/a 5/23/2016 Seattle Genetics, Inc.
Bovine whey protein concentrate
Treatment of cryptosporidiosis caused by the presence of Cryptosporidium parvum in the gastrointestinal tract of patients who are immunodeficient/immunocompromised or immunocompetent.
Biomune Systems, Inc.
Treatment of amyotrophic lateral sclerosis.
Mount Sinai Medical Center
Treatment of primary cutaneous CD30-positive T-cell lymphoproliferative disorders
Treatment of patients with angioimmunoblastic T-cell lymphoma
Treatment of patients with diffuse large B-cell lymphoma.
Treatment of patients with peripheral T-cell lymphoma, not otherwise specified
Treatment of enteropathy-associated T-cell lymphoma.
Treatment of adult T-cell leukemia/lymphoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 89 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
659
brigatinib n/a 4/28/2016660
brilliant blue G Brilliant Peel 12/16/2009661
Brilliant Blue G Dorc Ilm-Blue 7/31/2012662
Brimonidine Alphagan 2/7/2000 Allergan, Inc.663
Brivaracetam n/a 10/5/2005 UCB Pharma, Inc.664
Broxuridine Broxine/Neomark 9/18/1995 NeoPharm, Inc.665
n/a 5/13/2015 Acerta Pharma BV666
bryostatin 1 n/a 3/31/2015
Treatment of anaplastic lymphoma kinase-positive (ALK+), c-ros 1 oncogene positive (ROS1+), or epidermal growth factor receptor positive (EGFR+) non-small cell lung cancer (NSCLC).
ARIAD Pharmaceuticals, Inc.
For use in ophthalmic surgery to aid in the removal of the inner limiting membrane of the eye.
Fluron GmbH - Subsidiary of Geuder AG
To selectively stain the thickened internal limiting membrane, which has formed onto the inner side of the retina in vitreo-retinal disorders
Dutch Opthalmic Research Center
Treatment of anterior ischemic optic neuropathy.
Treatment of symtomatic myoclonus
Radiation sensitizer in the treatment of primary brain tumors.
Bruton's Tyrosine Kinase (Btk) Inhibitor
Treatment of chronic lymphocytic leukemia (CLL).
Treatment of Fragile X Syndrome.
Neurotrope BioScience, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 90 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
667
bryostatin-1 n/a 12/3/2001 GPC Biotech, Inc.668
Bucillamine n/a 10/22/2015 Treatment of cystinuria669
budesonide Nefecon 5/17/2010 Pharmalink AB670
budesonide Uceris 5/6/2013 Santarus, Inc.671
Buffered Ursodeoxycholic Acid Ursocarb 9/3/2004 Digestive Care, Inc.672
bupivacaine Transdur 6/18/2008673
bupivacaine hydrochloride n/a 5/31/2016674
Busulfan Spartaject 4/21/1994675
Busulfan Spartaject 7/7/1997 SuperGen, Inc.676
busulfan Partaject 11/25/2002 SuperGen, Inc.
For use in combination with paclitaxel in the treatment of esophageal cancer
Revive Therapeutics, Ltd.
To slow the progression of immunoglobulin A nephropathy & delay kidney failure in patients affected by the disease.
Treat of ulcerative colitis in pediatric patients aged 0 through 16 years.
Treatment of pruritus in patients with Alagille Syndrome
Relief of persistent pain associated with postherpetic neuralgia
Impax Laboratories, Inc.
treatment of pain associated with postherpetic neuralgia.
Grace Therapeutics, LLC
For use as preparative therapy for malignancies treated with bone marrow transplantation.
Sparta Pharmaceuticals, Inc.
Treatment of primary brain malignancies.
Preparative therapy for pediatric patients undergoing bone marrow transplantation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 91 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
677
Buthionine sulfoxamine n/a 10/5/2005678
Buthionine sulfoxamine n/a 10/5/2005679
butylidenephthalide n/a 8/5/2015 Treatment of Malignant Glioma680
Butyrylcholinesterase n/a 9/30/1992681
Butyrylcholinesterase n/a 3/25/1992682
C1 esterase inhibitor (human) n/a 8/21/1996683
C1 esterase inhibitor (Human) Cinryze 11/30/2016684
C20-D3-retinyl acetate n/a 9/16/2010 Treatment of Stargardt's disease685
C21H27O10P Minnelide 3/30/2015 Treatment of gastric cancer.
Use as a modulator of chemotherapy for the treatment of pediatric patients with primary malignant brain tumors
USC-CHLA Institute for Pediatric Clinical Research
For use in children as a modulator of chemotherapy for the treatment of pediatric patients with neuroblastoma
USC-CHLA Institute for Pediatric Clinical Research
Everfront Biotech, Inc.
Treatment of post-surgical apnea.
Shire Laboratories Inc.
For the reduction and clearance of toxic blood levels of cocaine encountered during a drug overdose.
Shire Laboratories Inc.
Treatment and prevention of angioedema caused by C1-esterase inhibitor deficiency.
Alpha Therapeutic Corporation
Treatment of acute antibody mediated rejection following kidney transplantation
Shire ViroPharma, Inc.
Alkeus pharmaceuticals, Inc.
Minneamrita Therapeutics, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 92 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
686C24H28N8O7S2 n/a 4/2/2015 Curis, Inc.
687C66H100N6O27 n/a 3/11/2013 GenSpera, Inc.
688
caffeine and sodium benzoate n/a 8/22/2012689
Calcium acetate n/a 6/27/1989 Pharmedic Company690
n/a 12/20/2010691
Calcium carbonate 6/6/1990692
Calcium gluconate Calgonate 11/20/1997 Calgonate Corp.693
Calcium gluconate gel H-F Gel 5/21/1991694
Calcium gluconate gel 2.5% n/a 9/10/1990695
calfactant Infasurf 9/5/2000 ONY, Inc.
Treatment of diffuse large B-cell lymphoma (DLBCL).
Treatment of of hepatocellular carcinoma
Treatment of seizure prolongation in patients undergoing electroconvulsive therapy
Luitpold Pharmaceuticals, Inc.
Treatment of hyperphosphatemia in end stage renal disease.
calcium benzoate and risperidone
Treatment of pediatric patients with schizophrenia
University of California, Los Angeles
R & D Calcium Carbonate/600
Treatment of hyperphosphatemia in patients with end stage renal disease.
R & D Laboratories, Inc.
For use as a wash for hydrofluoric acid spills on human skin.
For use in the emergency topical treatment of hydrogen fluoride (hydrofluoric acid) burns.
Calgonate Corporation
Emergency topical treatment of hydrogen fluoride (hydrofluoric acid) burns.
Paddock Laboratories, Inc.
Treatment of acute respiratory distress syndrome (ARDS)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 93 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
696camostat n/a 5/18/2011 Treatment of chronic pancreatitis NIXS Corporation
697cannabidiol n/a 11/14/2013 Treatment of Dravet syndrome. GW Pharma Ltd.
698cannabidiol n/a 11/29/2016 Talent biotechs Ltd.
699
cannabidiol n/a 4/22/2015 GW Pharma Ltd.700
cannabidiol n/a 4/19/2016 GW Pharma Ltd.701
cannabidiol n/a 7/1/2014 Treatment of Dravet syndrome702
cannabidiol n/a 8/20/2014703
cannabidiol n/a 6/13/2016 Treatment of infantile spasms GW Research Ltd.704
cannabidiol n/a 7/23/2015 Treatment of infantile spasms705
cannabidiol n/a 11/17/2014706
cannabidiol n/a 2/23/2016 Treatment of Fragile X syndrome707
cannabidiol n/a 9/24/2014 Treatment of glioma708
cannabidiol n/a 7/14/2015 Talent biotechs Ltd.
Treatment of Graft versus Host Disease
Treatment of neonatal hypoxic ischemic encephalopathy
Treatment of tuberous sclerosis complex.
Insys Development Company, Inc.
Treatment of glioblastoma multiforme
Insys Development Company, Inc.
Insys Development Company, Inc.
Treatment of pediatric schizophrenia (pediatrics is defined as 0 through 16 years of age)
Insys Development Company, Inc.
Zynerba Pharmaceuticals, Inc.
Insys Development Company, Inc.
Prevention of Graft versus Host Disease (GVHD).
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 94 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
709cannabidiol n/a 6/23/2014
710cannabidiol; n/a 2/27/2014 GW Pharma Ltd.
711 cannabidivarin n/a 11/30/2016 Treatment of Rett syndrome GW Research Ltd.712
cantharidin n/a 7/24/2012 Orenova Group, LLC713 cantrixil n/a 4/20/2015 Treatment of ovarian cancer CanTx, Inc.714
Caplacizumab n/a 4/14/2009 Ablynx NV715
n/a 10/28/2009716
n/a 2/14/2014 Treatment of systemic sclerosis717
capsaicin n/a 10/23/2002 Treatment of erythromelalgia718
Capsaicin n/a 9/29/2006719
Capsaicin n/a 8/3/2005720
capsaicin Qutenza 5/2/2003
Treatment of Lennox-Gastaut syndrome
Insys Development Company, Inc.
Treatment of Lennox-Gastaut syndrome
Treatment of perforating diseases, including Kyrle's disease, reactive perforating collangenosis, perforating folliculitis and elastosis perforans serpiginosa
Treatment of thrombotic thrombocytopenic purpura
caprine hyperimmune serum against HIV lysate
Treatment of amyotrophic lateral sclerosis
Daval International Limited
caprine hyperimmune serum against HIV lysate
Daval International Limited
Acorda Therapeutics, Inc.
Treatment of intermetatarsal neuroma (Morton's Neuroma) that does not respond to conservative treatment and requires either neurectomy or neurolysis
Centrexion Corporation
Treatment of postherpetic neuralgia.
TheraQuest Biosciences, LLC
Treatment of painful HIV-associated neuropathy
Acorda Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 95 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
721
carbenoxolone n/a 7/2/2014722
carbetocin (nasal spray) n/a 4/11/2014723
carbon dioxide n/a 12/17/2015 Capnia, Inc.724
carbon monoxide n/a 8/16/2016 Proterris, Inc.725
carbon monoxide n/a 9/28/2012 Treatment of sickle cell disease726
n/a 2/10/2015 Treatment of AL amyloidosis727
cardiotrophin-1 n/a 6/24/2008 Digna Biotech, S.L.728
cardiotrophin-1 n/a 10/16/2014 Digna Biotech, S.L.729
cardiotrophin-1 n/a 9/13/2011 Treatment of acute liver failure Digna Biotech S.L.730
carglumic acid Carbaglu 6/17/2014 Treatment of organic acidemias Orphan Europe SARL731
carisbamate n/a 3/16/2012 SK Life Science, Inc.
Treatment of Huntington's Disease
Oxalys Pharmaceuticals, Inc.
Treatment of Prader Willi syndrome
Ferring Pharmaceuticals, Inc.
Treatment of trigeminal neuralgia
Treatment of idiopathic pulmonary fibrosis (IPF).
Hillhurst Biopharmaceuticals, Inc.
carboxy pyrrolidine hexanoyl pyrrolidine carboxylate
Glaxo Group Limited, England d/b/a GlaxoSmithKline
To protect the liver from ischemia-reperfusion injury inherent to the procedure of transplantation.
Prevention of ischemia-reperfusion injury in kidney transplant recipients
Management of patients with infantile spasms
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 96 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
732carlumab n/a 10/24/2006 Treatment of pancreatic cancer Centocor, Inc.
733Carmustine n/a 7/3/2000
734
Cascara sagrada fluid extract n/a 3/21/1989 Intramed Corporation735
n/a 2/18/2016736
catumaxomab n/a 7/1/2009 Treatment of gastric cancer737
Catumaxomab Removab 6/9/2006 Treatment of ovarian cancer738
n/a 12/20/2013 DiaVacs, Inc.739
cediranib n/a 12/13/2010 Treatment of glioblastoma740
Ceftriaxone sodium Rocephin 3/28/2006741
celiprolol n/a 1/5/2015742
Apocell 3/18/2013
Treatment of intracranial malignancies.
Direct Therapeutics, Inc.
Treatment of oral drug overdosage to speed lower bowel evacuation.
CaspaCIDe System (combination product) consisting of allogeneic T Cell genetically modified to contain an inducible caspase 9 gene and Rimiducid
Replacement T-cell therapy for the treatment of immunodeficiency and graft versus host disease after allogeneic hematopoietic stem cell transplant
Bellicum Pharmaceuticals, Inc.
Neoviibiotech North America, Inc.
Neoviibiotech North America, Inc.
CD40/CD80/CD86 modified autologous dendritic cell therapy
Treatment Type 1 diabetes mellitus patients with residual beta cell function
National Institutes of Health
Treatment of amyotrophic lateral sclerosis
Mass General Hospital
Treatment of Ehlers-Danlos syndrome
Acer Therapeutics, Inc.
cell based therapeutic composed of allogeneic donor apoptotic cells
Prevention of graft versus host disease
Enlivex Therapeutics Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 97 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
743
n/a 7/10/2002744
cellspan esophageal implant n/a 11/29/2016 Biostage, Inc.745
cenersen Aezea 6/18/2008 Eleos, Inc.746
cenersen Aezea 2/27/2008 Eleos, Inc.747
cenersen n/a 5/8/2006 Eleos, Inc.748
ceramide n/a 8/8/2016 Treatment of liver cancer Keystone Nano, Inc.749
cerebrolysin n/a 4/5/2016750
cerliponase alfa n/a 4/1/2013
Cells produced using the AastromReplicelle System and SC-I Therapy Kit
For use in patients receiving high dose chemotherapy who are unable to generate an acceptable dose of peripheral blood stem cells and who have a sufficient bone marrow aspirate without morphological evidence of tumor
Aastrom Biosciences Incorporated
Restore the structure and function of the esophagus subsequent to esophageal damage due to cancer, injury, or congenital abnormality
Treatment of stage IIB through IV melanoma
Treatment of chronic lymphocytic leukemia
Treatment of acute myeloid leukemia
Treatment of frontotemporal dementia, including all subvariants
EVER Neuro Pharma GmbH
Treatment of neuronal ceroid lipofuscinosis type 2
BioMarin Pharmaceutical, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 98 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
751
cethromycin Restanza(Tm) 9/9/2009752
cethromycin n/a 2/28/2007753
cethromycin Restanza(Tm) 9/9/2009754
n/a 2/10/1999 Treatment of multiple myeloma. NeoRx Corporation755
chelmab-y or chelamusab-Y Oncorad Ov103 4/24/1990 Treatment of ovarian cancer. Cytogen Corporation756
chenodeoxycholic acid n/a 2/12/2007 Baxalta US, Inc.757
Chenodeoxycholic acid Chenofalk 1/29/2004758
chenodiol Chenadal 3/22/2010 Retrophin, Inc.759
n/a 7/28/2016760
n/a 11/25/2014
Prophylactic treatment of tularemia due to Francisella tularensis.
Advanced Life Sciences, Inc.
Prophylactic treatment of patients exposed to inhalation anthrax.
Advanced LIfe Sciences, Inc.
Prophylactic treatment of plague due to Yersinia pestis
Advanced Life Sciences, Inc.
Chelating agent delivering Holmium-166
Treatment of cerebrotendinous xanthomatosis
Treatment of cerebrotendinous xanthomatosis
Dr. Falk Pharma GmbH
Treatment of cerebrotendinous xanthomatosis
chimeric antigen receptor directed against the target protein CD4 lentivirally transfected T cells
Treatment of peripheral T-cell lymphoma
iCell Gene Therapeutics
chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2
Treatment of mucopolysaccharidosis III Type B (MPS IIIB, Sanfilippo Syndrome Type B)
BioMarin Pharmaceutical, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 99 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
761
n/a 10/4/2005762
n/a 3/18/2013 Treatment of ovarian cancer763
n/a 9/26/2013 Treatment of pancreatic cancer764
n/a 11/20/2012 Treatment of gastric cancer765
n/a 10/31/2006 Treatment of pancreatic cancer Morphotek, Inc.766
n/a 8/3/2000 Biosynexus, Inc.767
Peridex 8/18/1986768
Patul-End 2/18/1997 Ear Foundation769
chloropyramine n/a 10/22/2010 Treatment of pancreatic cancer770
chloroquine n/a 5/20/2015 DualTpharma B.,V.
Chimeric monoclonal antibodies, c-alphaStx2
For treatment of shiga-toxin producing bacterial infection
Thallion Pharmaceuticals, Inc.
chimeric monoclonal antibody against Claudin 6
Ganymed Pharmaceuticals AG
chimeric monoclonal antibody against claudin-18 splice variant 2
Ganymed Pharmaceuticals AG
chimeric monoclonal antibody to claudin 18 splice variant 2
GANYMED Pharmaceuticals AG
Chimeric monoclonal antibody to mesothelin
Chimeric, humanized monoclonal antibody to staphylococcus
Prophylaxis of Staphylococcus epidermidis sepsis in low birth weight (1500 grams or less) infants.
Chlorhexidine gluconate mouthrinse
For use in the amelioration of oral mucositis associated with cytoreductive therapy used in conditioning patients for bone marrow transplantation therapy.
Procter & Gamble Company
chlorobutanol/propylene glycol/hydrochloric acid
Treatment of patulous eustachian tube
CurFAKtor Pharmaceuticals, LLC
Treatment of glioblastoma multiforme.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 100 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
771Chlorotoxin n/a 12/18/2007 Treatment of malignant glioma Morphotek, Inc.
772chlorotoxin n/a 12/2/2008 Morphotek, Inc.
773
n/a 6/23/2015 Blaze Bioscience, Inc.774
cholest-4-en-3-one, oxime n/a 2/17/2009 Genentech, Inc.775
Choline chloride Intrachol 7/20/2006776
Choline chloride Intrachol 2/10/1994777
choline tetrathiomolybdate n/a 8/25/2011 Treatment of Wilson's disease.778
n/a 12/1/1997 Curis, Inc.779
n/a 12/22/2014 JDS Therapeutics, LLC780
Ciliary neurotrophic factor n/a 1/30/1992
Treatment of stage IIb, IIc, III & IV melanoma
chlorotoxin conjugated to indocyanine green dye
Diagnostic for the management of malignant brain tumors
Treatment of spinal muscular atrophy.
Prevention and/or treatment of choline deficiency in patients on long-term parenteral nutrition
Alan L. Buchman, MD, MSPH
Treatment of choline deficiency, specifically the choline deficiency, hepatic steatosis, and cholestasis, associated with long-term parenteral nutrition.
Alan L. Buchman, MD, MSPH
Wilson Therapeutics AB
Chondrocyte-alginate gel suspension
For use in correcting vesicoureteral reflux in the pediatric population.
chromium picolinate and chromium histidinate
Treatment of pediatic polycystic ovary syndrome (0 through 16 years of age)
Treatment of amyotrophic lateral sclerosis.
Regeneron Pharmaceuticals Inc
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 101 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
781
cinacalcet Sensipar 9/7/2016 Amgen, Inc.782
Cintredekin Besudotox n/a 4/30/2010783
ciprofloxacin n/a 6/1/2011 Aradigm Corporation784
ciprofloxacin DPI 2/2/2010785
n/a 4/17/2014786
n/a 1/19/2010787 Cisplatin n/a 9/6/2016 Treatment of anal cancer Privo Technologies788
Cisplatin ChemoThin Wafer n/a 11/3/2015 Privo Technologies789
Slit Cisplatin For Inhalation 3/20/2007 Treatment of osteosarcoma
Treatment of secondary hyperparathyroidism (HPT) in pediatric patients with chronic kidney disease receiving dialysis
Treatment of idiopathic pulmonary fibrosis
Insys Development Company, Inc.
The management of bronchiectasis
Ciprofloxacin Pulmosphere(R)
Management of pulmonary infection due to Pseudomonas aeruginosa in cystic fibrosis patients
Bayer HealthCare Pharmaceuticals, Inc.
ciprofloxacin dry powder inhaler
Treatment of non-cystic fibrosis bronchiectasis
Bayer HealthCare Pharmaceuticals, Inc.
cis-adamantane-2-spiro-3'-8'-[4'-[2'-(4'-morpholinyl)ethoxy]phenyl]-1',2,'4'-trioxaspiro[4.5] decane mesylate
Treatment of malaria caused by Plasmodium falciparum, Plasmodium vivax, Plasmodium ovale, or Plasmodium malariae
Medicines for Malaria Venture (MMV)
Treatment of anatomically accessible oral cancers (lip, tongue, gum, floor of mouth, salivary gland, and other oral cavity)
Cisplatin in liposomal formulation
Eleison Pharmaceuticals LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 102 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
790
Cisplatin/epinephrine Intradose 4/3/2000791
Cisplatin/epinephrine Intradose 9/7/2000792
civamide Zucapsaicin 12/9/2002793
civamide n/a 2/17/2009794
cladribine n/a 3/19/2015795
Cladribine Mylinax 4/19/1994796
n/a 4/26/2011797
Clazosentan Erajet 2/16/2006798
clenbuterol Spiropent, Ventipulmin 10/27/2014799
Clindamycin Cleocin 10/28/1988 Pfizer Inc.
Treatment of squamous cell carcinoma of the head and neck.
Matrix Pharmaceutical, Inc.
Treatment of metastatic malignant melanoma.
Matrix Pharmaceutical, Inc.
Treatment of postherpetic neuralgia of the trigeminal nerve
Winston Laboratories, Inc.
Treatment of postherpetic neuralgia.
Winston Laboratories, Inc.
Treatment of neuromyelitis optica and neuromyelitis optica spectrum disorder
Chord Therapeutics S.a.r.l.
Treatment of the chronic progressive form of multiple sclerosis.
Johnson & Johnson Pharmaceutical R & D, LLC
clarithromycin, rifabutin, clofazimine
Treatment of pediatric Crohn's disease.
RedHill Biopharma Ltd.
Treatment of cerebral vasospasm following subarachnoid hemorrhage
Actelion Pharmaceuticals Ltd.
Adjunctive therapy with enzyme replacement therapy in the treatment of Pompe disease
Duke University Medical Center
Prevention of Pneumocystis carinii pneumonia in AIDS patients.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 103 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
800
Clindamycin Cleocin 10/28/1988 Pharmacia & Upjohn801
Clindamycin hydrochloride n/a 8/9/2006 Treatment of sarcoidosis802
clofarabine Clofarex 3/14/2002803
clofazimine Lamprene 6/25/2014 Treatment of active tuberculosis804
Clonazepam Klonopin 8/4/1994805
Clonazepam Intranasal Spray n/a 12/19/2007806
Clotrimazole n/a 4/24/1995 Treatment of sickle cell disease. Brugnara, Carlo M.D.807
Clotrimazole n/a 6/14/2005 AesRx, LLC808
Clotrimazole n/a 3/13/2006809
clotrimazole otic solution n/a 5/10/2016810
n/a 6/18/2012811
Novoseven 6/18/2004 Novo Nordisk, Inc.
Treatment of Pneumocystis carinii pneumonia associated with AIDS patients.
Autoimmunity Research Foundation
Treatment of acute myelogenous leukemia
Genzyme Corp (Ilex Products, Inc.)
Novartis Pharmaceuticals Corp.
Treatment of hyperekplexia (startle disease).
Hoffmann-La Roche, Inc.
Treatment of recurrent acute repetitive seizures
Jazz Pharmaceuticals, Inc.
Topical treatment of children and adults with pouchitis
Treatment of Huntington's disease
EnVivo Pharmaceuticals, Inc.
Treatment of fungal otitis externa (otomycosis).
Laboratorios SALVAT S.A.
CNDO-109-activated allogeneic natural killer cells
Treatment of acute myeloid leukemia
Coronado Biosciences, Inc.
Coagulation factor VIIa (recombinant)
Prevention of bleeding episodes in Glanzmann's thrombasthenia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 104 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
812Coenzyme Q10 n/a 3/5/2001
813
n/a 3/14/2011 Treatment of Friedreich's Ataxia.814
Colchicine n/a 9/25/2007 Treatment of Behcet's Syndrome AR Scientific, Inc.815
n/a 10/13/2016816
n/a 5/18/2016817
n/a 5/2/2016 Treatment of soft tissue sarcoma Immune Design Corp.818
n/a 11/19/2012819
Combretastatin A4 phosphate n/a 5/8/2006 Treatment of ovarian cancer820
Combretastatin A4 Phosphate n/a 7/23/2003
Treatment of Huntington's disease
Integrative Therapeutics, Inc.
coenzyme Q10 and d-alpha-tocopherol
NBI Pharmaceuticals, Inc.
Combination of an inactivated purified rabies virus (IPRV) and the PIKA adjuvant (Poly I:C, kanamycin and calcium)
Treatment of hepatocellular carcinoma
Yisheng US Biopharma, Inc.
combination of disulfiram and copper gluconate
Treatment of glioblastoma multiforme.
Cantex Pharmaceuticals, Inc.
combinatorial regimen of LV305 (lentiviral vector encoding NY-ESO-1 gene) and G305 (NY-ESO-1 recombinant protein plus GLA-SE)
combretastatin A 1 diphosphate
Treatment of acute myelogenous leukemia
Mateon Therapeutics, Inc.
Mateon Therapeutics, Inc.
Treatment of anaplastic thyroid cancer, medullary thyroid cancer, and stage IV papillary or follicular thyroid cancer
Mateon Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 105 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
821
n/a 3/10/2016822
n/a 8/6/2013823
Transmid 12/3/2001824
conjugated bile acids Cobartin 7/18/2003 Jarrow Formulas, Inc.825
Contulakin-G n/a 7/7/2005 Cognetix, Inc.826
copanlisib n/a 2/5/2015827
Copper 64-DOTA-TATE Netmedix (Tm) 5/18/2016 RadioMedix, Inc.828
copper histidine n/a 5/14/2012 Treatment of Menkes disease829
Cordycepin n/a 7/5/2007 OncoVista, Inc.830
corifungin n/a 8/22/2011
competetive small molecule inhibitor of the enzyme O-GlcNAcase
Treatment of progressive supranuclear palsy.
Merck & Company, Inc.
Conjugate of a dengue virus specific small chemical ligand and an amphiphilic PEG based polymer
Treatment of dengue fever (includes dengue hemorrhagic fever and dengue shock syndrome)
NanoViricides Incorporated
conjugate of human transferrin and a mutant diphtheria toxin (CRM 107)
Treatment of malignant tumors of the central nervous system
Xenova Biomedix Limited
Treatment of steatorrhea in patients with short bowel syndrome
Intrathecal treatment of neuropathic pain associated with spinal cord injury
Treatment of follicular lymphoma
Bayer HealthCare Pharmaceuticals, Inc.
Diagnostic for the management of neuroendocrine tumors.
Stephen G. Kaler, M.D.
Treatment of TdT-positive acute lymphocytic leukemia
Treatment of amebic meningoencephalitis.
Ctr for Discovery & Innovation in Parasitic Diseas
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 106 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
831corifungin n/a 7/6/2011
832Xerecept 4/6/1998
833Coumarin Onkolox 12/22/1994 Drossapharm LTD
834coversin; rEV675 n/a 9/8/2016 Akari Therapeutics Plc
835
Coxsackievirus A21 Cavatak 12/15/2005 Viralytics Limited836
Creatine Creapure 10/11/2005837
creatine Creapure 2/12/2002 Avicena Group, Inc.838
crenolanib n/a 3/18/2011 Treatment of soft tissue sarcoma839
crenolanib n/a 10/31/2012840
crenolanib besylate n/a 12/20/2010 Treatment of malignant glioma841
cridanimod n/a 1/12/2011842 crizotinib Xalkori 10/31/2012 Treatment of neuroblastoma Pfizer, Inc.
Treatment of visceral leishmaniasis
Sandler Center for Drug Discovery
Corticotropin-releasing factor, human
Treatment of peritumoral brain edema.
Neurobiological Technologies, Inc.
Treatment of renal cell carcinoma.
Treatment of paroxysmal nocturnal hemoglobinuria
Treatment of stage II (T4), stage III, and stage IV melanoma
Treatment of Huntington's disease
Marathon Pharmaceuticals, LLC
Treatment of amyotrophic lateral sclerosis
AROG Pharmaceuticals, LLC
Treatment of acute myelogenous leukemia
AROG Pharmaceuticals, LLC
AROG Pharmaceuticals, LLC
Treatment of progesterone receptor negative endometrial cancer in conjunction with progesterone therapy.
Xenetic Biosciences, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 107 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
843crizotinib Xalkori 9/28/2012 Pfizer, Inc.
844n/a 12/9/2015 Treatment of mastocytosis. Patara Pharma, LLC
845
n/a 12/30/1991 ImmuCell Corporation846
cultured skin substitute (CSS) Novaderm 8/22/2016 Regenicin Inc.847
n/a 8/15/2003848
n/a 11/5/2009849
n/a 12/20/2006 Lux Biosciences, Inc.
Treatment of anaplastic large cell lymphoma
cromolyn sodium for inhalation
Cryptosporidium hyperimmune bovine colostrum IgG concentrate
Treatment of diarrhea in AIDS patients caused by infection with Cryptosporidium parvum.
Treatment of burns requiring skin grafting.
cultured, partially T-Cell depleted, allogenic thymic tissue for transplantation
As a therapy for primary immune deficiency resulting from athymia associated with complete DiGeorge Syndrome
Duke University Medical Center
cyclic pyranopterin monophosphate (cPMP)
Treatment of molybdenum cofactor deficiency type A (MoCD)
Alexion Pharmaceuticals, Inc.
Cyclo {{(E,Z)-(2S,3R,4R)-3-hydroxy-4-methyl-2-(methylamino)nona-6,8-dienoyl}-L-2-aminobytyrl-N-methyl-glycyl-N-methyl-L-leucyl-L-valyl-N-methyl-L-leucyl-L-alanyl-D-alanyl-N-methyl-L-leucyl-N-methyl-L-leucyl-N-methyl-L-valyl}
Treatment and chronic control of non-infectious posterior, intermediate and pan-uveitis
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 108 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
850
n/a 6/24/2013 Treatment of acromegaly851
cyclocreatine n/a 6/18/2012 Lumos Pharma852
cyclophosphamide Cyrevia(Tm) 6/17/2011853
cyclophosphamide Cyrevia(Tm) 6/7/2011 Treatment of systemic sclerosis.854
Cyclosporin A Mitogard 10/29/2004 Maas Biolab,LLC855
cyclosporine n/a 2/17/2009856
cyclosporine n/a 2/17/2009857
Cyclosporine n/a 11/25/2003858
Cyclosporine Pluminiq 11/25/2003
cyclo(-y-aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonly-L-phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt
Aspireo Pharmaceuticals Limited
Treatment of creatine transporter deficiency
Prevention of graft versus host disease following allogeneic hematopoietic stem cell transplant
Accentia Biopharmaceuticals, Inc.
Accentia Biopharmaceuticals, Inc.
Treatment of amyotrophic lateral sclerosis and its variants
Prophylaxis of graft-versus-host disease
Sigmoid Pharma Limited
Treatment of graft-versus-host disease
Sigmoid Pharma Limited
Prophylaxis of organ rejection in patients receiving allogeneic lung transplant
APT Pharmaceuticals, Inc.
Treatment of acute rejection in patients requiring allogenic lung transplants
APT Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 109 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
859
n/a 8/1/1991 Allergan, Inc.860
n/a 8/1/1991 Allergan, Inc.861
cyclosporine A n/a 9/30/2008 Santen SAS862
Cyclosporine A n/a 5/4/2007 Santen SAS863
cyclosporine A Nova22007 4/9/2008 Santen SAS864
n/a 12/6/2000865
Cyclosporine ophthalmic Optimmune 11/9/1988 University Of Georgia866
cyclosporine; ciclosporin 11/23/2010867
n/a 12/29/2015 Vical Incorporated
cyclosporine 2% ophthalmic ointment
For use in corneal melting syndromes of known or presumed immunologic etiopathogenesis, including Mooren's ulcer
Cyclosporine 2% ophthalmic ointment
Treatment of patients at high risk of graft rejection following penetrating keratoplasty
Prevention of corneal graft rejection
Treatment of vernal keratoconjunctivitis
Treatment of herpes simplex virus stromal keratitis
Cyclosporine in combination with omega-3 polyunsaturated fatty acids
Prevention of solid organ graft rejection.
RTP Pharma Corporation
Treatment of severe keratoconjunctivitis sicca associated with Sjogren's syndrome.
Neurostat(R)/Ciclomulsion(R)
Treatment of moderate to severe traumatic brain injury.
NeuroVive Pharmaceutical AB
Cyclo{-Asn-Leu-d-Phe-Al[(N5-acetyl-N5-hydroxy-Orn)-(N5-acetyl-N5-hydroxy-Orn)-(N5-hydroxy-Orn)]-} . Al(III)
Treatment of invasive aspergillosis.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 110 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
868
Cys-His-Ala-Val-Cys n/a 2/14/2008869 Cysteamine Lynovex 9/11/2014 Treatment of cystic fibrosis NovaBiotics Ltd.870
Cysteamine n/a 5/1/1986 Thoene, Jess G., M.D.871
cysteamine n/a 5/9/2008 Horizon Orphan LLC872
cysteamine n/a 9/11/2013 Treatment of pancreatic cancer Horizon Orphan LLC873
cysteamine n/a 8/6/2008874
Adgvcftr.10 3/9/1995 Treatment of cystic fibrosis. GenVec, Inc.875
n/a 1/8/1993 Treatment of cystic fibrosis. Genetic Therapy, Inc.876 cytarabine liposome Depocyt 1/30/2007 Treatment of gliomas Bruce Frankel, MD877
n/a 8/22/2008878
n/a 6/17/2011
For use in conjunction with melphalen for the treatment of malignant melanoma, AJCC stages IIB, IIC, III and IV
Adherex Technologies, Inc.
Treatment of nephropathic cystinosis.
Treatment of Huntington's disease
Treatment of neuronal ceroid lipofuscinoses (Batten disease)
Raptor Pharmaceuticals, Inc.
Cystic fibrosis Tr gene therapy (recombinant adenovirus)
Cystic fibrosis transmembrane conductance regulator gene
Cytarabine:daunorubicin liposome injection
Treatment of acute myeloid leukemia
Celator Pharmaceuticals, Inc.
cytochrome C, flavin mononucleotide and thiamin diphosphate
Treatment of mitochondrial disorders
NBI Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 111 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
879
n/a 12/17/2014880
n/a 9/23/2006 Vical Inc.881
n/a 6/3/2005882
n/a 1/28/1991 Bayer Corporation883
D-mannitol and L-proline Prodarsan 4/20/2009 DNage B.V.884
d-methadone n/a 6/2/2016
cytochrome P450 isoform 2B1 gene transfected human embryonic kidney 293 cells encapsulated in polymeric cellulose sulphate
Treatment of pancreatic cancer in combination with ifosfamide
PharmaCyte Biotech, Inc.
Cytomegalovirus DNA Vaccine w/Copolymer/Benzalkonium Chloride
For use in prevention of clinically significant CMV viremia, CMV disease and associated complications in at-risk hematopoietic cell transplant and solid organ transplant populations
Cytomegalovirus DNA vaccine with plasmids expressing pp65 and gB genes
Prevention of clinically significant cytomegalovirus (CMV) viremia, CMV disease and associated complications in at-risk hematopoietic cell transplant and solid transplant populations
Astellas Pharma Global Development, Inc.
Cytomegalovirus immune globulin intravenous (human)
For use in conjunction with ganciclovir sodium for the treatment of cytomegalovirus pneumonia in bone marrow transplant patients.
Treatment of Cockayne syndrome
Treatment of postherpetic neuralgia
Relmada Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 112 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
885D-peptide Nerofe 2/4/2011
886Pulmadex 10/23/2002 Treatment of cystic fibrosis Demegen, Inc.
887
n/a 11/5/2014888
dabrafenib Tafinlar 2/8/2016889
dabrafenib Tafinler 10/20/2014890
dabrafenib and trametinib 9/1/2016891
dabrafenib and trametinib n/a 10/29/2015892
dacetuzumab n/a 8/13/2004 Treatment of multiple myeloma. Seattle Genetics, Inc.893
dacetuzumab n/a 10/6/2005 Seattle Genetics, Inc.894
dacomitinib n/a 3/3/2015 Pfizer, Inc.
Treatment of acute myelogenous leukemia
Immune System Key, Ltd
D-peptide of the sequence AKRHHGYKRKFH - NH2
d6-tetrabenazine, deutetrabenazine
Treatment of Huntington's Disease
Teva Branded Pharmaceutical Products R&D, Inc.
Treatment of malignant glioma with BRAF V600 mutation
Novartis Pharmaceuticals Corporation
Treatment of patients with BRAF mutation positive non-small cell lung cancer
Novartis Pharmaceuticals Corp
Tafinlar(R) Capsules A Nd Mekinist(R) Tablets
Treatment of patients with anaplastic thyroid cancer and locally advanced or metastatic papillary thyroid cancer whose tumors harbor a BRAF V600 mutation.
Novartis Pharmaceuticals Corp.
Treatment of patients with BRAF mutation positive non-small cell lung cancer.
Novartis Pharmaceuticals Corp.
Treatment of chronic lymphocytic leukemia
Treatment of non-small cell lung cancer with EGFR, HER2, HER4, or DDR2 mutations.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 113 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
895
dalbavancin Dalvance 3/30/2015896
dantrolene sodium n/a 2/17/2016 Treatment of Wolfram Syndrome897
dantrolene sodium n/a 9/25/2012 Treatment of heat stroke898
Dapsone n/a 11/7/1994899
Dapsone USP Dapsone 1/8/1992900
Dapsone USP Dapsone 12/24/1991901
daratumumab Humax (R) - Cd38 11/9/2015902
daratumumab n/a 8/20/2015903
daratumumab n/a 8/6/2015
Treatment of acute osteomyelitis in children (0 through 16 years of age)
Durata Therapeutics International B.V.
Washington University in St. Louis
Eagle Pharmaceuticals, Inc.
Prophylaxis of toxoplasmosis in severely immunocompromised patients with CD4 counts below 100.
Jacobus Pharmaceutical Company, Inc.
For the combination treatment of Pneumocystis carinii pneumonia in conjunction with trimethoprim.
Jacobus Pharmaceutical Company
Prophylaxis for Pneumocystis carinii pneumonia.
Jacobus Pharmaceutical Company
Treatment of diffuse large B-cell lymphoma.
Janssen Research and Development, LLC
Treatment of mantle cell lymphoma.
Janssen Research & Development, LLC
Treatment of follicular lymphoma
Janssen Research & Development, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 114 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
904Darinaparsin n/a 9/13/2010 Solasia Pharma K.K.
905dasiprotimut-T Biovax Id 6/17/2010
906dasiprotimut-T Biovaxid 10/28/2009
907Daunorubicin liposomal n/a 9/5/2008 Diatos USA, LLC
908davunetide n/a 12/7/2009
909 DCVAC OvCa n/a 5/14/2013 Treatment of ovarian cancer SOTIOS a.s.910
DEAE-rebeccamycin n/a 3/1/2004 Treatment of bile duct tumors Helsinn Healthcare SA911
debrase Debridase 8/20/2003 MediWound, Ltd.912
n/a 9/19/2016 Treatment of sickle cell disease EpiDestiny Inc.913 defactinib n/a 2/12/2015 Treatment of ovarian cancer Verastem, Inc.914
deferiprone n/a 7/31/2008 Treatment of Friedreich's ataxia ApoPharma, Inc.915
deferiprone n/a 9/1/2011 Treatment of superficial siderosis ApoPharma, Inc.916
n/a 1/28/2005917
n/a 12/21/1998
Treatment of peripheral T-cell lymphoma.
Treatment of mantle cell lymphoma
Biovest International, Inc.
Treatment of follicular lymphoma
Biovest International, Inc.
Treatment of acute myeloid leukemia
Treatment of progressive supranuclear palsy.
Allon Therapeutics, Inc.
Debridement of acute, deep dermal burns in hospitalized patients
decitabine and tetrahydrouridine
deferoxamine starch conjugate
Treatment of acute iron poisoning
Biomedical Frontiers, Inc.
deferoxamine starch conjugate
Treatment of chronic iron overload resulting from conventional transfusional treatment of beta-thalassemia major and sickle cell anemia.
Biomedical Frontiers, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 115 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
918
defibrotide n/a 1/8/2007919
Defibrotide n/a 7/5/1985 Crinos International920
deflazacort Calcort 9/16/2010921
deflazacort n/a 8/16/2013922
deflazacort n/a 10/22/2015923
dehydrated alcohol n/a 3/16/2012924
dehydrated alcohol Ablysinol 9/11/2013925
Fidelin 8/19/2003 Paladin Labs, Inc.926
n/a 1/29/1997 Pharmadigm, Inc.
For the prevention of hepatic veno-occlusive disease.
Jazz Pharmaceuticals, Inc.
Treatment of thrombotic thrombocytopenic purpura.
Treatment of Duchenne muscular dystrophy
University of Rochester Medical Center
Treatment of Duchenne muscular dystrophy
Marathon Pharmaceuticals, LLC
Treatment of pediatric (0 through 16 years of age) juvenile idiopathis arthritis (JIA) International League of Associations for Rheumatology (ILAR) categories excluding systemic JIA.
Marathon Pharmaceuticals, LLC
Treatment of trigeminal neuralgia
Luitpold Pharmaceuticals, Inc.
Treatment of hypertrophic obstructive cardiomyopathy
Belcher Pharmaceuticals, LLC
Dehydroepiandrosterone (DHEA)
Replacement therapy in individuals with adrenal insufficiency
Dehydroepiandrosterone sulfate sodium
Treatment of serious burns requiring hospitalization.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 116 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
927
n/a 1/28/1997 Pharmadigm, Inc.928
n/a 12/2/2011929
n/a 12/3/2015 Treatment of glioma. GW Research Ltd.930
demcizumab n/a 4/30/2014 Treatment of pancreatic cancer931
n/a 6/7/2010932
dendritic hybrid cell vaccine Neuroblaxin 9/23/2011 Treatment of neuroblastoma933
n/a 1/6/2016 Immune Design Corp.934
denileukin diftitox Ontak 4/30/2010 Eisai, Inc.935
denileukin diftitox n/a 7/12/2013 Eisai Inc.
Dehydroepiandrosterone sulfate sodium
To accelerate the re-epithelialization of donor sites in those hospitalized burn patients who must undergo autologous skin grafting.
delta-1,4,9(11)-pregnatriene-17-alpha,21-dihydroxy-16-alpha-methyl-3,20-dione
Treatment of Duchenne muscular dystrophy
ReveraGen Biopharma
delta-9-tetrahydrocannibinol and cannabidiol
OncoMed Pharmaceuticals, Inc.
dendritic cells pulsed with synthetic peptides derived from antigens MAGE1, HER-2, AIM-2,TRP-2, gp100 and interleukin-13 receptor alpha
Treatment of glioblastoma or brain stem glioma
ImmunoCellular Therapeutics Ltd.
Orbis Health Solutions, LLC
dendritic-cell targeting, lentiviral vector encoding the NY-ESO-1 gene
Treatment of soft tissue sarcoma.
Treatment of peripheral T-cell lymphoma
Treatment of cutaneous T-cell lymphoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 117 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
936denileukin diftitox n/a 6/29/2011 Eisai, Inc.
937
n/a 7/7/2016938
Dermagraft n/a 12/13/2010939
Deslorelin Somagard 11/5/1987940
n/a 10/26/2004 Treatment of pemphigus vulgaris Peptimmune, Inc.941
deutetrabenazine n/a 1/13/2015942
dexamethasone phosphate n/a 12/2/2008943
n/a 7/24/2012 EryDel S.p.A.944
dexanabinol n/a 8/11/2004 Pharmos Corporation945
dexpramipexole n/a 10/11/2007
Treatment of peripheral T-cell lymphoma (PTCL)
deoxythymidine monophosphate and deoxycytidine monophosphate
Treatment of thymidine kinase 2 deficiency.
Columbia University Medical Center
Treatment of epidermolysis bullosa
Shire Regenerative Medicine, Inc.
Treatment of central precocious puberty.
Roberts Pharmaceutical Corp.
Desmoglein 3 synthetic peptide (PI-0824)
Treatment of Tourette syndrome in the pediatric population (defined as 0 through 16 years of age)
TEVA Pharmaceuticals
Treatment of corneal graft rejection.
EyeGate Pharmaceuticals, Inc.
dexamethasone sodium phosphate encapsulated in autologous erythrocytes
Treatment of ataxia-telangiectasia
For the attenuation or amelioration of the long-term neurological sequelae associated with moderate and severe traumatic brain injury
Treatment of amyotrophic lateral sclerosis
Knopp Biosciences LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 118 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
946
dexrazoxane hydrochloride n/a 8/19/2014 Satiscor, LLC947
dextran 1 n/a 3/21/2003 Treatment of cystic fibrosis BCY LifeSciences Inc.948
Dextran 70 Dehydrex 3/5/1990949
Dextran and deferoxamine Bio-Rescue 3/8/1991950
Uendex 10/5/1990951
Dextran sulfate sodium n/a 11/19/1987952
diacerein n/a 10/15/2014953
6/11/1992954
diannexin n/a 10/28/2009
Prevention of cardiomyopathy for chilren and adolescents 0 through 16 years of age treated with anthracyclines
Treatment of recurrent corneal erosion unresponsive to conventional therapy.
Holles Laboratories, Inc.
Treatment of acute iron poisoning.
Biomedical Frontiers, Inc.
Dextran sulfate (inhaled, aerosolized)
For use as an adjunct to the treatment of cystic fibrosis.
Kennedy & Hoidal, M.D.'s
Treatment of aquired immunodeficiency syndrome.
Ueno Fine Chemicals Industry, Ltd.
Treatment of epidermolysis bullosa
Castle Creek Pharmaceuticals
Dianeal peritoneal dialysis solution with 1.1% amino acids
Nutrineal (Peritoneal Dialysis Solution With 1.1% Amino Acid
For use as a nutritional supplement for the treatment of malnourishment in patients undergoing continuous ambulatory peritoneal dialysis.
Baxter Healthcare Corporation
Prevention of ischemia-reperfusion injury in solid organ transplants
Astellas Pharma Global Development, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 119 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
955
diazepam (intranasal) n/a 11/16/2015956
diazepam (intranasal) n/a 7/31/2012957
diazepam auto-injector n/a 5/30/2013958
Diazepam buccal soluble film n/a 11/10/2016 MonoSol Rx, LLC959
Diazepen(Tm) 5/26/2016960
diazoxide n/a 12/3/2012 Sedogen, LLC961
diazoxide choline n/a 5/13/2014 Essentialis, Inc.962
Diethyldithiocarbamate Imuthiol 4/3/1986 Treatment of AIDS.
Management of acute repetitive seizures.
Neurelis Pharmaceuticals, Inc.
Management of patients with acute repetitive seizures
Acorda Therapeutics, Inc.
Management of selected, refractory patients with epilepsy on stable regimens of antiepileptic drugs, who require intermittent use of diazepam to control bouts of increased seizure activity
Meridian Medical Technologies-a Pfizer subsidiary
Treatment of selected, refractory patients with epilepsy who are on stable regimens of antiepileptic drugs (AED) and who require intermittent use of diazepam to control bouts of increased seizure activity (acute repetitive seizures)
diazepam subcutaneous injection
Treatment of acute repetitive seizures.
Xeris Pharmaceuticals, Inc.
Treatment of Prader Willi Syndrome
Treatment of Prader-Willi Syndrome
Connaught Laboratories
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 120 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
963
n/a 4/14/2004 CIS-US964
diferuloylmethane n/a 6/13/2003 Treatment of cystic fibrosis965 digitoxin n/a 11/2/2001 Treatment of ovarian cancer SimRx Advisors LLC966
digitoxin n/a 10/18/2001 SimRx Advisors LLC967
digitoxin n/a 5/27/2005 Treatment of cystic fibrosis968
digoxin immune fab (ovine) n/a 2/3/2012 Velo Bio, LLC969
Digoxin immune fab(ovine) Digidote 3/11/1985970
Eurartesim 1/8/2007971
Dihydrotestosterone Androgel -Dht 2/5/1996
diethylenetriaminepentaacetate (DPTA)
For treatment of patients with known or suspected internal contamination with plutonium, americium or curium to increase the rates of elimination.
Allertein Therapeutics, LLC
Treatment of soft tissue sarcomas
Silver Pharmaceuticals
Treatment of severe preeclampsia and eclampsia
Treatment of life-threatening acute cardiac glycoside intoxication manifested by conduction disorders, ectopic ventricular activity and (in some cases) hyperkalemia.
Boehringer Mannheim Corp.
dihydroartemisinin and piperaquine
Treatment of uncomplicated malaria caused by "Plasmodium falciparum," Plasmodium vivax," Plasmodium malariae," or "Plasmodium ovale."
Sigma-Tau Pharmaceuticals, Inc.
Treatment of weight loss in AIDS patients with HIV-associated wasting.
Besins Internaitonal, US Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 121 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
972dimebon n/a 5/12/2009 Medivation, Inc.
973
n/a 11/24/1999974
dimethyl fumarate n/a 9/11/2013 Treatment of Friedreich's Ataxia Gino Cortopassi975
Dimethyl sulfoxide n/a 11/22/1994976
dimethyl sulfoxide n/a 5/9/2008977
Dimethylsulfoxide n/a 4/6/1998978
Dimethylsulfoxide n/a 4/15/1997979
dinaciclib n/a 8/25/2011
Treatment of Huntington's Disease.
Dimerizing drug that binds to mutated Fas protein/drug-binding domain fusion protein (FKBP)
Treatment of acute graft-versus-host disease in patients undergoing bone marrow transplantation.
Bellicum Pharmaceuticals, Inc.
Treatment of increased intracranial pressure in patients with severe, closed-head injury, also known as traumatic brain coma, for whom no other effective treatment is available.
Abela Pharmaceuticals, Inc.
For use in combination with antimicrobial drugs for the treatment of drug resistant tuberculosis
Abela Pharmaceuticals, Inc.
Treatment of palmar-plantar erythrodysethesia syndrome.
Cancer Technologies, Inc.
Topical treatment for the prevention of soft tissue injury following extravastion of cytotoxic drugs.
Cancer Technologies, Inc.
Treatment of chronic lymphocytic leukemia.
Merck Sharp & Dohme Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 122 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
980
Alec 7/28/1988 Forum Products, Inc.981
diphenylcyclopenone n/a 6/13/2003 Lloyd E. King, Jr.982
Diphenylcyclopropenone gel Samcyprone (Tm) 4/15/2015983
dipraglurant n/a 12/29/2015 Addex Pharma SA984
Immther 3/1/1990985
Disodium clodronate n/a 6/16/1993986
Bonefos 3/5/1990987
Disufenton sodium n/a 8/22/2016 Oblato, Inc.
Dipalmitoylphosphatidylcholine /phosphatidylglycerol
Prevention and treatment of neonatal respiratory distress syndrome.
Treatment of chronic severe forms of alopecia areata (Alopecia Totalis [AT]/Alopecia Universalis [AU])
Treatment of malignant melanoma stage IIB to IV
RXi Pharmaceuticals Corp.
Treatment of levodopa-induced dyskinesias
Disaccharide tripeptide glycerol dipalmitoyl
Treatment of pulmonary and hepatic metastases in patients with colorectal adenocarcinoma.
ImmunoTherapeutics, Inc.
Treatment of hypercalcemia of malignancy.
Discovery Experimental & Development, Inc.
Disodium clodronate tetrahydrate
Treatment of increased bone resorption due to malignancy.
Anthra Pharmaceuticals, Inc.
Treatment of glioblastoma multiforme.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 123 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
988
n/a 9/19/2016 Treatment of glioma.989
n/a 8/20/2009 Treatment of ovarian cancer990
n/a 2/6/2014 ViroMed Co., Ltd.991
n/a 12/13/2010 Treatment of multiple myeloma.992
n/a 7/24/2012993
n/a 7/24/2012
disulfide-stabilized single-chain variable region fragment of the anti-EGFR wild type/EGFRvIII dual specificity monoclonal antibody with variable fragments fused to a truncated version of the Pseudomonas exotoxin A, PE38, and KDEL
Darell D. Bigner, MD, PhD
DNA plasmid expressing diphtheria toxin triggered by the presence of oncofetal gen H19
BioCancell Therapeutics Ltd.
DNA plasmid vector (pCK-HGFX7) expressing human hepatocyte growth factor
Treatment of amyotrophic lateral sclerosis
DNA plasmid vector expressing eIF5Ak50
Senesco Technologies, Inc.
DNA plasmid vector expressing eIF5Ak50R protein, siRNA directed against the nastive eIF5A mRNA, and PEI (polyethyleneimine)
Treatment of diffuse large B cell lymphoma
Senesco Technologies, Inc.
DNA plasmid vector expressing eIF5Ak50R protein, siRNA directed against the native eIF5A mRNA, and PEI (polyethyleneimine)
Treatment of mantle cell lymphoma
Senesco Technologies, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 124 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
994
n/a 4/4/2005 Treatment of ovarian cancer. Celsion Corporation995
Leuvectin 4/28/2000 Vical Incorporated996
O-Vax 9/21/2000997
docosahexaenoic acid n/a 4/27/2015 Treatment of sickle cell disease998
docosahexaenoic acid n/a 6/1/2015999
docosahexaenoic acid, DHA n/a 12/17/20141000
n/a 2/11/2016 Treatment of sickle cell disease NuvOx Pharma1001
n/a 12/2/2015 NuvOx Pharma1002
domperidone n/a 9/2/2011
DNA plasmid vector expressing human IL-12 gene
DNA-lipid complex (DMRIE/DOPE)/plasmid vector (VCL-1102, Vical) expressing human interleukin-2
Treatment of renal cell carcinoma.
DNP-Modified autologous tumor vaccine
Adjuvant therapy for the treatment of ovarian cancer
AVAX Technologies, Inc.
Sancilio & Company, Inc.
Treatment of short bowel syndrome
Sancilio and Company, Inc.
Treatment of primary sclerosing cholangitis
Sancilio and Company, Inc.
dodecafluoropentane emulsion
dodecafluoropentane emulsion (DDFPe)
Radiosensitizer to be used during radiation treatment of glioblastoma multiforme (GBM).
Treatment of hypoprolactinemia in breastfeeding mothers, and in some hypoprolactinemic conditions following the use of cabergoline or bromocriptine in mothers who wish to initiate or return to breastfeeding
Thomas W. Hale, RPh, PhD
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 125 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1003
Doripenem n/a 7/16/2004 Shionogi, Inc.1004
n/a 6/9/20151005
n/a 1/26/2015 Treatment of pancreatic cancer Silenseed Ltd1006
n/a 8/6/2010 Treatment of pancreatic cancer1007
dovitinib n/a 9/26/20131008
DOXO-EMCH n/a 4/18/2011 CytRx Corporation1009
doxofylline n/a 2/14/2014 Treatment of bronchiectasis1010
doxorubicin n/a 8/25/2009 Delcath Systems, Inc.1011
doxorubicin n/a 4/6/2015 Louis D. Falo, Jr.
Treatment of bronchopulmonary infection in patients with cystic fibrosis who are colonized with Pseudomonas aeruginosa or Burkholderia cepacia.
double stranded oligomer AD00370 RNA interference-based liver targeted therapeutic
Treatment of Alpha-1 Antitrypsin deficiency
Arrowhead Research Corporation
double stranded RNA which targets the mutated KRAS oncogene
Double-stranded DNA plasmid carrying the gene for the diphtheria toxin A (DT-A) chain
BioCancell Therapeutics Israel Ltd.
Treatment of adenoid cystic carcinoma
Novartis Pharmaceuticals Corporation
Treatment of adenocarcinoma of the pancreas.
Alitair Pharmaceuticals, Inc.
Treatment of hepatocellular carcinoma.
Treatment of cutaneous T-cell lymphoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 126 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1012
n/a 10/7/2005 Supratek Pharma Inc.1013
Doxorubicin Transdrug 3/14/2005 BioAlliance Pharma1014
n/a 2/20/2008 Treatment of gastric cancer Supratek Pharma, Inc.1015
DPX-Survivac n/a 7/14/2015 Treatment of ovarian cancer.1016
n/a 8/7/2014 Birken AG1017 Duramycin n/a 12/11/1997 Treatment of cystic fibrosis. Lantibio, Inc.1018
dusquetide n/a 8/10/2016 Soligenix, Inc.1019
duvelisib n/a 4/15/20131020
duvoglustat hydrochloride n/a 6/18/2007 Treatment of Pompe disease1021
Dynamine n/a 10/16/1991 Mayo Foundation1022
Dynamine n/a 2/5/1990 Mayo Foundation1023
E-selectin antagonist n/a 5/13/2015 GlycoMimetics, Inc.
Doxorubicin HCl with pluronic L-61 and pluronic F-127
Treatment of esophageal carcinoma
doxorubicin PIHCA nanoparticles
Treatment of hepatocellular carcinoma
doxorubicin with pluronics F-127 and L-61
Immunovaccine Technologies, Inc.
dry extract from Betulae Cortex (birch bark)
Treatment of epidermolysis bullosa
Treatment of macrophage activation syndrome
Treatment of chronic lymphocytic leukemia and small lymphocytic lymphoma
Infinity Pharmaceuticals
Amicus Therapeutics, Inc
Treatment of hereditary motor and sensory neuropathy type I (Charcot-Marie-Tooth disease).
Treatment of Lambert Eaton myasthenic syndrome.
Treatment of acute myeloid leukemia (AML)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 127 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1024
n/a 2/8/20161025
echinomycin n/a 5/21/2015 OncoImmune, Inc.1026
echothiophate iodide n/a 6/2/2014 Treatment of Stargardt's disease Makindus, Inc.1027
ecopipam hydrochloride n/a 7/21/20091028
ecopipam hydrochloride n/a 9/29/20101029
eculizumab Soliris 6/24/20131030
eculizumab n/a 6/12/2014 Treatment of Myasthenia Gravis.1031
eculizumab n/a 3/5/2001
Echinocandin B,1-[(4R,5R)-4-hydroxy-N2-[[4’’-(pentyloxy)[1,1’:4’,1’’-terphenyl]-4-yl]carbonyl]-5-[2-(trimethylammonio)ethoxy]-L-ornithine]-4-[(4S)-4-hydroxy-4-(4-hydroxyphenyl)-L-allothreonine]
Treatment of candidemia and invasive candidiasis infections caused by susceptible Candida spp.
Cidara Therapeutics, Inc.
Treatment of acute myeloid leukemia.
Symptomatic treatment of self injurious behaviors in patients with Lesch-Nyhan disease.
Psyadon Pharmaceuticals, Inc.
Treatment of Tourette's syndrome in children 0-16 years old.
Psyadon Pharmaceuticals, Inc.
Treatment of neuromyelitis optica
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Treatment of idiopathic membranous glomerular nephropathy
Alexion Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 128 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1032
eculizumab Soliris 10/18/20111033
Eculizumab n/a 9/21/2000 Treatment of dermatomyositis1034
eculizumab Soliris 1/10/20141035
edaravone n/a 5/12/20151036
edaravone n/a 3/12/2015 Treeway B.V.1037
Edotreotide Onalta(Tm) 7/28/20051038
efaproxiral n/a 7/28/20041039
Efdispo (tm) Efdispo (Tm) 1/15/2013 Treatment of Ewings Sarcoma.1040
eflornithine n/a 2/4/20111041
Eflornithine n/a 9/29/2006 Treatment of anaplastic glioma
Treatment of Shiga-Toxin producing escherichia coli hemolytic uremic syndrome
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Prevention of delayed graft function after renal transplantation
Alexion Pharmaceuticals, Inc.
Treatment of amyotrophic lateral sclerosis
Mitsubishi Tanabe Pharma Corporation
Treatment of amyotrophic lateral sclerosis
Treatment of somatostatin receptor-positive neuroendocrine gastroenteropancreatic tumors
Molecular Insight Pharmaceuticals, Inc. (Progenics Subsidiary)
Adjunct to whole brain radiation therapy for the treatment of brain metastases in patients with breast cancer
Allos Therapeutics, Inc.
TDP Biotherapeutics, Inc.
Treatment of Familial Adenomatous Polyposis
Cancer Prevention Pharmaceuticals
Orbus Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 129 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1042
eflornithine n/a 11/23/2010 Treatment of neuroblastoma1043
eflornithine HCL n/a 4/7/2015 Treatment of gastric cancer1044
eflornithine plus sulindac n/a 1/22/20131045
eicosapentaenoic acid n/a 3/8/20111046
elacytarabine n/a 6/18/2008 Clavis Pharma ASA1047
Elcatonin n/a 9/25/1995 Innapharma, Inc.1048
Elesclomol n/a 12/18/20071049
eltoprazine HCl n/a 2/8/20161050
emodepside n/a 9/13/20161051
emricasan n/a 11/20/2013
Cancer Prevention Pharmaceutical, Inc.
Cancer Prevention Pharmaceuticals, Inc.
Treatment of familial adenomatous polyposis
Cancer Prevention Pharmaceuticals, Inc.
Treatment of familial adenomatous polyposis
S.L.A. Pharma Ltd. (UK)
Treatment of acute myeloid leukemia (AML)
Intrathecal treatment of intractable pain.
Treatment of metastatic melanoma in combination with paclitaxel
Synta Pharmaceuticals Corporation
Treatment of levodopa-induced dyskinesia associated with Parkinson's disease.
Amarantus BioScience Holdings, Inc.
Treatment of onchocerhiasis ("river blindness") caused by the filarial nematode onchocerca volvulus
Bayer HealthCare Pharmaceuticals, Inc.
Treatment of liver transplant recipients with reestablished fibrosis to delay the progression to cirrhosis and end stage liver disease
Conatus Pharmaceuticals Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 130 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1052Enadoline hydrochloride n/a 1/28/1997 Treatment of severe head injury.
1053
Betarx 7/5/1995 VivoRx1054
n/a 8/19/2015 Seres Health, Inc.1055
enchochleate amphotericin B n/a 1/10/20141056
encochleated amikacin n/a 3/3/20161057
encorafenib n/a 11/19/20131058
encorafenib + binimetinib n/a 11/19/2013 Array BioPharma, Inc.1059
n/a 1/21/2016 ENB Therapeutics, LLC1060
n/a 5/10/2016 SCYNEXIS, Inc.1061
n/a 2/6/20141062
eniluracil n/a 12/15/2005
Warner-Lambert Company
Encapsulated porcine islet preparation
Treatment of type I diabetic patients who are already on immunosuppression.
encapsulated spores from fecal microbiota
Treatment of recurrent Clostridium difficile infection (CDI)
Treatment of visceral leishmaniasis
Aquarius Biotechnologies, Inc.
Treatment of non-tuberculous mycobacteria (NTM) infections
Aquarius Biotechnologies, Inc.
Treatment of Stage IIB-IV melanoma positive for BRAF mutation
Array BioPharmas, Inc.
Treatment in Stage IIB-IV melanoma positive for the BRAF mutation.
Endothelin receptor B antagonist
Treatment of malignant melanoma Stages IIB to IV.
enfumafungin derivative B-(1,3)-D-glucan synthestis inhibitor
Treatment of invasive Candida infections, including candidemia
Engineered variant of recombinant human fibroblast growth factor 19
Treatment of primary biliary cirrhosis
NGM Biopharmaceuticals, Inc.
Treatment of hepatocellular carcinoma.
Adherex Technologies, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 131 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1063ensituximab n/a 10/21/2010 Treatment of pancreatic cancer.
1064
n/a 12/22/2014 Treatment of neuroblastoma Ignyta, Inc.1065
enzastaurin n/a 3/4/20091066
Enzastaurin n/a 9/19/20051067
epacadostat n/a 2/11/2016 Incyte Corporation1068
n/a 10/5/1987 Chiron Vision1069
Epoetin alfa n/a 12/20/19931070
Epoetin alpha Procrit 8/27/19871071
Epoetin alpha Procrit 3/7/19891072
Epoetin beta Marogen 10/22/1987 Chugai-USA, Inc.
Neogenix Oncology, Inc.
entrectinib;N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide
Treatment of diffuse large B-cell lymphoma
Denovo Biopharma LLC
Treatment of glioblastoma multiforme
Denovo Biopharma LLC
Treatment of stage IIB-IV melanoma.
Epidermal growth factor (human)
For acceleration of corneal epithelial regeneration and the healing of stromal tissue in the condition of non-healing corneal defects.
Treatment of myelodysplastic syndrome.
Johnson & Johnson Pharmaceutical Research & Dev.,
Treatment of anemia associated with end stage renal disease.
R. W. Johnson Pharmaceutical Research Institute
Treatment of HIV associated anemia related to HIV infection or HIV treatment.
R. W. Johnson Pharmaceutical Research Institute
Treatment of anemia associated with end stage renal disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 132 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1073epratuzumab n/a 9/30/2008 Immunomedics, Inc.
1074epratuzumab Lymphocide 7/13/1998 Immunomedics, Inc.
1075Eprodisate Kiacta(Tm) 4/6/1999
1076
n/a 6/12/2014 FFCanVac Pte Ltd1077
eptifibatide and iloprost n/a 4/20/2012 Treatment of purpura fulminans Thrombologic1078
erdosteine n/a 12/20/2013 Treatment of bronchiectasis.1079
Erlotinib Hydrochloride Tarceva 7/18/2003 Treatment of malignant gliomas Genentech, Inc1080
n/a 8/19/19871081
estradiol Gel n/a 10/31/20061082
etarfolatide n/a 2/16/2000 Endocyte, Inc.
Treatment of acute lymphoblastic leukemia
Treatment of non-Hodgkin's lymphoma
Treatment of secondary amyloidosis
C. T. Development America, Inc.
Epstein-Barr virus-specific autologous cytotoxic T lymphocytes
Treatment of Epstein-Barr virus positive head and neck cancers (which includes nasopharyngeal carcinoma)
Alitair Pharmaceuticals, Inc.
Erythropoietin (recombinant human)
Treatment of anemia associated with end stage renal disease.
McDonnell Douglas Corp
Estrogen replacement therapy in females with Turner syndrome
Ascend Therapeutics US, LLC
For the identification of ovarian carcinomas
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 133 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1083
ethanolamine Ethamolin 1/17/2014 QOL Medical, LLC1084
Ethyl eicosapentaenoate n/a 4/6/2000 Laxdale Ltd.1085
Etiocholanedione n/a 11/3/1995 Treatment of aplastic anemia. SuperGen, Inc.1086
Etiocholanedione n/a 5/7/1996 SuperGen, Inc.1087
etirinotecan pegol n/a 4/18/2011 Treatment of ovarian cancer. Nektar Therapeutics1088
etrolizumab n/a 8/22/2016 Genentech, Inc.1089
everolimus Afinitor 7/23/20121090
everolimus n/a 6/2/20141091
everolimus ointment n/a 9/10/20151092
evinacumab n/a 2/8/2016
Prophylactic use in pediatric patients (age 0 through 16 years) with esophageal varices that are at risk of bleeding to obliterate varices and to prevent bleeding
Treatment of Huntington's disease.
Treatment of Prader-Willi syndrome.
Treatment of pediatric patients with ulcerative colitis
Treatment of hepatocellular carcinoma
Novartis Pharmaceuticals Corporation
Treatment of diffuse large B-cell lymphoma
Novartis Pharmaceuticals Corp.
Topical treatment of Tuberous Sclerosis Complex-related skin lesions
Aucta Pharmaceuticals, LLC
Treat of homozygous familial hypercholesterolemia.
Regeneron Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 134 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1093
Prochymal(R) 12/14/2005 Mesoblast, Inc.1094
n/a 5/8/2014 iCell Science AB1095
Prochymal(R) 4/30/2010 Mesoblast, Inc.1096
n/a 12/20/20101097
exendin-(9-39) n/a 6/1/20111098
Exisulind n/a 2/14/19941099
n/a 11/5/2009
Ex vivo cultured adult human mesenchymal stem cells
Treatment of acute graft versus host disease
ex vivo cultured human mesenchymal stromal cells
Prevention of graft rejection following solid organ transplantation
ex-vivo cultered adult human mesenchymal stem cells
Treatment of Type 1 diabetes patients with residual beta cell function
ex-vivo expanded autologous bone marrow-derived mesenchymal stem cells
Treatment of Amyotrophic Lateral Sclerosis
TCA Cellular Therapy, LLC
Treatment of congential hyperinsulinenic hypoglycemia and other causes of hyperinsulinemic hypoglycemia in adults and children
The Children's Hospital of Philadelphia
For the suppression and control of colonic adenomatous polyps in the inherited disease adenomatous polyposis coli.
OSI Pharmaceuticals, Inc.
Exon 44 specific phosphorothioate oligonucleotide
Treatment of Duchenne Muscular Dystrophy in patients who have a mutation correctable by skipping of exon 44of the dystrophin gene.
BioMarin Pharmaceutical, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 135 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1100
n/a 1/23/20131101
n/a 8/25/20091102
n/a 1/23/20131103
n/a 1/23/20131104
n/a 1/23/20131105
n/a 8/20/2009 Intercytex Ltd.1106
n/a 8/22/2013 ReNeuron Ltd
Exon 45 specific phosphorothioate oligonucleotide
Treatment of Duchenne Muscular Dystrophy patients bearing mutations that can be corrected by skipping exon 45
BioMarin Pharmaceutical, Inc.
Exon 51 specific phosphorothioate oligonucleotide
Treatment of Duchenne Muscular Dystrophy.
BioMarin Pharmaceutical, Inc.
Exon 52 specific phosphorothiate oligonucleotide
Treatment of Duchenne Muscular Dystrophy patients bearing mutations that can be corrected by skipping exon 52
BioMarin Pharmaceutical, Inc.
exon 53 specific phosphorothioate oligonucleotide
treatment of Duchenne Muscular Dystrophy patients bearing mutations that can be corrected by skipping exon 53
BioMarin Pharmaceutical, Inc.
Exon 55 specific phosphorothioate oligonucleotide
Treatment of Duchenne Muscular Dystrophy patients bearing mutations that can be corrected by skipping exon 55
BioMarin Pharmaceutical, Inc.
expanded allogeneic human dermal fibroblasts in hypothermosol(r)-FRS
Treatment of Dystrophic Epidermolysis Bullosa.
expanded human allogeneic neural retinal progenitor cells extracted from neural retina
Treatment of retinitis pigmentosa
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 136 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1107
n/a 5/14/2012 Treatment of multiple myeloma1108
n/a 11/19/2012 Treatment of sickle cell disease Invenux, LLC1109
ezatiostat hydrochloride Telintra 1/9/2013 Telik, Inc.1110
n/a 3/8/19951111
n/a 1/10/2014 Treatment of hemophilia A Genentech1112
n/a 5/21/20031113
Fampridine Neurelan 6/2/19971114
n/a 5/2/20161115 farletuzumab n/a 6/16/2006 Treatment of ovarian cancer Morphotek, Inc.1116
fecal microbiota n/a 3/10/2014 Rebiotx, Inc.1117
fenfluramine HCI Brabafen 12/20/2013 Zogenix, Inc.
Extract of sea cucumber, sea sponge, shark fin, sea urchin, and sargassum grass
Unicorn Pacific Corporation
extract of sorghum bicolor extract
Treatment of myelodysplastic syndrome
Facilitated DNA Plasmid Vaccine
Treatment of cutaneous T cell lymphoma.
Wyeth-Lederle Vaccines and Pediatrics
factor VIII mimetic bispecific antibody
Factor XIII [A2] homodimer, recombinant DNA origin
Treatment of congenital FXIII deficiency
Novo Nordisk Pharmaceuticals, INc.
Treatment of chronic, incomplete spinal cord injury.
Acorda Therapeutics, Inc.
Fancalen (lentiviral vector containing the Fanconi anemia-A (FANCA) gene
Treatment of Fanconi anemia type A patients.
Centro de Investigacion Biomedica en Red (CIBER)
Treatment of recurrent Clostridium difficile infection (Clostridium difficile gastrointestinal disease)
For the Treatment of Dravet Syndrome
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 137 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1118Fenobam hydrochloride n/a 11/20/2006 Treatment of fragile X syndrome Neuropharm, Ltd.
1119fenretinide n/a 9/4/2013 CerRx, Inc.
1120fenretinide n/a 2/1/2007 Cancer Research UK
1121fenretinide n/a 9/4/2013 CerRx, Inc.
1122
Fenretinide n/a 4/7/2010 McGill University1123
Fenretinide n/a 10/5/2005 Treatment of neuroblastoma1124
n/a 6/26/2003 Degussa AG1125
ferumoxytol n/a 4/6/2012 ArsNova Partners, LLC1126
ferumoxytol Feraheme 4/29/2011
Treatment of peripheral T-cell lymphoma
Treatment of Ewing's sarcoma family of tumors.
Treatment of cutaneous T-cell lymphoma
Treatment of Pseudomonas aeruginosa lung infections in cystic fibrosis patients
USC-CHLA Institute for Pediatric Clinical Research
ferric hexacyanoferrate (II) "Prussian Blue"
Treatment of patients with known or suspected internal contamination with radioactive or non-radioactive cesium or thallium
For use in magnetic resonance imaging to assess, and monitor treatment of solid tumor malignancies previously diagnosed in pediatric patients (age 16 years and younger)
For use in MR imaging for the mangement of brain tumors
Edward A. Neuwelt, MD
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 138 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1127
ferumoxytol n/a 10/7/20111128
fexinidazole n/a 4/4/20161129
fialuridine n/a 7/24/19921130
n/a 2/1/2007 Kuros Biosurgery AG1131
Fibrinogen (human) n/a 8/23/19951132
n/a 9/5/1988 Melville Biologics, Inc.1133
Fibronectin Peptide n/a 6/29/2011
For use in magnetic resonance imaging in brain metastases
Oregon Health & Science University
Treatment of human African trypanosomiasis (HAT) or sleeping sickness
Sanofi US Services, Inc.
Adjunctive treatment of chronic active hepatitis B.
Oclassen Pharmaceuticals, Inc.
Fibrin-based agent containing a N-terminally modified parathyroid hormone fragment TGplPTH1-34
Treatment of solitary (unicameral) bone cysts
For the control of bleeding and prophylactic treatment of patients deficient in fibrinogen.
Alpha Therapeutic Corporation
Fibronectin (human plasma derived)
Treatment of non-healing corneal ulcers or epithelial defects which have been unresponsive to conventional therapy and the underlying cause has been eliminated.
Prevention of burn injury progression of acute, deep dermal burns in hospitalized patients.
NeoMatrix Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 139 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1134
fidaxomicin Dificid 12/13/20101135
filanesib n/a 5/6/2014 Treatment of multiple myeloma. Array BioPharma, Inc.1136
filgrastim n/a 1/27/20151137
filociclovir n/a 11/29/2010 Microbiotix, Inc.1138
fingolimod n/a 4/30/20101139
firtecan pegol n/a 4/18/2011 Treatment of neuroblastoma.1140
n/a 10/19/20161141
n/a 9/19/2016 Treatment of narcolepsy Theranexus S.A1142
n/a 11/23/20101143
florilglutamic acid n/a 5/11/2016 Piramal Imaging S.A.1144
Floxuridine, FUDR n/a 12/22/2004 Franco Muggia, M.D.
Treatment of pediatric Clostridium difficile infection
Optimer Pharmaceuticals, Inc.
Treatment of amyotrophic lateral sclerosis (ALS)
Neurovision Pharma GmbH
Treatment of active cytomegalovirus infections
Treatment of chronic inflammatory demyelinating polyneuropathy
Novartis Pharmaceutical Corporation
Enzon Pharmaceuticals, Inc.
fixed dose combination formulation of macitentan and tadalafil
Treatment of pulmonary arterial hypertension
Actelion Clinical Research, Inc.
fixed dose combination of modafinil and flecainide
FliC Flagellin Deletion Variant TLR5 Agonist
Prevention of death following a potentially lethal dose of total body irradiation during or after a radiation disaster
Cleveland BioLabs, Inc.
Diagnostic for the management of glioma
Intraperitoneal treatment of gastric cancer.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 140 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1145
flubendazole n/a 1/23/20141146
flubendazole n/a 10/25/20131147
n/a 4/7/2015 For the diagnosis of glioma1148
Fludarabine phosphate Fludara 4/18/19891149
flunarizine Sibelium 1/6/19861150
flunarizine n/a 6/24/20131151
flunarizine n/a 7/20/20161152
Fluorouracil n/a 4/18/1990
Treatment of lymphatic filariasis caused by nematodes of the family Filariodidea, in children and adults.
Janssen Research and Development, LLC
Treatment of onchocerciasis caused by Onchocerca volvulus
Janssen Research & Development, LLC
fluciclovine (18F)(anti-1-amino-3-fluorocyclobutane-1-carboxylic acid, labelled with Fluorine-18)
Blue Earth Diagnostics Ltd.
Treatment and management of patients with non-Hodgkins lymphoma.
Berlex Laboratories, Inc.
Treatment of alternating hemiplegia.
Janssen Research Foundation
Treatment of alternating hemiplegia
Marathon Pharmaceuticals, LLC
Treatment of hemiplegic migraine
Xenon Pharmaceuticals, Inc.
For use in combination with interferon alpha-2a, recombinant, for the treatment of advanced colorectal carcinoma.
Hoffmann-La Roche, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 141 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1153
Fluorouracil n/a 10/27/19891154
Fluorouracil n/a 6/29/2000 Ethypharm SA1155
Fluoxetine Prozac 4/14/2004 Hollander, Eric MD1156 Fluoxetine Prozac 4/30/1999 Treatment of autism. Neuropharm, Ltd.1157
fluticasone propionate n/a 1/19/20111158
fluticasone propionate n/a 12/29/20151159
n/a 6/8/20161160
n/a 6/8/20161161
fosbretabulin tromethamine n/a 12/29/20151162
fosbretabulin tromethamine n/a 6/8/2016 Treatment of glioma1163
fosfomycin/tobramycin n/a 11/28/2008
For use in combination with interferon alpha-2a, recombinant, for the treatment of esophageal carcinoma.
Hoffmann-La Roche, Inc.
Treatment of glioblastoma multiforme.
Treatment of body dysmorphic disorder in children and adolescents
Treatment of pediatric and adult eosinophilic esophagitis
Adare Pharmaceuticals, Inc.
Treatment of eosinophilic esophagitis
Banner Life Sciences, LLC
folate binding protein peptide: Glu-Ile-Trp-Thr-His-Ser-Tyr-Lys-Val
Treatment (including prevention of recurrence) of ovarian cancer
Galena Biopharma, Inc.
Folate Binding Protein Peptide: Glu-Ile-Trp-Thr-Phe-Ser-Thr-Lys-Val
Treatment (including prevention of recurrence) of ovarian cancer
Galena Biopharma, Inc.
Treatment of neuroendocrine tumors
Mateon Therapeutics, Inc.
Mateon Therapeutics, Inc.
Treatment of pulmonary infections associated with cystic fibrosis
CURx Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 142 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1164
fostamatinib disodium n/a 8/25/20151165
FTV1+GDP-fucose n/a 3/18/20111166
n/a 9/22/2008 Treatment of Ewing's sarcoma1167
Ni-0501 3/26/2010 NovImmune S.A.1168
n/a 6/9/2015 XOMA (US) LLC1169
n/a 10/16/2013 Versartis, Inc.1170
n/a 1/11/20061171
G17DT Immunogen Gastrimmune(Tm) 7/10/2002 Cancer Advances, Inc.
Treatment of immune thrombocytopenic purpura
Rigel Pharmaceuticals, INc.
To improve homing to bone (treatment of myeloablation) in patients receiving hematopoietic stem cell transplantation.
America Stem Cell, Inc.
Full Phosphorothioate Antisense Oligonucleotide against EWS-Fli-1 nanoparticles
The Cure Our Children Foundation
Fully human anti-interferon gamma monoclonal antibody
Treatment of hemophagocytic lymphohistiocytosis
fully human IgG2 monoclonal antibody that binds insulin receptors
Treatment of congenital hyperinsulinism
fusion protein analog with recombinant human growth hormone (rhGH) at once-a-month dosing
Treatment of growth hormone deficiency
Fusion protein consisting of human immunoglobim G1 constant region Fc region fused to the human receptor binding domain of ectodysplasin-A1
Treatment of X-linked hypohidrotic ectodermal dysplasia
Edimer Pharmaceuticals, Inc.
Treatment of adenocarcinoma of the pancreas
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 143 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1172G17DT Immunogen Gastrimmune(Tm) 7/18/2002 Treatment of gastric cancer Cancer Advances, Inc.
1173gabapentin Neurontin 7/5/1995
1174
Gallium (68Ga) edotreotide Somakit Toc 7/1/20151175
n/a 10/25/20131176
Gamma hydroxybutyrate n/a 12/3/19871177
Gammalinolenic acid n/a 7/27/1994 Zurier, Robert B. M.D.1178
ganaxolone n/a 3/24/20151179
ganaxolone n/a 4/14/2016 Treatment of status epilepticus.1180
Ganaxolone n/a 5/25/1994 Treatment of infantile spasms.1181
ganitumab n/a 11/23/2010 Treatment of pancreatic cancer. Nantcell1182
n/a 7/7/2009 Treatment of pancreatic cancer Astrimmune Ltd.
Treatment of amyotrophic lateral sclerosis
Warner-Lambert Company
Diagnostic for the clinical management of neuroendocrine tumors.
Advanced Accelerator Applications, USA
Gallium-68 (DOTA0-Phel-Tyr3)octreotide
The management of neuroendocrine tumors
University of Iowa Hospital & Clinics
Treatment of narcolepsy and the auxiliary symptoms of cataplexy, sleep paralysis, hypnagogic hallucinations and automatic behavior.
Biocraft Laboratories, Inc.
Treatment of juvenile rheumatoid arthritis.
Treatment of Protocadherin 19 (PCDH19) female epilepsy.
Marinus Pharmaceuticals, Inc.
Marinus Pharmaceuticals, Inc.
Marinus Pharmaceuticals, Inc.
Gastrin 17C Diphtheria Toxoid Immunogen
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 144 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1183
gemcabene n/a 2/6/20141184
gemcitabine n/a 12/30/2015 TARIS Biomedical LLC1185
gemcitabine n/a 1/11/2016 InnoPharmax, Inc.1186
gemcitabine ready-to-use n/a 6/24/2015 Treatment of pancreatic cancer1187
gemcitabine ready-to-use n/a 6/24/2015 Treatment of ovarian cancer1188
n/a 2/4/2009 Memgen, LLC1189
n/a 4/29/2002 Treatment of malignant glioma Aettis, Inc.1190
n/a 12/11/1997 Treatment of Crohn's disease.1191
Geneticin n/a 6/6/2005 Treatment of amoebiasis.1192
gentamicin C2 congener n/a 3/29/2016 Treatment of cystic fibrosis
Treatment of homozygous familial hypercholesterolemia
Gemphire Therapeutics, Inc.
Treatment of upper tract urothelial carcinoma
Treatment of cholangiocarcinoma.
Sun Pharmaceutical Industries Ltd.
Sun Pharmaceutical Industries Ltd.
gene encoding chimeric CD40 ligand
Treatment of chronic lymphocytic leukemia
genetically engineered herpes simplex virus (G207)
Genetically engineered human recombinant IgG4 monoclonal antibody directed against human TNF alpha
Celltech Chiroscience Limited
ProcesScience, Inc (PSI)
La Jolla Pharmaceuticals Company
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 145 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1193
Septopal 1/31/19911194
Gentamicin liposome injection Maitec 7/10/19901195
gevokizumab n/a 7/27/2010 Treatment of Behcet's disease XOMA (US) LLC1196
gevokizumab n/a 2/21/2014 XOMA (US) LLC1197
gevokizumab n/a 8/20/2012 XOMA (US) LLC1198
Girentuximab Rencarex 3/22/20011199
givinostat n/a 4/12/2013 Italfarmaco SpA1200
glatiramer acetate Copaxone 11/14/20071201
Copaxone 6/5/20011202
n/a 6/8/2016 AMMTeK
Gentamicin impregnated PMMA beads on surgical wire
Treatment of chronic osteomyelitis of post-traumatic, postoperative, or hematogenous origin.
Lipha Pharmaceuticals, Inc.
Treatment of disseminated Mycobacterium avium-intracellulare infection.
Liposome Company, Inc.
Treatment of pyoderma gangrenosum
Treatment of non-infectious intermediate, posterior or pan uveitis, or chronic non-infectious anterior uveitis
Treatment of renal cell carcinoma.
Wilex Biotechnology GmbH
Treatment of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
Treatment of amyotrophic lateral sclerosis (ALS).
Teva Neurosciences, Inc.
Glatiramer acetate for injection
Treatment of primary-progressive multiple sclerosis
Teva Pharmaceuticals USA
glibentek (glibenclamide oral suspension)
Treatment of neonatal diabetes (monogenic forms of diabetes that present in infancy)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 146 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1203n/a 1/12/2011 Treatment of glioma.
1204
glucagon n/a 12/5/2012 Biodel, Inc.1205
glucagon infusion G-Pump (Tm) 9/25/20141206
glufosfamide n/a 9/18/20061207
n/a 8/16/2010 Treatment of glioma1208
glyburide n/a 9/29/20151209
glyburide n/a 2/8/20161210
glycafilin n/a 8/22/20081211
Glyceryl tri (4-phenylbutyrate) n/a 5/24/2005 Ucyclyd Pharma, Inc1212
n/a 2/14/1995 Moser, Hugo W. M.D.1213
glycopyrrolate 2% n/a 6/17/2010 Treatment of Frey's syndrome
glioma derived cell lysates and irradiated cells
Epitopoietic Research Corp.
Prevention of hypoglycemia in the congenital hyperinsulinism population
Prevention of chronic, severe hypoglycemia related to congential hyperinsulinism
Xeris Pharmaceuticals, Inc.
For treatment of pancreatic cancer.
Eleison Pharmaceuticals LLC
Glutathione pegylated liposomal doxorubicin hydrochloride
to-BBB technologies BV
Treatment of acute spinal cord injury
Remedy Pharmaceuticals, Inc.
Treatment of acute subarachnoid hemorrhage
Remedy Pharmaceuticals, Inc.
Prevention of delayed graft function after solid organ transplantation
ProtAffin Biotechnologie AG
Treatment of spinal muscular atrophy
Glyceryl trioleate and glyceryl trierucate
Treatment of adrenoleukodystrophy.
Wellesley Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 147 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1214
n/a 9/27/2012 Cytheris, Inc.1215
n/a 10/23/2013 Treatment of Fragile X Syndrome1216
n/a 2/11/2015 Treatment of Rett syndrome1217
Proenzy 4/30/20101218
GNE Lipoplex n/a 11/13/2008 Gradalis, Inc.1219
GNE plasmid(H001) n/a 3/26/2010 HIBM Research Group1220
golimumab Simponi 3/16/2012 Janssen Biotech, Inc.1221
golimumab Simponi Aria 4/2/20151222
golnerminogene pradenovec Tnferade(Tm) Biologic 10/28/2009 Treatment of pancreatic cancer. GenVec, Inc.1223
Gossypol n/a 10/22/19901224
n/a 3/25/1997 Genzyme Corporation1225
n/a 8/27/2008 Treatment of cystic fibrosis Savara Inc.
glycosylated recombinant human interleukin-7
Treatment of progressive multifocal leukoencephalopathy
glycyl-L-2-methylprolyl-L-glutamic Acid
Neuren Pharmaceuticals, Ltd.
glycyl-L-2-methylpropyl-L-glutamic acid
Neuren Pharmaceuticals, Ltd.
Glyeraldehyde-3-phosphate dehydrogenase
Treatment of pediatric multiple sclerosis
BPT Pharmaceuticals, Inc.
Treatment of hereditary inclusion body myopathy-2
Treatment of hereditary inclusion body myopathy type 2
Treatment of pediatric ulcerative colitis
Treatment of polyarticular juvenile idiopathic arthritis in patients 0 through 18 years of age.
Janssen Research & Development, LLC
Treatment of cancer of the adrenal cortex.
Reidenberg, Marcus M. M.D.
Gp100 adenoviral gene therapy
Treatment of metastatic melanoma.
granulocyte macrophage colony stimuling factor
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 148 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1226
n/a 7/24/2013 Treatment of soft tissue sarcoma Oncos Therapeutics1227
n/a 8/7/19891228
n/a 2/23/20161229
guadecitabine n/a 9/29/20151230
guanfacine Tenex 8/5/1999 Treatment of fragile X syndrome.1231
guanfacine and amphetamine n/a 3/17/2016 Genco Sciences, LLC1232
gusperimus trihydrochloride n/a 6/29/2011 Nordic Group B.V.1233
GVAX melanoma n/a 12/23/2010 Aduro BioTech, Inc.1234
gylceryl tri (4-pheynlybutyrate) n/a 9/3/2009
granulocyte-macrophage colony stimulating factor-coding oncolytic adenovirus, Ad5/3-D24-GMCSF
Growth hormone releasing factor
For the long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion.
Valeant Pharmaceuticals North America
growth hormone releasing hormone, 1-44 human amide
Treatment of growth hormone deficiency
Hollenbeck Pharmaceuticals, Inc.
Treatment of acute myeloid leukemia.
Astex Pharmaceuticals, Inc.
Watson Laboratories, Inc.
Treatment of pediatric patients with Tourette's syndrome (0 through 16 years of age)
Treatment of Wegener's granulomatosis
Treatment of stage IIb to IV melanoma
For intermittent or chronic treatment of patients with cirrhosis and any grade hepatic encephalopathy.
Horizon Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 149 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1235
n/a 11/3/2016 Treatment of acromegaly Amryt Research Ltd.1236
n/a 12/22/20111237 H-Leu-Pro-Pro-Ser-Arg-OH n/a 9/20/2012 Treatment of Kaposi sarcoma Immuno Tech, Inc.1238
n/a 5/14/2013 NoNO, Inc.1239
n/a 4/11/2014 NoNO, Inc.1240
Halofuginone Stenorol 2/7/2000 Treatment of systemic sclerosis.1241
halofuginone hydrobromide n/a 10/13/20111242
Hanferon Hanferon 3/9/2012 Treatment of Behcet's disease
H-(4-amino-3-iodo)-D-Phe-c[Cys-(3-iodo)-Tyr-D-Trp-Lys-Val-Cys]-Thr-NH2
H-(D)p-Benzoylphenylalanyl-(D)seryl-(D)tryptophanyl-(D)seryl-(D)pentafluorophenylalanyl-(D)cyclohexylalanyl-(D)arginyl-(D)arginyl-(D)arginyl-(D)glutaminyl-(D)arginyl-(D)arginine acetate salt
For use in combination with cisplatin and pemetrexed for the treatment of patients with mesothelioma
CanBas Company, Ltd.
H-Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-alys-aleu-Ser-Ser-Ile-Glu-Ser-Asp-Val-OH
Treatment of subarachnoid hemorrhage
H-Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Lys-Leu-Ser-Ser-Ile-Glu- Ser-Asp-Val-OH
Treatment of acute ischemic stroke patients presenting within 3 hours of symptom onset
Collgard Biopharmaceuticals Ltd.
Treatment of Duchenne Muscular Dystrophy
Halo Theraeputics, LLC
HanAll BioPharma Co., Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 150 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1243
n/a 11/13/20121244
n/a 4/19/20161245
Cadi Mw 11/21/2002 CPL, Inc.1246
Cadi Mw 9/3/2004 Active tuberculosis1247
Cadi-Mw 7/31/20121248
n/a 9/23/2014 Treatment of pancreatic cancer1249
Heat Shock Protein 70 n/a 3/18/2011 ALS Biopharma, LLC1250
n/a 8/27/2014
Hantaan virus and Puumala virus DNA vaccines
Prevention of hemorrhagic fever with renal syndrome caused by Hantaan virus and Puumala virus.
Surgeon General of the U. S. Army
HDF-LV-RTS-MMP1 (human dermal fibroblasts genetically modified with LV-RTS-MMP1) and Veledimex
Treatment of localized scleroderma.
Fibrocell Technologies, Inc.
heat killed mycobacterium w immunomodulator
Adjuvant to multi-drug therapy in the management of multibacillary leprosy
heat killed Mycobacterium w immunomodulator
Cadila Pharmaceuticals Limited, Inc.
heat killed mycobacterium w immunomodulator
Treatment of non-small cell lung cancers that express desmocollin-3
Cadila Pharmaceuticals Limited
heat killed whole cell mycobacterium obuense
Immodulon Therapeutics Limited
Treatment of amyotrophic lateral sclerosis
hematopoietic stem and progenitor cells expanded ex-vivo with a low molecular weight aryl hydrocarbon receptor (AHR) antagonist
Hematopoietic support in patients with acute lymphoblastic leukemia (ALL)
Novartis Pharmaceuticals Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 151 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1251
Ladilen 11/9/20161252
Heme arginate Normosang 3/1/1994 Orphan Europe1253
Heme arginate Normosang 3/10/1988 Orphan Europe SARL1254
Hemex 12/20/19931255
n/a 12/18/2007 Treatment of cardiogenic shock Apex Bioscience, Inc.1256
heparan sulfate mimetic n/a 5/29/2014 Treatment of pancreatic cancer1257
n/a 10/24/20111258
Heparin sodium n/a 6/29/2006 Treatment of cystic fibrosis1259
n/a 9/18/2006
hematopoietic stem cells modified with a lentiviral vector containing the CD18 (Integrin Beta 2) gene
Treatment of leukocyte adhesion deficiency Type I
Centro de Investigacion Biomedica en Red (CIBER)
Treatment of myelodysplastic syndromes.
Treatment of symptomatic stage of acute porphyria.
Hemin and zinc mesoporphyrin
Treatment of acute porphyric syndromes.
Bonkovsky, Herbert L. M.D.
Hemoximer (pyridoxalated hemoglobin polyoxyethylene)
Momenta Pharmaceuticals, Inc.
heparin activated recombinant human fibroblast growth factor 1 (FGF1)in combination with a surgically implanted biodegradable device
Treatment of patients with a confirmed traumatic complete spinal cord injury where no motor or sensory function is preserved below the injury(Scale A)
BioArctic Neuroscience AB
Ockham Biotech Limited
Heparin-binding epidermal growth factor-like growth factor
Prevention and treatment of necrotizing enterocolitis (NEC) in preterm infants with birth weight less than 1,500 grams
Trillium Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 152 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1260
Nabi-Hb 3/8/19951261
Hepabig Gene 5/6/2013 Green Cross Corp.1262
Civacir(Tm) 11/14/20021263
hepcortespenlisimut-L n/a 12/17/2014 Immunitor, Inc.1264
n/a 12/23/2014 Treatment of pancreatic cancer Karcinolys S.A.S.1265
Herpes simplex virus gene n/a 10/16/1992 Genetic Therapy, Inc.1266
n/a 1/13/20121267
n/a 3/9/20121268
hexasodium phytate n/a 12/2/2012 Treatment of calciphylaxis1269
n/a 6/27/2016 Treatment of systemic sclerosis iBio, Inc.1270
HIRMAb-IDS n/a 5/15/2013
Hepatitis B immune globulin intravenous (human)
Prophylaxis against hepatitis B virus reinfection in liver transplant patients.
Biotest Pharmaceuticals Corporation
hepatitis B virus neutralizing human monoclonal antibody
Prevention of hepatitis B recurrence following liver transplantation
Hepatitis C virus immune globulin (human)
Prophylaxis of hepatitis C infection in liver transplant recipients.
Biotest Pharmaceuticals Corporation
Treatment of hepatocellular carcinoma
herpes simplex type 1 virus containing cellular B-myb gene as tumor-specific promoter
Treatment of primary and metastatic brain tumors.
heterologous human adult liver derived progenitor cells (HHALPC)
Treatment of urea cycle disorders
Promethera Biosciences
heterologous human liver derived progenitor cells
Treatment of Crigler-Najjar syndrome
Promethera Biosciences
Laboratoris Sanifit, S.L.
high molecular weight multimer of E3-Fc, an endostatin C-terminus and IgG1-Fc fusion protein
Treatment of mucopolysaccharidosis Type II (Hunter Syndrome)
ArmaGen Technologies, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 153 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1271n/a 1/13/2016 Treatment of retinal detachment ONL Therapeutics, Inc
1272n/a 3/11/2013 Treatment of retinal detachment ONL Therapeutics, Inc
1273
Histamine Maxamine 2/1/2000 EpiCept Corporation1274
Histamine Ceplene 12/15/1999 EpiCept Corporation1275
Histrelin n/a 5/3/19911276
n/a 1/21/2016 Treatment of uveal melanoma. Immunocore Ltd1277
Allovectin-7 9/30/1999 Vical Incorporated1278
homoharringtonine n/a 2/8/20021279
n/a 1/12/2009 Gliknik, Inc.1280
n/a 9/1/2011 ISA Therapeutics BV
His-His- Ile-Tyr-Leu-Gly-Ala-Val-Asn-Tyr-Ile-Tyr-NH2
His-His-Ile-Tyr-Leu-Gly-Ala-Val-Asn-Tyr-Ile-Tyr
For use as an adjunct to cytokine therapy in the treatment of malignant melanoma.
Adjunct to cytokine therapy in the treatment of acute myeloid leukemia.
Treatment of acute intermittent porphyria, hereditary coproporphyria, and variegate porphyria.
Anderson, Karl E., M.D.
HLA-A2-selective gp100-specific T cell redirector
HLA-B7/Beta2M DNA Lipid (DMRIE/DOPE) Complex
Treatment of invasive and metastatic melanoma (Stages II, III, and IV).
Treatment for chronic myelogenous leukemia
American BioScience, Inc.
HPV-16 cancer therapeutic trojan peptide vaccine
Treatment of HPV-16 expressing head and neck squamous cell carcinoma.
HPV16 E6/E7 synthetic long peptides vaccine
Treatment of epithelial neoplasias of the vulva positive for human papilloma virus type 16.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 154 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1281
Remitogen 11/28/2001 PDL BioPharma, Inc.1282
n/a 11/9/2015 Bone Therapeutics SA1283
Allob 1/10/2014 Treatment of osteonecrosis Bone Therapeutics SA1284
n/a 9/12/2008 Mesoblast, Inc.1285
n/a 4/4/20051286
n/a 9/27/2004 Treatment of Hodgkin's disease1287
Humax-Cd4 8/13/2004 Treatment of mycosis fungoides1288
n/a 7/29/2008 Treatment of multiple myeloma
Hu1D10, humanized monoclonal antibody
For use in the treatment of 1D10+ B cell non-Hodgkin's lymphoma
human allogeneic bone marrow derived osteoblastic cells
Treatment of osteogenesis imperfecta.
human allogeneic bone marrow derived osteoblastic cells
Human Allogeneic HLA-matched, Umbilical Cord Blood Hematopoietic Progenitor Cells Ex Vivo Expanded on Allogeneic Mesenchymal Precursor Cells
Treatment of insufficient hematopoietic stem cell production in patients with hematologic malignancies who have failed treatment with conventional chemotherapy.
Human alpha 2,6 sialyltransferase adenoviral gene therapy
Treatment of patients with invasive (malignant) brain and central nervous system tumors lacking alpha 2,6 sialyltransferase.
Falk Center for Molecular Therapeutics
Human anti-CD30 monoclonal antibody
Bristol-Myers Squibb, Inc.
human anti-CD4 monoclonal antibody
Emergent Product Development Seattle, LLC
human anti-cellular adhesion molecule-1 monoclonal antibody
BioInvent International AB
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 155 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1289
n/a 3/24/2008 Treatment of osteonecrosis1290
human coagulation factor VIII Octanate 12/3/20121291
human coagulation factor XI Hemoleven 11/8/20071292
n/a 11/3/2015 Shire1293
n/a 12/20/20061294
n/a 6/2/20161295
n/a 6/19/2014
Human autologous bone-forming cell derived from bone marrow stem cells
Bone Therapeutics S.A.
Immune tolerance induction in hemophilia A patients with inhibitors
OCTAPHARMA USA, Inc.
Treatment of severe congential Factor XI deficiency.
Laboratoire francais du Fractionnement et des Biot
human cystic fibrosis transmembrane conductance regulator (hCFTR) messenger RNA (mRNA) complexed with branched polyethyleneimine (bPEI)
Treatment of patients with cystic fibrosis.
Human cytomegalovirus immunoglobulin
Prevention of congenital cytomegalovirus (CMV) infection following primary CMV infection in pregnant women
Biotest Pharmaceuticals Corporation
human donor hematopoietic stem and progenitor cells (HSPC) that have been treated ex vivo with TBX-4000
Enhancement of cell engraftment in patients receiving hematopoietic stem cell transplant.
Taiga Biotechnologies, Inc.
human fully IgG1 antibody specific for CD33
Treatment of acute myeloid leukemia
Boehringer Ingelheim Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 156 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1296human gammaglobulin Oralgam 5/25/2001
1297human gammaglobulin Oralgam 11/14/2003
1298
human gammaglobulin Oralgam 9/16/20021299
Q-Cells 9/11/2013 Q Therapeutics, Inc.1300
human haptoglobin n/a 11/19/2013 Treatment of sickle cell disease1301
Human Hemin n/a 8/6/20131302
n/a 5/11/2011 Treatment of acute liver failure Alfact Innovation SAS1303
human heterologous liver cells n/a 2/14/20111304
n/a 10/16/2013 Treatment of systemic sclerosis MedImmune1305
Human immune globulin Flebogamma(R) 5% Dif 3/29/2006 Instituto Grifols, S.A.
Treatment for juvenile rheumatoid arthritis
Latona Life Sciences, Inc.
Treatment of idiopathic inflammatory myopathies
Latona Life Sciences, Inc.
Treatment of gastrointestinal disturbances (to include constipation, diarrhea, and abdominal pain) associated with regression-onset autism in pediatric patients.
Latona Life Sciences, Inc.
human glial restricted progenitor cells and their progeny
Treatment of amyotrophic lateral sclerosis
BioProducts Laboratory Limited
Prevention of ischemia reperfusion injury in patients undergoing solid organ transplantation
Borders Technology Management Ltd
human Hepatocarcinoma-Intestine-pancreas/pancreatitis associated protein
Treatment of urea cycle disorders
Promethera Biosciences S.A./N.V.
human IgG1k monoclonal antibody
Treatment of post-polio syndrome
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 157 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1306n/a 1/10/2006 Bristol-Myers Squibb
1307human insulin (rDNA) n/a 4/6/2015 Nutrinia, Ltd.
1308
n/a 2/11/2013 Orban Biotech, LLC1309
n/a 6/6/20131310
n/a 2/18/20111311
human laminin-111 n/a 9/23/2011 Prothelia, Inc.1312
n/a 1/26/2015 Treatment of glioma.1313
n/a 7/7/2005 IRX Therapeutics, Inc.1314
n/a 7/6/2012 Treatment of ovarian cancer Galileo Research srl1315
n/a 5/29/2014 AbbVie, Inc.
Human immunoglobin anti-CD30 monoclonal antibody
Treatment of CD30+ T-cell lymphoma
Treatment of short bowel syndrome (SBS).
human insulin beta chain peptide with incomplete Freund's adjuvant vaccine
Treatment of Type 1 diabetes patients with residual beta cell function
human interleukin-3 genetically conjugated to diphtheria toxin protein
Treatment of blastic plasmacytoid dendritic cell neoplasm
Stemline Therapeutics, Inc.
human interleukin-3 genetically conjugated to diphtheria toxin protein
Treatment of acute myeloid leukemia.
Stemline Therapeutics, Inc.
Treatment of merosin (laminin-alpha2) deficient congential muscular dystrophy type 1A.
human leukocyte antigen-A2 restricted peptides
Stemline Therapeutics, Inc.
human leukocyte-derived cytokine mixture
Neoadjuvant therapy in patients with squamous cell carcinoma of the head and neck
human MHC non-restricted cytotoxic T-cell line
Human Monoclonal Antibody against Epidermal Growth Factor Receptor Linked to Monomethylauristatin F
Treatment of glioblastoma multiforme
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 158 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1316
n/a 11/2/2004 CuraGen Corporation1317
n/a 10/15/20131318
n/a 11/26/2013 Dyax Corporation1319
Aerumab 11 9/18/2006 Kenta Biotech Limited1320
n/a 1/6/2010 Kenta Biotech Limited1321
n/a 5/18/2016 Pfizer, Inc.1322
n/a 12/16/2013 Omeros Corporation
Human monoclonal antibody against platelet-derived growth factor D
To slow the progression of IgA nephropathy and delay kidney failure in patients affected by the disease.
human monoclonal antibody againt human interleukin 13 (IL-13)
Treatment of eosinophilic esophagitis
Novartis Pharmaceuticals Corporaton
human monoclonal antibody directed against active plasma kallikrein
Treatment of hereditary angioedema (HAE)
Human monoclonal antibody directed against serotype 011 Pseudomonas aeruginosa
Treatment of hospital acquired pneumonia caused by serotype 011 positive Pseudomonas aeruginosa
human monoclonal antibody directed against serotype O1 Pseudomonas aeruginosa
Treatment of pneumonia caused by serotype O1 positive Pseudomonas aeruginosa
human monoclonal antibody directed against tissue factor pathway inhibitor
Treatment of hemophilia A and hemophilia B patients with or without inhibitors, which includes routine prophylaxis to prevent or reduce the frequency of bleeding in hemophilia A and hemophilia B patients.
Human monoclonal antibody inhibitor of mannan-binding lectin-associated serine protease-2 (MASP-2)
Prevention (inhibition) of complement-mediated thrombotic microangiopathy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 159 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1323
n/a 4/30/2010 Treatment of ovarian cancer Ortho Biotech1324
n/a 2/29/20161325
n/a 4/30/2014 Cambryn Biologics1326
human plasminogen n/a 6/7/2010 Kedrion, S.p.A.1327
Tromplate 6/27/2013 Prophylix Pharma AS1328
Octaplex 2/1/2008 Octapharma USA, Inc.1329
n/a 5/4/20161330
n/a 7/6/2012 FibroGen, Inc.
Human monoclonal antibody targeting CC-chemokine ligand 2 (CNTO 888)
human monoclonal antibody targeting human sclerostin
Treatment of osteogenesis imperfecta.
Mereo Biopharma 3 Limited
Human plasma derived conagulation protein-Factor XI
For the treatment of congenital Factor XI deficiency
Treatment of ligneous conjunctivitis
human platelet antigen-1a immunoglobulin (anti-HPA-1a)
Prevention of fetal and neonatal alloimmune thrombocytopenia
Human prothrombin complex concentrate
Reversal of anticoagulation therapy in patients needing treatment of serious or life threatening bleeding and/or needing urgent surgery or invasive procedures
human recombinant bone morphogenetic protein 4
Treatment of glioblastoma multiforme.
HyperStem Therapeutics, Inc.
human recombinant DNA-derived, IgG1 kappa monoclonal anti-body to connective growth factor
Treatment of idiopathic pulmonary fibrosis
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 160 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1331
n/a 2/4/2016 AbbVie, Inc.1332
n/a 12/22/20141333
human retinal progenitor cells n/a 7/23/2012 jCyte, Inc.1334
n/a 10/14/2015 Treatment of ovarian cancer. Atara Biotherapeutics1335
n/a 2/4/2011 Neuralstem, Inc.1336
Vaxsyn Hiv-1 11/20/1989 Treatment of AIDS. MicroGeneSys, Inc.1337
n/a 7/20/2006 Treatment of pancreatic cancer GemVax A/S
human recombinant dual-variable domain immunoglobulin G1 monoclonal antibody that selectively neutralizes tumor necrosis factor-alpha and interkeukin-17A
Treatment of pediatric patients 0 to
human recombinant mesencephalic, astrocyte derived neurotrophic factor
Treatment of retinitis pigmentosa
Amarantus BioScience Holdings, Inc.
Treatment of retinitis pigmentosa
human soluble receptor-Fc fusion protein that targets human activin A
human spinal cord derived neural stem cells
Treatment of amyotrophic lateral sclerosis
Human T-lymphotropic virus type III Gp160 antigens
Human telomerase reverse transcriptase peptide vaccine
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 161 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1338
n/a 10/22/2014 Treatment of pancreatic cancer1339
n/a 10/1/2009 Treatment of liver cancer1340
Pneumostem 11/26/20131341
n/a 12/23/20151342
n/a 2/23/2016 treatment of mesothelioma.1343
n/a 1/20/20161344
n/a 6/24/2013 Treatment of neuroblastoma
human trtravalent, bi-specific monoclonal antibody that binds and co-inhibits two growth factor receptors; insulin-like growth factor receptor 1 (IGF-1R) and v-erb-b2 avian erythroblastic leukemia viral oncogene homolog 3 (ErbB3)
Merrimack Pharmaceuticals, Inc.
human tumor necrosis factor coupled to the C terminus of CNGRCG peptide
Molecular Medicine S.p.A. (Molmed)
human umbilical cord blood-derived mesenchymal stem cells
Prevention of bronchopulmonary dysplasia
MEDIPOST America, Inc.
Human Wilms tumor protein 1 peptides
Treatment of acute myeloid leukemia
SELLAS Life Sciences Group
Human wilms tumor protein 1 peptides
SELLAS Life Sciences Group
Human/Murine Chimeric Monoclonal antibody to Endoglin
Treatment of soft tissue sarcoma.
Tracon Pharmaceuticals, Inc.
Humanized 3F8-IgG1 monoclonal antibody
Memorial Sloan-Kettering Cancer Center
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 162 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1345
n/a 2/10/2016 MedImmune, LLC1346
n/a 9/29/2006 MacroGenics, Inc.1347
n/a 6/30/20141348
Humanized anti-tac Zenapax 3/5/19931349
n/a 4/29/2014 MorphoSys AG1350
n/a 12/1/2014 MorphoSys AG1351
n/a 2/17/20121352
n/a 5/26/2011 MENTRIK Biotech, LLC1353
humanized IgG2 antibody n/a 7/22/2008
humanized affinity-optimized, afucosylated immunoglobulin G1 kappa monoclonal antibody against CD19
Treatment of neuromyelitis optica and neuromyelitis optica spectrum disorders
Humanized anti-CD3 monoclonal antibody
Treatment of recent-onset Type I diabetes
humanized anti-IL-6R receptor neutralizing IgG2 monoclonal antibody
Treatment of neuromyelitis optica and neuromyelitis optica spectrum disorder
Chugai Pharma USA, LLC
Prevention of acute graft-vs-host disease following bone marrow transplantation.
Hoffmann-La Roche, Inc.
humanized Fc engineered monoclonal antibody against CD19
Treatment of patients with chronic lymphocytic leukemia/small lymphocytic lymphoma
humanized Fc engineered monoclonal antibody against CD19
Treatment of diffuse large B-cell lymphoma.
humanized IgG1 anti-serum amyloid A monoclonal antibody
Treatment of AA amyloidosis and AL amyloidosis
Prothena Therapeutics Limited
humanized IgG1 monoclonal anti-CD20 antibody
Treatment of follicular lymphoma
Treatment of vaso-occlusive crisis in patients with sickle cell disease.
Selexys Pharmaceuticals Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 163 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1354
n/a 12/12/2011 Opsona Theraeputics1355
n/a 7/27/20161356
n/a 8/16/2016 Vaccinex, Inc.1357
n/a 8/30/20161358
n/a 5/22/2014 Treatment of multiple myeloma.1359
n/a 9/11/20141360
n/a 8/5/2010 Biogen Idec, Inc.1361
n/a 8/20/2015 Forty Seven, Inc.
humanized IgG4 monoclonal antibody
Prevention of ischemia/reperfusion injury associated with solid organ tranplantation
humanized IgG4 monoclonal antibody against total complement component 1, subcomponent s (C1s)
Treatment of autoimmune hemolytic anemia (AIHA)
True North Therapeutics, Inc.
humanized IgG4 monoclonal antibody that binds to the SEMA4D antigen
Treatment of Huntington's disease
humanized IgG4 monoclonal antibody to Toll-Like Receptor 2 (TLR2)
Treatment of myelodysplastic syndrome
Opsona Therapeutics Ltd.
humanized immunoglobulin monoclonal antibody against CD38
Sanofi U.S. Services, Inc.
humanized monoclonal antibodies hu1B7 and hu11E6
Treatment of Bordetella pertussis
Synthetic Biologics, Inc.
humanized monoclonal antibody against human integrin alphaVbeta6
Treatment of idiopathic pulmonary fibrosis
humanized monoclonal antibody of the IgG4 kappa isotype targeting CD47
Treatment of acute myeloid (myelogenous) leukemia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 164 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1362
n/a 5/5/20151363
n/a 4/26/2011 Treatment of pancreatic cancer. Morphotek, Inc.1364
n/a 4/29/2011 Treatment of soft tissue sarcoma Morphotek1365
n/a 6/18/2008 Stromedix, Inc.1366
n/a 11/17/20111367
n/a 12/3/2009 Treatment of scleroderma MedImmune1368
hyaluronic acid n/a 3/19/2002 CoTherix
humanized monoclonal antibody that targets the a-subunit of the human IL-3 receptor also known as the CD123 antigen; (JNJ-56022473)
Treatment of acute myeloid leukemia
Janssen Research & Development, LLC
humanized monoclonal antibody to tissue factor
Humanized monoclonal antibody to TumorEndothelial Marker-1
humanized monocloncal antibody against human integrin alphaVbeta6
Treatment of fibrosis-associated chronic allograft nephropathy in kidney transplant patients.
humanized single chain monoclonal antibody(scFV: IgG1 hinge: IgG1 CH2 and CH3 domains)
Treatment of chronic lymphocytic leukemia
Emergent Product Development Seattle, LLC
humanized, afucosylated IgG1 kappa monoclonal antibody
Treatment of emphysema in patients due to alpha-1 antitrypsin deficiency
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 165 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1369
Hydralazine n/a 4/9/20041370
Transkrip(R) 3/17/20111371
Transkrip(R) 3/17/20111372
n/a 3/9/2015 Alsonex Pty Ltd1373
Chronocort(R) 9/3/2015 Diurnal Ltd.1374
Chronocort 3/18/2015 Diurnal Limited1375
Duocort 6/18/20081376
hydrocortisone oral granules Infacort(R) 5/13/2015 Diurnal Limited1377
n/a 6/16/2016 Mallinckrodt Inc.1378
Hydroxocobalamin n/a 9/22/2000 Jazz Pharmaceuticals1379
Trappsol 5/17/2010 Sphingo Biotech, LLC
Treatment of severe intrapartum hypertension (diastolic blood pressure greater than or equal to 110 or systolic blood pressure greater than or equal to 160) associated with severe preeclampsia/eclampsia of pregnancy
Bioniche Pharma USA LLC
hydralazine - magnesium valproate
Treatment of myelodysplastic syndrome
Neolpharma S.A.DE C.V.
hydralazine - magnesium valproate
Treatment of cutaneous T-cell lymphoma.
Neolpharma S.A.DE C.V.
hydrocinnamate-[Orn-Pro-dCha-Trp-Arg](CH3COO)
Treatment of amyotrophic lateral sclerosis
hydrocortisone modified release capsules
Treatment of adrenal insufficiency.
hydrocortisone modified release capsules
Treatment of congenital adrenal hyperpasia
hydrocortisone modified release tabs
Treatment of adrenal insufficiency
Shire ViroPharma, Inc.
Treatment of pediatric adrenal insufficiency (0 through 16 years of age).
hydromorphone hydrochloride (intrathecal infusion)
Treatment of complex regional pain syndrome
Treatment of acute cyanide poisoning
Hydroxy-Propyl-Beta-Cyclodextrin
Treatment of Niemann Pick Disease, Type C
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 166 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1380
Siklos 7/24/20131381
hydroxyurea Pedroxa 3/16/20151382
hydroxyurea n/a 4/15/20051383
Hypericin n/a 2/7/2000 Soligenix, Inc.1384
Hypericin n/a 8/3/2000 HyBiopharma, Inc.1385
Meveol 11/5/2009 Treatment of cystic fibrosis1386
I(131)-TM-601 (chlorotoxin) n/a 2/14/2002 Treatment of malignant glioma Morphotek, Inc.1387
ibalizumab n/a 10/20/2014 TaiMed Biologics, Inc.1388
ibrutinib n/a 9/8/2014 Pharmacyclics, LLC
hydroxycarbamide (hydroxyurea)
Treatment of sickle cell disease in patients under 18 years of age
addmedica Laboratories
Treatment of symptomatic sickle cell disease in pediatric patients, less than 17 years of age.
Ebelle D'Ebelle Pharmaceuticals LLC
Treatment of pediatric patients with sickle cell anemia.
UPM Pharmaceuticals, Inc.
Treatment of cutaneous T-cell lymphoma.
Treatment of glioblastoma multiforme.
Hypothiocyanite and lactoferrin
Alaxia Biotechnologies Sante
Treatment of HIV-1 infection in treatment experienced adult patients with documented multi-antiretroviral class resistance and evidence of HIV-1 replication despite ongoing antiretroviral therapy
Treatment of follicular lymphoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 167 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1389
ibrutinib Imbruvica 2/2/2016 Pharmacyclics, LLC1390
ibrutinib n/a 6/23/2016 Pharmacyclics, LLC1391
ibrutinib n/a 5/16/2013 Treatment of multiple myeloma Pharmacyclics, LLC1392
ibrutinib Imbruvica 2/5/2015 Pharmacyclics, LLC1393
ibrutinib Imbruvica 2/5/2015 Pharmacyclics, LLC1394
ibrutinib n/a 10/23/2013 Pharmacyclics, LLC1395 ibudilast n/a 6/1/2015 Treatment of Krabbe disease MediciNova, Inc.1396
ibudilast n/a 10/6/2016 MediciNova, Inc.1397
Ibuprofen i.v. solution Salprofen 10/29/1996 Farmacon-IL, LLC1398
idebenone n/a 3/25/2004 Treatment of Friedreich's ataxia1399
idebenone n/a 2/16/20071400
idebenone n/a 5/22/2009
Treatment of patients with extranodal marginal zone lymphoma (mucosa associated lymphoid tissue [MALT type] lymphoma)
Treatment of chronic Graft versus Host disease.
Treatment of nodal marginal zone lymphoma
Treatment of splenic marginal zone lymphoma
Treatment of diffuse large B-cell lymphoma
Treatment of amyotrophic lateral sclerosis
Prevention of patent ductus arteriosus.
Santhera Pharmaceuticals LLC
Treatment of Duchenne muscular dystrophy
Santhera Pharaceuticals Limited
Treatment of mitochondrial myopathy, encephalopathy, lactic acidosis with stroke-like episodes syndrome (MELAS)
Santhera Pharmaceuticals Limited
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 168 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1401
idebenone n/a 10/31/20061402
idelalisib n/a 9/26/2013 Gilead Sciences, Inc.1403
idelalisib n/a 10/15/2013 Gilead Sciences, Inc.1404
idelalisib n/a 10/15/2013 Gilead Sciences, Inc.1405
idelalisib n/a 10/15/2013 Gilead Sciences, Inc.1406
idelalisib n/a 8/25/2011 Gilead Sciences, Inc.1407
Idoxuridine n/a 4/8/1996 NeoPharm, Inc.1408
IDUA-HIRMAb fusion protein n/a 1/10/20081409
idursulfase beta n/a 2/11/2013 Green Cross Corp.1410
idursulfase IT n/a 9/3/2009
Treatment of Leber's hereditary optic neuropathy.
Santhera Pharmaceuticals Limited
Treatment of lymphoplasmacytic lymphoma with or without Walenstrom's macroglobulinemia
Treatment of splenic marginal zone lymphoma
Treatment of extranodal marginal zone lymphoma
Treatment of nodal marginal zone lymphoma.
Treatment of chronic lymphocytic leukemia
Treatment of nonparenchymatous sarcomas.
Treatment of mucopolysaccharidosis Type 1 (MPS)
ArmaGen Technologies, Inc.
Treatment of Hunter Syndrome (mucopolysaccharidoses)
For treatment of neurocognitive symptoms associated with Hunter Syndrome
Shire Human Genetic Therapies
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 169 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1411
iduvec n/a 1/21/2011 Zebraic Corporation1412
iferanserin (S-MPEC) n/a 12/13/20101413
ifosfamide n/a 8/7/19851414
n/a 6/22/2011 Treatment of multiple myeloma.1415
n/a 6/24/2013 Treatment of malignant glioma1416
n/a 4/6/2000 Treatment of astrocytic glioma.1417
iloprost n/a 4/6/20121418
imatinib mesylate n/a 5/6/20141419
Imciromab pentetate Myoscint 1/25/1989 Centocor, Inc.1420
imetelstat n/a 12/23/20151421
imetelstat n/a 6/11/2015 Treatment of myelofibrosis
Treatment of Mucopolysaccharidosis Type I
For use in pulmonary arterial hypertension.
Sam Amer & Company, Inc.
Treatment of soft tissue and bone sarcomas
Bristol-Myers Squibb Company
IgG1 chimeric monoclonal antibody
Immune System Therapeutics Ltd
IL-12 secreting dendritic cells loaded with autologous tumor lysate
Activartis Biotech GmbH
IL-4 Pseudomonas Toxin Fusion Protein (IL-4(38-37)-PE38KDEL)
Medicenna Therapeutics, Inc.
Treatment of pulmonary arterial hypertension
Algorithum Sciences, LLC
Treatment of progressive multifocal leukencephalopathy
Inhibikase Therapeutics, Inc.
Detecting early necrosis as an indication of rejection of orthotopic cardiac transplants.
Treatment of myelodysplastic syndrome
Janssen Research & Development, LLC
Janssen Research & Development, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 170 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1422Imexon n/a 11/8/1996 Treatment of multiple myeloma.
1423imexon Amplimexon 8/12/2005 Treatment of ovarian cancer.
1424
Imiquimod n/a 12/3/2014 UroGen Pharma Ltd1425
Elad 7/16/2004 Vital Therapies, Inc.1426
immune globulin (human) n/a 3/2/2010 Octapharma USA, Inc.1427
3/17/20041428
n/a 3/31/2016 Baxalta US, Inc.1429
Carimune Nf 5/4/2004 ZLB Bioplasma AG1430
Hizentra 8/18/2014 CSL Behring1431
n/a 9/10/2015 Hansa Medical AB1432
n/a 6/1/2015 Treatment of Hemophilia A
AmpliMed Corporation
AmpliMed Corporation
Treatment of carcinoma in situ (CIS) of the urinary bladder
Immortalized human liver cells found in the extracorporeal liver assist device
Treatment of fulminant hepatic failure (acute liver failure)
Treatment of Guillain-Barre syndrome
Immune Globulin (Human) containing high titers of West Nile virus antibodies
Omr-Igg-Am (Tm) 5% (Wnv)
Treatment of the West Nile virus infection
OMRIX Biopharmaceuticals, Ltd.
immune globulin infusion 10% (human) with recombinant human hyaluronidase)
Treatment of chronic inflammatory demyelinating polyneuropathy
Immune Globulin Intravenous (human)
Treatment for Guillain Barre Syndrome
Immune Globulin Subcutaneous (Human), 20% Liquid
Treatment of chronic inflammatory demyelinating polyneuropathy (CIDP)
immunoglobulin G degrading enzyme of Streptococcus pyogenes
Prevention of antibody mediated organ rejection in solid organ transplant patients.
immunologically active synthetic peptides
Apitope International NV
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 171 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1433
n/a 3/16/1994 Cytel Corporation1434
n/a 5/12/1992 ALK Laboratories, Inc.1435
Inalimarev and falimarev n/a 1/10/20061436
n/a 9/4/2014 Biostage, Inc.1437
Indium-111 pentetreotide Neuroendomedix 6/16/20061438
Inecalcitol n/a 5/14/2014 Hybrigenics, S.A.1439
inecalcitol n/a 8/3/2015 Hybrigenics, S.A.
Immunotherapeutic vaccine consisting of (PAM)2-Lys-Ser-Ser-Gln-Tyr-Ile-Lys-Ala-Asn-Ser-Lys-Phe-Ile-Gly-Ile-Thr-Glu-Ala-Ala-Ala-Phe-Leu-Pro-Ser-Asp-Phe-Phe-Pro-Ser-Val
Treatment of chronic active hepatitis B infection in HLA-A2 positive patients.
Imported fire ant venom, allergenic extract
For skin testing of victims of fire ant stings to confirm fire ant sensitivity and if positive, for use as immunotherapy for the prevention of IgE-mediated anaphylactic reactions.
Treatment of adenocarcinoma of the pancreas
Therion Biologics Corporation
inBreath airway transplant system
To restore the structure and/or function of the trachea subsequent to tracheal damage due to cancer, injury or infection
Treatment of neuroendocrine tumors
RadioIsotope Therapy of America (RITA) Foundation
Treatment of chronic lymphocytic leukemia
Treatment of acute myeloid leukemia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 172 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1440infliximab Remicade 10/23/2002 Centocor, Inc.
1441Infliximab Remicade 5/21/2003 Treatment of chronic sarcoidosis Centocor, Inc.
1442
n/a 10/2/20141443
n/a 1/29/20101444
inolimomab Leukotac 10/23/2002 Jazz Pharmaceuticals1445
Inosine pranobex Isoprinosine 9/20/19881446
inotuzumab ozogamicin n/a 3/25/2013 Pfizer, Inc.1447
n/a 9/5/20081448
Roferon-A 10/27/19891449
Roferon-A 5/11/1990
Treatment of juvenile rheumatoid arthritis
Inhibitor of primary and secondary KIT and PDGFR alpha kinase mutants found in GIST
Treatment of gastrointestinal stromal tumors (GIST)
Deciphera Pharmaceuticals, LLC
Inhibitor of Tissue Factor Pathway Inhibitor (TFPI)
Treatment of hemophilia A and hemophilia B.
Baxter Healthcare Corporation, Baxter BioScience
Treatment of graft versus host disease
Treatment of subacute sclerosing panencephalitis.
Newport Pharmaceuticals
Treatment of B-cell acute lymphoblastic leukemia
insecticidal toxin derived from Bacillus thuringiensis
Treatment of soil transmitted helminth infection strongyloidiasis
University of California, San Diego
Interferon alfa-2a (recombinant)
For use in combination with fluorouracil for the treatment of esophageal carcinoma.
Hoffmann-La Roche, Inc.
Interferon alfa-2a (recombinant)
For the treatment of metastatic malignant melanoma in combination with Teceleukin.
Hoffmann-La Roche, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 173 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1450
Roferon-A 5/14/19901451
Roferon-A 4/18/19881452
Roferon-A 5/2/19901453
Interferon beta-1a Rebif 3/11/1996 EMD Serono, Inc.1454
n/a 7/24/1992 Biogen, Inc.1455
Rebif 12/2/1992 EMD Serono, Inc.1456
Interferon gamma n/a 10/11/20071457
interferon gamma n/a 11/4/2011 Treatment of Friedreich's ataxia Roberto Testi, MD1458
interferon gamma-1b Actimmune 10/1/2014 Treatment of Friedreich's Ataxia1459
Interferon gamma-1b Actimmune 12/4/1995
Interferon alfa-2a (recombinant)
For the concomitant administration with fluorouracil for the treatment of advanced colorectal cancer.
Hoffmann-La Roche, Inc.
Interferon alfa-2a (recombinant)
Treatment of renal cell carcinoma.
Hoffmann-La Roche, Inc.
Interferon alfa-2a (recombinant)
For the concomitant administration with Teceleukin for the treatment of metastatic renal cell carcinoma.
Hoffmann-La Roche, Inc.
Treatment of patients with secondary progressive multiple sclerosis.
Interferon beta-1a (recombinant human)
Treatment of acute non-A, non-B hepatitis.
Interferon beta-1a (recombinant)
Treatment of symptomatic patients with AIDS including all patients with CD4 T-cell counts less than 200 cells per mm3.
Treatment of idiopathic pulmonary fibrosis
mondoBIOTECH Laboratories AG
Horizon Pharma Ireland Limited
Treatment of renal cell carcinoma.
Horizon Pharma Ireland Limited
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 174 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1460Interferon-alfa-1b n/a 4/17/2001 Treatment of multiple myeloma Ernest C.Borden
1461
Infradure Biopump 6/14/2012 Treatment of chronic hepatitis D Medgenics, Inc.1462
Kineret 9/15/20151463
Antril 9/23/19911464
Antril 10/16/19921465
Interleukin-1 Trap n/a 4/4/20051466
Interleukin-2 Teceleukin 5/3/19901467
Interleukin-2 Teceleukin 5/11/19901468
Interleukin-2 Teceleukin 2/5/1990
interferon-alpha secreting autologous micro-organ tissue converting into a biopump
interleukin-1 receptor antagonist anakinra
Treatment of Still's disease including systemic juvenile idiopathic arithritis and adult-onset Still's disease.
Swedish Orphan Biovitrum AB
Interleukin-1 receptor antagonist, human recombinant
Treatment of juvenile rheumatoid arthritis.
Swedish Orphan Biovitrum AB (publ) (SOBI)
Interleukin-1 receptor antagonist, human recombinant
Prevention and treatment of graft versus host disease in transplant recipients.
Swedish Orphan Biovitrum AB (publ) (SOBI)
Treatment of Still's disease including juvenile rheumatoid arthritis and adult-onset Still's disease
Regeneron Pharmaceuticals, Inc.
In combination with interferon alfa-2a for the treatment of metastatic renal cell carcinoma.
Hoffmann-La Roche, Inc.
In combination with interferon alfa-2a for the treatment of metastatic malignant melanoma.
Hoffmann-La Roche, Inc.
Treatment of metastatic renal cell carcinoma.
Hoffmann-La Roche, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 175 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1469Interleukin-2 Teleleukin 2/6/1990
1470
Ifrs 7/31/2001 Digestive Care, Inc.1471
Intravenous immune globulin Vitigam 7/26/2007 GammaCan Ltd.1472
Octagam(R) 7/31/2008 Octapharma USA, Inc.1473
intraventricular nimodipine n/a 5/28/20151474
Iobenguane I 131 Azedra Ultratrace 1/18/20061475
iobenguane I 131 n/a 7/5/20071476
Iobenguane Sulfate I-123 Omaclear 10/17/20051477
iobenguane sulfate I-123 Omaclear 10/21/20051478
n/a 8/1/1984 David E. Kuhl, M.D.1479
n/a 11/7/1988 Immunomedics, Inc.
Treatment of metastatic malignant melanoma.
Hoffmann-La Roche, Inc.
Intraoral fluoride releasing system
Prevention of dental caries due to radiation-induced xerostomia in patients with head and neck cancer
Treatment of Stage IIB to IV malignant melanoma
Intravenous immune globulin (human) 10%
Treatment of stiff-person syndrome
Treatment of subarachnoid hemorrhage
Edge Therapeutics, Inc.
Treatment of neuroendocrine tumors
Molecular Insight Pharmaceuticals
Treatment of neuroendocrine tumors
Jubilant DraxImage, Inc.
For scintigraphic detection, localization and staging of neuroblastoma.
Brogan Pharmaceuticals, LLC
For the detection, localization, and staging of pheochromocytomas.
Brogan Pharmaceuticals, Inc.
Iodine 131 6B-iodomethyl-19-norcholesterol
For use in adrenal cortical imaging.
Iodine I 123 murine monoclonal antibody to hCG
Detection of hCG producing tumors such as germ cell and trophoblastic cell tumors.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 176 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1480
Pentacea 2/22/20001481
131ichtnt-1 2/12/19991482
isavuconazonium sulfate n/a 10/20/20141483
Isobutyramide 12/18/19921484
Isobutyramide n/a 5/25/19941485
n/a 6/9/20151486
n/a 6/12/2014 Celgene Corporation1487
isofagomine tartrate n/a 1/10/2006 Treatment of Gaucher disease1488
Isoindolin-1,3-Di Thione n/a 11/21/2016 P2D, Inc.
Iodine I 131 bis(indium-diethylenetriaminepentaacetic acid)tyrosyllysine/hMN-14 x m734 F(ab')2 bispecific monoclonal antibody
Treatment of small-cell lung cancer.
IBC Pharmaceuticals, L.L.C.
Iodine I-131 radiolabeled chimeric MAb tumor necrosis treatment (TNT-1B)
Treatment of glioblastoma multiforme and anaplastic astrocytoma.
Peregrine Pharmaceuticals, Inc.
Treatment of invasive candidiasis/candidemia
Astellas Pharma Global Development, Inc.
Isobutyramide Oral Solution
Treatment of beta-hemoglobinopathies and beta-thalassemia syndromes.
Perrine, Susan P., M.D.
Treatment of sickle cell disease and beta thalassemia.
Alpha Therapeutic Corporation
isocitrate dehydrogenase 1 (IDH1)-mutant inhibitor
Treatment of acute myeloid leukemia (AML)
Agios Pharmaceuticals, Inc.
isocitrate dehydrogenase 2-mutant inhibitor
Treatment of acute myelogenous leukemia
Amicus Therapeutics, Inc.
Treatment of Frontotemporal dementia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 177 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1489
isotretinoin n/a 4/10/20141490
Itraconazole n/a 5/19/20161491
n/a 8/16/2016 Pulmatrix, Inc1492
Itraconazole suspension n/a 10/30/20081493
ivabradine n/a 3/23/2016 Amgen, Inc.,1494
n/a 5/19/1999 Glovax Co., Ltd.1495
ketotifen n/a 2/25/2015 Treatment of mastocytosis1496
Kre-Celazine n/a 4/1/20131497
Procysteine 7/30/19961498
Procysteine 6/14/1994
Treatment of congenital ichthyosis
Patagonia Pharmaceuticals, LLC
Treatment of basal cell carcinoma nevus syndrome.
HedgePath Pharmaceuticals, Inc
itraconazole inhalation powder
Treatment of pulmonary fungal infections in patients with cystic fibrosis
Topical treatment of fungal otitis externa (otomycosis)
Fairfield Clinical Trials, LLC
Treatment of pediatric patients (0 through 16 years of age) with dilated cardiomyopathy.
Japanese encephalitis vaccine (live, attenuated)
Prevention of Japanese encephalitis.
Valcrest Pharmaceuticals, LLC
Treatment of juvenile rheumatoid arthritis joint and related tissue inflammation in the pediatric population
All American Pharmaceutical & Natural Foods Corpor
L-2-oxothiazolidine-4-carboxylic acid
Treatment of amyotrophic lateral sclerosis.
Transcend Therapeutics, Inc.
L-2-oxothiazolidine-4-carboxylic acid
Treatment of adult respiratory distress syndrome.
Transcend Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 178 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1499L-5 Hydroxytrytophan n/a 11/1/1984
1500
L-5-hydroxytryptophan n/a 1/20/19991501
n/a 1/12/20111502
n/a 1/18/2006 CepTor Corporation1503
n/a 9/11/2013 Soligenix, Inc.1504
L-asparaginase n/a 3/27/2014 ERYTECH Pharma S.A.1505
Graspa 1/6/2010 Erytech Pharma1506
Graspa 7/6/2012 Treatment of pancreatic cancer. ERYTECH Pharma1507
L-baclofen Neuralgon 1/30/19921508
L-baclofen n/a 12/17/19911509
L-baclofen n/a 7/13/1990 Fromm, Gerhard M.D.
Treatment of postanoxic intention myoclonus.
Watson Laboratories, Inc.
Treatment of tetrahydrobiopterin deficiency.
Watson Laboratories, Inc.
L-amino acids, vitamins and minerals combined with omega-3 fatty acids
Treatment of patients with pediatric Crohn's disease
Immunopath Profile, Inc.
L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal
Treatment of Duchenne and Becker muscular dystrophy
L-arginyl-L-isoleucyl-L-valyl-L-prolyl-L-alanine-amide
Treatment of acute radiation syndrome
Treatment of acute myeloid leukemia
L-asparaginase encapsulated in red blood cells
Treatment of acute lymphoblastic leukemia
L-asparaginase encapsulated in red blood cells
Treatment of intractable spasticity in children with cerebral palsy.
Osmotica Pharmaceutical Corp.
Treatment of spasticity associated with spinal cord injury or multiple sclerosis.
Osmotica Pharmaceutical Corp.
Treatment of trigeminal neuralgia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 179 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1510
L-baclofen n/a 1/6/19981511
L-cycloserine n/a 8/1/1989 Treatment of Gaucher's disease. Lev, Meir M.D.1512
L-cysteine n/a 5/16/19941513
L-glutamine n/a 8/1/2001 Treatment of sickle cell disease Emmaus Medical, Inc.1514
L-glutamyl-L-tryptophan n/a 10/20/19991515
L-ornithine phenylacetate n/a 4/7/20101516
n/a 9/13/20111517
n/a 9/18/20091518
n/a 6/13/2016
Treatment of trigeminal neuralgia
Osmotica Pharmaceutical Corp.
For the prevention and lessening of photosensitivity in erythropoietic protoporphyria.
Brigham and Women's Hospital
Treatment of AIDS-related Kaposi's sarcoma.
Implicit Bioscience Pty Ltd
Treatment of hyperammonemia and resultant hepatic encephalopathy (HE) in patients with acute liver failure or acute on chronic liver disase
Ocera Therapeutics, Inc.
L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser
Treatment of neuropathic pain in patients with sarcoidosis.
Araim Pharmaceuticals, Inc.
L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser
Prevention of delayed graft function following renal transplant
Araim Pharmaceuticals, Inc.
L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser
Treatment to increase survival and improve functioning of pancreatic islets following transplantation
Araim Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 180 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1519
n/a 6/13/2016 Treatment of sarcoidosis.1520
n/a 4/26/20051521
L-tyrosine-L-serine-L-leucine Cms-024 9/10/20041522
L. reuteri n/a 8/1/20131523
Lactic acid Aphthaid 6/29/19991524
n/a 1/15/20021525
n/a 1/15/20021526
n/a 3/24/2015
L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser
Araim Pharmaceuticals, Inc.
L-threonyl-L-prolyl-L-prolyl-L-threonine
Treatment of neuropathic pain associated with spinal cord injury
Nyxis Neurotherapies, Inc.
Treatment of hepatocellular carcinoma.
CMS Peptides Patent Holding Company Limited
Prevention of necrotizing enterocolitis in preterm infants with birth weight less than or equal to 1,500 grams
Infant Bacterial Therapeutics
Treatment of severe aphthous stomatitis in severely, terminally immunocompromised patients.
Frontier Pharmaceutical, Inc.
lactic acid bacteria (Lactobacilli, Bifidobacteria, and Steptococci)
Treatment of active chronic pouchitis
VSL Pharmaceuticals, Inc.
lactic acid bacteria (Lactobacilli, Bifidobacteria, and Streptococcus species)
Prevention of disease relapse in patients with chronic pouchitis
VSL Pharmaceuticals, Inc.
Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis
Prevention of necrotizing enterocolitis (NEC) in premature infants with very low birth weight less than or equal to 1500 grams
Sigma-Tau Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 181 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1527
lactobacillus brevis CD2 n/a 10/4/2011 Treatment Behcet's disease1528
lactobacillus plantarum n/a 11/30/2016 New Biotic, LLC1529
laminin-111 (human) n/a 7/21/2011 Prothelia, Inc.1530
lanreotide acetate n/a 9/8/20111531
Laromustine Cloretazine 3/27/20141532
laromustine Onrigin 10/21/20041533
Latrodectus immune F(ab)2 Analatro 6/18/20011534
lebrikizumab n/a 3/9/2015 Genentech, Inc.1535
ledipasvir/sofosbuvir Harvoni 10/12/2016 Gilead Sciences, Inc.1536
Leflunomide n/a 10/18/19961537
lenalidomide Revlimid 4/29/2015 Celgene Corporation
VSL Pharmaceuticals, Inc.
Treatment of amyotrophic lateral sclerosis (ALS)
Treatment of Duchenne Muscular Dystrophy
Treatment of symptoms associated with carcinoid syndrome
Ipsen Biopharmaceuticals, Inc.
Treatment of acute myelogenous leukemia
Nanotherapeutics, Inc.
Treatment of acute myelogenous leukemia
Vion Pharmaceuticals, Inc.
Treatment of black widow spider envenomations
Instituto Bioclon, S.A. de C.V.
Treatment of idiopathic pulmonary fibrosis
Treatment of chronic hepatitis C virus (HCV) infection in pediatric patients
Prevention of acute and chronic rejection in patients who have received solid organ transplants.
Williams, MD, James W.
Treatment of extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 182 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1538lenalidomide Revlimid 1/17/2007 Celgene Corporation
1539lenalidomide Revlimid 3/28/2011 Celgene Corporation
1540lenalidomide Revlimid 9/17/2013 Celgene Corporation
1541Stargen (Tm) 4/30/2010 Treatment of Stargardt disease
1542
n/a 3/23/20161543
Ushstat(Tm) 5/17/20101544
Thalagen 1/11/20061545
lenvatinib n/a 3/27/2014 Eisai, Inc.1546
lenvatinib n/a 3/26/2014 Eisai, Inc.1547
lestaurtinib n/a 9/3/20091548
Lestaurtinib n/a 3/24/2006
Treatment of chronic lymphocytic leukemia
Treatment of diffuse large B-cell lymphoma
Treatment of follicular lymphoma
Lentiviral vector containing the human ABCA4 gene
Sanofi US Services, Inc.
lentiviral vector containing the human liver and erythroid pyruvate kinase gene
Treatment of pyruvate kinase deficiency.
Centro de Investigacion Biomedica en RED (CIBER)
Lentiviral vector containing the human MY07A gene
Treatment of retinitis pigmentosa associated with Usher syndrome 1B gene defect.
Sanofi US Services, Inc.
Lentiviral vector encoded with a human beta-globin gene plasmid
Treatment of beta-thalassemia major and beta-thalassemia intermedia
Memorial Sloan-Kettering Cancer Center
Treatment of hepatocellular carcinoma
Treatment of stage IIB to Stage IV melanoma
Treatment of Philadelphia-negative classic myeloproliferative disorders
Teva Branded Pharmaceutical Products R&D, Inc.
Treatment of acute myeloid leukemia
Teva Branded Pharmaceutical Products R&D, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 183 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1549
letermovir n/a 12/12/20111550
leukocyte interleukin Multikine 5/4/2007 CEL-SCI Corporation1551
n/a 1/26/2015 Treatment of cystic fibrosis Celtaxsys, Inc.1552
Leupeptin n/a 9/18/19901553
levetiracetam Keppra 4/30/2010 Treatment of neonatal seizures1554
levocarnitine Carnitor 11/15/19891555
Levocarnitine Carnitor 4/7/19971556
Levocarnitine Carnitor 11/22/1993
Prevention of human cytomegalovirus viremia and disease in at risk populations
Merck Sharpe & Dhome Corporation
Neoadjuvant therapy in patients with squamous cell carcinoma of the head and neck
leukotriene A4 hydrolase inhibitor
For use as an adjunct to microsurgical peripheral nerve repair.
Neuromuscular Adjuncts, Inc.
University of California
1. Prevention of secondary carnitine deficiency in valproic acid toxicity 2. Treatment of secondary carnitine deficiency in valproic acid toxicity
Sigma-Tau Pharmaceuticals, Inc.
Treatment of zidovudine-induced mitochondrial myopathy.
Sigma-Tau Pharmaceuticals, Inc.
Treatment of pediatric cardiomyopathy.
Sigma-Tau Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 184 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1557
Levofloxacin n/a 2/27/2008 Horizon Orphan LLC1558
levomefolate calcium n/a 2/10/2015 Cox Biosciences LLC1559
levothyroxine sodium n/a 4/26/20111560
liposomal amikacin Arikace 3/9/2006 Insmed, Inc.1561
liposomal amikacin Arikace 3/25/2013 Insmed, Inc.1562
liposomal amikacin Arikace 8/25/2009 Insmed, Inc.1563 Liposomal amphotericin B Ambisome 12/10/1996 Treatment of histoplasmosis. Fujisawa USA, Inc.1564
Liposomal annamycin n/a 6/17/2005
Treatment of pulmonary infections due to Pseudomonas aeruginosa and other bacteria in patients with cystic fibrosis patients
Treatment of megaloblastic anemia caused by folate deficiency
The preservation of organ function in brain-dead organ donors.
Fera Pharmaceuticals, LLC
Treatment of bronchopulmonary Pseudomonas aeruginosa infections in cystic fibrosis patients
Treatment infections caused by non-tuberculous mycobacteria
Treatment of bronchiectasis in patients with Pseudomonas aeruginosa or other susceptible microbial pathogens
Treatment of acute lymphoblastic leukemia
Callisto Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 185 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1565
Liposomal annamycin n/a 6/17/20051566
liposomal busulfan Busulipo 6/24/2013 Pharmalink AB1567
n/a 12/27/2006 Management of bronchiectasis Aradigm Corporation1568
n/a 4/19/2006 Management of cystic fibrosis Aradigm Corporation1569
n/a 6/1/2012 Aradigm Corporation1570
Liposomal cisplatin Lipova-Pt 5/23/2006 Treatment of ovarian cancer1571
Liposomal cyclosporin A Cyclospire 4/30/1998 Vernon Knight, M.D.1572
liposomal cyclosporine n/a 5/11/2009 PARI Pharma GmbH1573
n/a 6/24/2008 PARI Pharma GmbH1574
Sarcodoxome 12/27/2006 GP-Pharm SA
Treatment of acute myeloid leukemia
Callisto Pharmaceuticals, Inc.
For use as a conditioning regimen for patients with malignancies undergoing autologous or allogenic hematopoietic stem cell transplantation
Liposomal ciprofloxacin for inhalation
Liposomal ciprofloxacin for inhalation
liposomal ciprofloxacin plus ciprofloxacin
For the management of cystic fibrosis
Eleison Pharmaceuticals LLC
For aerosolized administration in the prevention and treatment of lung allograft rejection and pulmonary rejection events associated with bone marrow transplantation.
Treatment of bronchiolitis obliterans
liposomal cyclosporine for inhalation
Prevention of bronchiolitis obliterans.
Liposomal doxorubicin hydrochloride
Treatment of soft tissue sarcomas
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 186 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1575n/a 1/21/2015 Treatment of ovarian cancer
1576liposomal gadodiamide n/a 11/12/2008 Treatment of glioma.
1577liposomal glutathione n/a 5/18/2016
1578
Liposomal Glutathione n/a 4/30/20101579
Immther 6/10/1998 Treatment of osteosarcoma. Endorex Corp.1580
Immther 6/10/1998 Treatment of Ewing's sarcoma. Endorex Corp.1581
Liposomal nystatin Nyotran 6/13/20001582
n/a 4/27/20151583
n/a 12/5/2003 Bio-Path, Inc.1584
n/a 10/30/2008 Treatment of gliomas
liposomal encapsulated paclitaxel
Insys Development Company, Inc.
MedGenesis Therapeutix, Inc.
Treatment of Dengue Virus infection.
Your Energy Systems, LLC
Support of glutathione deficiency in individuals with inborn errors of metabolism of glutathione (IEMG)
Your Energy Systems, LLC
Liposomal N-Acetylglucosminyl-N-Acetylmuramly-L-Ala-D-isoGln-L-Ala -gylcerolidpalmitoyl
Liposomal N-Acetylglucosminyl-N-Acetylmuramly-L-Ala-D-isoGln-L-Ala -gylcerolidpalmitoyl
Treatment of invasive fungal infections.
The University of Texas
liposomal P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide
Treatment of acute myeloid leukemia
Bio-Path Holdings, Inc.
liposomal p-ethoxy growth receptor bound protein-2 antisense product
Treatment of chronic myelogenous leukemia
liposomal topotecan hydrochloride
MedGenesis Therapeutix, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 187 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1585liposomal vinorelbine n/a 10/12/2016
15869/28/2012
1587n/a 12/3/2014 Treatment of gastric cancer
1588n/a 6/20/1994
1589n/a 11/25/1991
1590
Lisofylline n/a 6/10/1999 Cell Therapeutics, Inc.1591
listeria monocytogenes n/a 9/5/2013 Treatment of pancreatic cancer Aduro BioTech, Inc.1592
lisuride n/a 1/17/2013 Sinoxa Pharma GmbH1593
n/a 12/13/2010 Medesis Pharma1594
n/a 11/4/2013 Advaxis, Inc.1595
n/a 5/21/2014 Treatment of osteosarcoma. Advaxis, Inc.
Treatment of cutaneous t-cell lymphoma (CTCL)
Taiwan Liposome Company, Ltd.
liposomal Ã}±-galactosylceramide
Lip. Alpha Galactosylceramide
Prevention of graft-versus-host disease
REGiMMUNE Corporation
liposome encapsulated paclitaxel
Insys Development Company, Inc.
Liposome encapsulated recombinant interleukin-2
Treatment of cancers of the kidney and renal pelvis
Oncothyreon Canada, Inc.
Liposome encapsulated recombinant interleukin-2
Treatment of brain and CNS tumors.
Oncothyreon Canada, Inc.
Treatment of patients undergoing induction therapy for acute myeloid leukemia.
Treatment of pulmonary arterial hypertension
lithium citrate tetrahydrate (in reverse micelle formulation)
Treatment of Huntington's disease.
live attenuated bioengineered Listeria monocytogenes immunotherapy
Treatment of human papilloma virus-associated head and neck cancer
live attenuated bioengineered Listeria monocytogenes immunotherapy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 188 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1596
n/a 12/20/2010 Marina Biotech1597
n/a 8/22/2016 Synlogic, Inc.1598
Not Determined 3/24/2015 Treatment of mesothelioma. Aduro BioTech, Inc.1599
n/a 5/4/2015 Treatment of pancreatic cancer Lokon Pharma AB1600
lomitapide n/a 3/3/20111601
lonafarnib n/a 4/18/20111602
Lonafarnib n/a 11/19/2013
live attenuated E. Coli expressing Beta catenin shRNA
Treatment of Familial Adenomatous Polyposis
live E. coli Nissle bacterium modified to assimilate ammonia
Treatment of urea cycle disorders
Live-attenuated Listeria monocytogenes actA/inlB/human mesothelin (hMeso) Strain hMeso38
LOAd703, a modified adenovirus 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand (TMZ-CD40L) and full length 4-1BBL
Treatment of Familial Chylomicronemia
Aegerion Pharmaceuticals, Inc.
Treatment of Hutchinson-Gilford progeria syndrome
The Progeria Research Foundation, Inc.
Treatment of Hepatitis Delta Virus (HDV)infection
Eiger Biopharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 189 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1603
n/a 2/27/2014 PROLOR Biotech, Ltd1604
losartan n/a 12/12/2011 Treatment of Marfan Syndrome1605
Ibsolvmir(R) 7/6/2011 TikoMed AB1606
Ibsolvmir 10/20/2009 TikoMed AB1607
lucerastat n/a 10/29/2015 Treatment of Fabry Disease.1608
Lucinactant Surfaxin 5/23/20061609
Lucinactant Surfaxin(R), Aerosurf(R) 10/21/2010 Treatment of cystic fibrosis.1610
Lucinactant Surfaxin 10/21/20051611
Lucinactant Surfaxin 10/18/19951612
Lucinactant Surfaxin 7/17/1995
long acting recombinantFactor VIIa-CTP3
Treatment and prophylaxis of bleeding episodes in patients with hemophilia A or B with inhibitors to factor VIII or factor IX
National Marfan Foundation
low molecular weight dextran sulfate
Treatment to mobilze progenitor cells prior to stem cell transplantation
low molecular weight dextran sulfate
Prevention of graft rejection during pancreatic islet transplantation
Actelion Pharmaceuticals Ltd
Prevention of bronchopulmonary dysplasia in premature infants
Windtree Therapeutics, Inc.
Windtree Therapeutics, Inc.
Treatment of bronchopulmonary dysplasia in premature infants.
Windtree Therapeutics, Inc.
Treatment of respiratory distress syndrome in premature infants.
Windtree Therapeutics, Inc.
Treatment of acute respiratory distress syndrome in adults.
Windtree Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 190 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1613lurbinectedin n/a 8/20/2012 Treatment of ovarian cancer PharmaMar USA, Inc.
1614
lutetium (177Lu)-edotreotide n/a 5/21/20151615
Thermodox (R) 3/17/2009 Celsion Corporation1616
n/a 9/10/20081617
MA09-hRPE cells n/a 2/2/20101618
Mafosfamide n/a 1/21/20031619
n/a 11/24/2008 Gliknik, Inc.1620
n/a 12/23/20151621
n/a 9/26/2013 CMC Contrast AB
Treatment of gastro-entero-pancreatic neuroendocrine tumors
ITG Isotope Technologies Garching GmbH
lyso-thermosensitive lipsomal doxorubicin
Treatment of hepatocellular carcinoma
lysosomal enzyme N-acetylgalactosamine-6-sulfate sulfatase
Treatment of mucopolysaccharidosis Type IVA (Morquio Syndrome)
Vivendy Therapeutics LTD
Treatment of Stargardt's macular dystrophy
Astellas Pharma Global Development Inc.
Treatment of neoplastic meningitis
Baxter Healthcare Corporation
MAGE-A3 cancer therapeutic Trojan peptide vaccine
Treatment of MAGE-A3 expressing head and neck squamous cell carcinoma
magnesium lysinate bis eicosapentaenoate
Treatment of familial adenomatous polyposis.
Thetis Pharmaceuticals
manganese (II) chloride tetrahydrate (with L-alanine and vitamin D3 as promoters of absorption)
Use as a targeted contrast agent for diagnostic MRI for the detection and localization of focal liver lesions in patients where use of gadolinium based contrast agents may be medically inadvisable or where gadolinium based contrast agents cannot be administered
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 191 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1622
n/a 2/27/20081623
Mannitol Bronchitol 2/11/2005 Pharmaxis Ltd.1624
n/a 4/27/20041625
maralixibat n/a 9/4/20131626
maribavir n/a 2/1/20071627
maribavir n/a 6/7/20111628
Marijuana n/a 5/25/19991629
marizomib n/a 9/21/2015 Treatment of malignant glioma.
Manganese superoxide dismutase mimetic
Prevention of radiation- or chemotherapy-induced oral mucositis in cancer patients
Galera Therapeutics, LLC
For use to facilitate clearance of mucus in patients with bronchiectasis and in patients with cystic fibrosis at risk for bronchiectasis
mannopentaose phosphate sulfate
Treatment of high-risk Stage II, Stage III, and Stage IV melanoma
Medigen Biotechnology Corp.
Treatment of progressive familial intrahepatic cholestasis
Shire Human Genetic Therapies, Inc.
Prevention of cytomegalovirus viremia and disease in the populations at risk.
Shire ViroPharma, Inc.
Treatment of clinically significant cytomegalovirus viremia and disease in at-risk patients.
Shire ViroPharma, Inc.
Treatment of HIV-associated wasting syndrome.
Multidisciplinary Association for Psychedelic Studies, Inc.
Triphase Research & Development I Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 192 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1630
Marizomib n/a 12/13/2013 Treatment of multiple myeloma1631
masitinib n/a 7/21/2009 AB Science1632 masitinib n/a 9/14/2005 Treatment of mastocytosis AB Science1633
masitinib n/a 4/20/2005 AB Science1634
masitinib mesylate n/a 3/18/2015 AB Science1635
masitinib mesylate n/a 9/14/2015 AB Science1636
Galardin 12/5/1991 Treatment of corneal ulcers. Glycomed, Inc1637
MaxAdFVIII n/a 3/3/2003 Treatment of Hemophilia A1638
n/a 7/14/2014 Treatment of ovarian cancer ImmunoGen, Inc.1639
n/a 5/12/2016 ImmunoGen, Inc.1640 mazindol n/a 7/6/2016 Treatment of narcolepsy NLS-0 Pharma AG1641
Mazindol Sanorex 12/8/1986 Collipp, Platon J. M.D.1642
Mecamylamine Inversine 10/14/1998 Targacept, Inc.
Triphase Research and Development I Corp.
Treatment of patients with pancreatic cancer
Treatment of malignant gastrointestinal stromal tumors
Treatment of amyotrophic lateral sclerosis.
Treatment of gastric cancer including cancer of the gastroesophageal junction
Matrix metalloproteinase inhibitor
GenStar Therapeutics Corporation
maytansinoid conjugated humanized monoclonal antibody against FOLRI
maytansinoid-conjugated humanized monoclonal antibody against CD37
Treatment of diffuse large B-cell lymphoma
Treatment of Duchenne muscular dystrophy.
Treatment of Tourette's syndrome.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 193 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1643mecasermin Iplex 12/3/2007 Insmed, Inc.
1644mecasermin rinfabate Iplex 7/23/2012
1645
mecasermin rinfabate n/a 9/20/2012 Premacure AB1646
Increlex 9/21/2015 Treatment of Rett Syndrome.1647
Medroxyprogesterone acetate Hematrol 2/22/20011648
Mefloquine HCl Mephaquin 7/22/1987 Mepha AG1649
n/a 9/6/2006 Caladrius Biosciences1650
Melanoma cell vaccine Canvaxin 10/13/19941651
Melanoma peptide vaccine n/a 3/29/20051652
Melanoma vaccine Melacine 12/20/1989
Treatment of myotonic dystrophy
Treatment of amyotropic lateral sclerosis
PCUT BioPartners, Inc.
Prevention of retinopathy of prematurity in premature infants born at risk for the disease
mecasermin, recombinant human insulin-like growth factor-1
Keck Graduate Institute of Applied Life Sciences
Treatment of immune thrombocytopenic purpura.
ZaBeCor Pharmaceutical Company, LLC
Treatment and prevention of chloroquine-resistant Falciparummalaria
Melanoma autologous dendritic cell vaccine
Treatment of stage IIIb through IV metastatic melanoma
Treatment of invasive melanoma.
CancerVax Corporation
Treatment of HLA-A2+ patients with stage IIB, IIC, III, and IV malignant melanoma
Bristol-Myers Squibb Research Inst
Treatment of stage III - IV melanoma.
Ribi ImmunoChem Research, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 194 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1653
n/a 9/13/20131654
Melatonin Circadin 7/9/20041655
melatonin n/a 11/3/2016 Therapicon SrL1656
Melatonin n/a 11/15/1993 Sack, Robert, M.D.1657
melatonin n/a 4/12/2013 Scharper S.p.A.1658
melphalan n/a 11/19/2012 Treatment of retinoblastoma Icon Bioscience, Inc.1659
melphalan n/a 11/19/2012 OncoTx, LLC1660
melphalan n/a 7/14/2015 Delcath Systems, Inc.1661
n/a 3/16/2015 Oncopeptides AB1662
melphalan hydrochloride n/a 11/12/2008 Delcath Systems, Inc.1663
melphalan hydrochloride n/a 11/12/2008 Delcath Systems, Inc.
melarsoprol-hydroxypropylbetadex
Treatment of human African trypanosomiasis (sleeping sickness)
Peter Kennedy, CBE, MD, PhD, DSc, FRCP FMedSci,
Treatment of non-24-hour sleep-wake disorder in blind individuals without light perception
Neurim Pharmaceuticals, Ltd.
Treatment of smith-magenis syndrome in combination with a beta-blocker
Treatment of circadian rhythm sleep disorders in blind people with no light perception.
Treatment of neonatal hypoxic ischemic encephalopathy
Treatment of Stage IIB through IV melanoma
Treatment of cholangiocarcinoma.
melphalan flufenamide hydrochloride
Treatment of plasma cell myeloma, also referred to as multiple myeloma
Treatment of patients with ocular (uveal) melanoma.
Treatment of patients with cutaneous melanoma.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 195 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1664melphalan hydrochloride n/a 5/22/2009 Delcath Systems, Inc.
1665melphalan hydrochloride n/a 9/26/2013 Delcath Systems, Inc.
1666
Menadione Sodium Bisulfite n/a 5/14/20141667
menadione sodium bisulfite n/a 5/14/20141668
menaquinone Menaquinonegold 9/4/2012 Treatment of calciphylaxis1669
menatetrenone Mk4 3/17/20111670
menatetrenone Mk4(R) 11/2/20101671
menatetrenone Mk4(R) 11/2/20101672
menatetrenone Mk4(R) 2/4/20111673
n/a 8/22/20111674
mepivacaine n/a 1/8/20071675
mepivacaine n/a 10/18/2006
Treatment of neuroendocrine tumors
Treatment of patients with hepatocellular carcinoma
For the treatment of autosomal dominant polycystic kidney disease
IC-MedTech Corporation
Treatment of autosomal dominant polycystic liver disease
IC-MedTech Corporation
Nu Science Trading, LLC
Treatment of acute myeloid leukemia
NBI Pharmaceuticals, Inc.
Treatment of hepatocellular carcinoma (HCC).
NBI Pharmaceuticals, Inc.
Treatment of acute promyelocytic leukemia.
NBI Pharmaceuticals, Inc.
Treatment of myelodysplastic syndrome.
NBI Pharmaceuticals, Inc.
menatetrenone and Vitamin D3
Treatment of myelodysplastic syndrome
NBI Pharmaceuticals, Inc.
Treatment of postherpetic neuralgia
Relmada Therapeutics, Inc.
Treatment of painful HIV-associated neuropathy
Relmada Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 196 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1676
mepolizumab n/a 5/28/2004 GlaxoSmithKline LLC1677
mepolizumab n/a 7/14/2011 GlaxoSmithKline LLC1678
mesalamine Canasa 1/19/20101679
n/a 9/10/20091680
Lialda 2/27/2008 Shire1681
n/a 9/10/20151682
Nurown 2/4/20111683
n/a 9/6/2016 Curis, Inc.1684
met-enkephalin; Met-N n/a 2/18/2011 Treatment of pancreatic cancer.1685
metadoxine n/a 12/16/2013 Treatment of Fragile X Syndrome Alcobra, Inc.
For first-line treatment in patients with hypereosinophilic syndrome
Treatment of Churg-Strauss Syndrome.
Treatment of pediatric ulcerative colitis
Aptalis Pharma US, Inc.
mesalamine and N-acetycysteine
Treatment of pediatric patients with ulcerative colitis (inclusive through age 16 years)
Altheus Therapeutics, Inc.
mesalamine; 5-aminosalicylic acid
Treatment of ulcerative colitis in pediatric patients (revised indication 10/21/2010).
mesencephalic, astrocyte-derived neurotrophic factor
Treatment of retinal artery occlusion.
Amarantus BioScience Holdings, Inc.
mesenchymal stromal cells secreting neurotrophic factors
Treatment of Amyotrophic Lateral Sclerosis
BrainStorm Cell Therapeutics Ltd.
mesylate synthetic small molecule inhibitor of HDAC and PI3K
Treatment of nuclear protein in testis (NUT) midline carcinoma
NBI Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 197 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1686
n/a 7/6/2016 Treatment of pancreatic cancer1687
metformin n/a 5/29/20141688
n/a 6/2/20161689
Methionine/L-methionine n/a 8/21/1996 Treatment of AIDS myelopathy. Genopia USA, Inc.1690
Methotrexate Amethopterin 8/28/2014 Treatment of Myasthenia Gravis1691
methotrexate n/a 8/20/2009 Orbona Pharma Ltd1692
n/a 3/18/20151693
methotrexate oral solution n/a 5/28/2015
metavert, N-hydroxy-4-(5-((4-methoxyphenyl)amino)-1,3,4-oxadiazol-2-yl)benzamide
Avenzoar Pharmaceuticals, Inc.
Treatment of pediatric polycystic ovary syndrome
EffRx Pharmaceuticals SA
methimazole and liothyronine sodium
Treatment of glioblastoma multiforme.
Musli Thyropeutics Ltd
The Universtiy of Kanasa Medical Center
Treatment of acute lymphoblastic leukemia
methotrexate oral liquid formulation
Treatment of acute lymphoblastic leukemia in pediatric patients aged 0 through 16 years.
Chesapeake Therapeutics
Treatment of acute lymphoblastic leukemia in pediatric patients (0 through 16 years of age)
Silvergate Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 198 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1694
methotrexate oral solution n/a 8/27/20151695
Methotrexate/Azone 10/15/19901696
Methoxsalen Uvadex 10/14/1998 Therakos, Inc.1697
methoxsalen Uvadex 5/12/1994 Therakos, Inc.
Treatment of oligoarticular juvenile idiopathic arthritis (persistent oligoarthritis, psoriatic juvenile idiopathic arthritis, enthesitis-related arthritis, or undifferentiated arthritis) and polyarticular juvenile idiopathic arthritis in children 0 through 16 years of age
Silvergate Pharmaeuticals, Inc.
Methotrexate with laurocapram
Topical treatment of mycosis fungoides.
Echo Therapeutics, Ltd.
For use in conjunction with the UVAR photopheresis system to treat graft versus host disease.
For the prevention of acute rejection of cardiac allografts
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 199 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1698
n/a 4/18/2011 Endotis Pharma1699
methylene blue n/a 7/24/20121700
methylene blue injection n/a 8/11/20121701
n/a 4/15/20131702
n/a 9/19/20161703
metreleptin n/a 8/22/2001
Methyl O-4-O-[2-[2-[2-[2-[[N-[(1R)-1-[[4-(aminoiminomethyl)phenyl]methyl]-2-oxo-2-(1-piperidinyl)ethyl]-N2-[(4-methoxy-2,3,6-trimethylphenyl)sulfonyl]-L-Ã?ÂÃÃ?Ã?Â&Ã?ýÃ?ÂÃÃ?ÂÃ?ñ-aspararginyl-4-aminobutanoyl-N6-[5-[(3aS,4S,6aR)-hexahydro-2-oxo-1H-thieno[
Prevention of ischemia reperfusion injury associated wth solid organ transplantation
Treatment of methemoglobinemia
Fera Pharmaceuticals, LLC
Treatment of congenital and acquired methemoglobinemia
Luitpold Pharmaceuticals, Inc.
methylparaben suberohydroxamic acid phenyl ester
Treatment of cutaneous T-cell lymphoma
TetraLogic Pharmaceuticals
methylprednisolone oral suspension
Treatment of adrenal insufficiency in pediatric patients
Generic Specialty Pharma
Treatment of leptin deficiency secondary to generalized lipodystrophy and partial familial lipodystrophy
Aegerion Pharnaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 200 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1704 metronidazole n/a 9/17/2008 Treatment of pouchitis Avivia Project BV1705
Metronidazole Metrogel 10/24/1991 Treatment of perioral dermatitis.1706
metronidazole n/a 4/26/2011 Treatment of pouchitis.1707
metronidazole n/a 4/5/20161708
Metronidazole 10% ointment n/a 11/8/2007 SLA Pharma (UK) Ltd1709
metyrapone n/a 9/25/20121710
metyrosine Demser 7/25/20081711
mexiletine n/a 9/2/20101712
mibefradil n/a 8/25/2009 Cavion, LLC1713
mibefradil n/a 10/16/2008 Treatment of pancreatic cancer. Cavion, LLC1714 mibefradil n/a 6/15/2007 Treatment of ovarian cancer Cavion, LLC1715
miconazole n/a 5/22/20141716
Microbubble contrast agent 11/16/1990 Cav-Con, Inc.
Galderma Laboratories, Inc.
S.L.A. Pharma Limited (UK)
Treatment of clostridium difficile infection in pediatric patients (0 through 16 years of age).
Appili Therapeutics, Inc.
Topical treatment of active perianal Crohn's disease
Treatment of Cushing's syndrome
Laboratoire HRA Pharma
Treatment of velocardiofacial syndrome associated psychosis.
Cerberus Princeton, LLC
Treatment of nondystrophic myotonia
University of Rochester Medical Center
Treatment of glioblastoma multiforme.
Treatment of fungal otitis externa (otomycosis)
Hill Dermaceuticals, Inc.
Filmix Neurosonographic Contrast Agent
Intraoperative aid in the identification and localization of intracranial tumors.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 201 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1717
n/a 1/25/2007 Thrombotargets Corp.1718
n/a 10/11/2007 Thrombotargets Corp.1719
midazolam n/a 7/24/20121720
midazolam n/a 10/20/20091721
midazolam n/a 2/8/20161722
midazolam n/a 2/18/2016 Treatment of status epilepticus
Microvesiculated modified glycosylated tissue factor
Treatment of non-life threatening, mild to severe bleeding episodes due to cutaneous injuries or dental procedures in hemophiliac patients
Microvesiculated modified glycosylated tissue factor
Treatment of non-life threatening, mild to severe bleeding episodes due to cutaneous injuries or dental procedures in patients with von Willebrand disease.
Treatment of nerve agent-induced seizures
Meridian Medical Technologies, Inc.
Rescue treatment of seizures in patients who require control of intermittent bouts of increased seizure activity (e.g. acute repetitive seizures, seizure clusters)
Upsher-Smith Laboratories, Inc.
Treatment of seizures induced by organophosporous insecticide poisoning.
Meridian Medical Technologies, Inc.
Shire Development LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 202 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1723
midazolam n/a 5/8/2006 UCB, Inc1724
midazolam n/a 2/4/2016 Treatment of status epilepticus1725
Midostaurin n/a 7/7/20091726 Midostaurin Rydapt 4/30/2010 Treatment of mastocytosis Novartis Oncology1727
mifamuritide Junovan 6/5/2001 Treatment of osteosarcoma1728
mifepristone Korlym 1/14/2016 Treatment of ovarian cancer.1729
Mifepristone n/a 2/7/2005 HRA Pharma1730
migalastat hydrochloride n/a 2/25/2004 Treatment of Fabry Disease1731
miglustat Zavesca (R) 11/12/20081732
milatuzumab n/a 6/24/2008 Immunomedics, Inc.
Treatment of bouts of increased seizure activity in selected refractory patients with epilepsy who are on stable regimens of anti-epileptic drugs and who require intermittent use of midazolam
Meridian Medical Technologies, Inc.
Treatment of acute myeloid leukemia
Novartis Pharmaceuticals Corporation
Millennium Pharmaceuticals, Inc.
Corcept Therapeutics, Inc.
Treatment of Cushing's syndrome secondary to ectopic ACTH secretion
Amicus Therapeutics, Inc.
Treatment of the neurological manifestations of Niemann-Pick disease,type C.
Actelion Pharmaceuticals Ltd
Treatment of chronic lymphocytic leukemia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 203 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1733milatuzumab n/a 3/10/2008 Treatment of multiple myeloma Immunomedics, Inc.
1734milciclib maleate n/a 9/20/2012
1735
miltefosine Miltex/Impavido 3/18/20091736
Minnelide Minnelide (Tm) 2/18/2013 Treatment of pancreatic cancer1737
Minocycline hydrochloride n/a 3/28/2006 Treatment of sarcoidosis1738
misoprostol Gymiso 1/10/20051739
Mitoguazone Apep 3/18/1994 ILEX Oncology, Inc.1740
Mitolactol n/a 1/23/1989 Targent, Inc..
Treatment of thymic epithelial tumors.
Tiziana Life Sciences PLC
Topical treatment of cutaneous lymphoma encompassing cutaneous manifestations of T-cell lymphoma and B-cell lymphoma
ExperGen Drug Development GmbH
Minneamrita Therapeutics, LLC
Autoimmunity Research Foundation
Treatment of intrauterine fetal death not accompanied by complete expulsion of the products of conception in the second and third trimesters of pregnancy.
Gynuity Health Projects, LLC
Treatment of diffuse non-Hodgkin's lymphoma, including AIDS-related diffuse non-Hodgkin's lymphoma.
Treatment of invasive carcinoma of the uterine cervix
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 204 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1741
Mitolactol n/a 7/12/1995 Targent, Inc.1742
mitomycin n/a 1/13/20111743
mitomycin n/a 6/1/20111744
mitomycin Mitogel 9/8/2014 TheraCoat, Ltd.1745
Mitomycin-C n/a 8/20/1993 IOP Inc.1746
n/a 10/7/20151747
n/a 2/4/20111748
n/a 2/4/20111749
Cea-Cide 11/24/1998 Treatment of pancreatic cancer. Immunomedics, Inc.
As adjuvant therapy in the treatment of primary brain tumors.
Prevention of recurrence of pterygium after its surgical excision.
Mobius Therapeutics, LLC
Prevention of corneal sub-epithelial haze formation following surface ablation laser keratectomy
Mobius Therapeutics, LLC
Treatment of upper tract urothelial cell cancers (transitional cell carcinoma of the renal pelvis and ureter)
Treatment of refractory glaucoma as an adjunct to ab externo glaucoma surgery.
Mixture of 2 anti-rabies humanized monoclonal immunoglobulin (Ig)G1k antibodies
Post-exposure prophylaxis against rabies virus infection
Synermore Biologics Co., Ltd
MLN4924-Inhibitor of Nedd8-activating enzyme (NAE)
Treatment of acute myelogenous leukemia.
Millennium Pharmaceuticals
MLN4924-Inhibitor of Nedd8-activating enzyme (NAE)
Treatment of Myelodysplastic syndrome
Millennium Pharmaceuticals, Inc.
MN14 monoclonal antibody to carcinoembryonic antigen
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 205 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1750
Cea-Cide 9/18/1998 Immunomedics, Inc.1751
Mocetinostat n/a 6/12/20141752
mocetinostat n/a 8/7/20141753
MOD-423 hGH analogue n/a 9/29/2010 OPKO Biologics Ltd.1754
modified a-cobratoxin n/a 9/1/20151755
n/a 4/8/2015 Treatment of uveal melanoma Aura Biosciences, Inc.1756
n/a 7/5/20161757
n/a 1/17/2013 Treatment of achondroplasia1758
n/a 8/20/2012 Gencia Corporation
MN14 monoclonal antibody to carcinoembryonic antigen
Treatment of small cell lung cancer
Treatment of myelodysplastic syndrome
Mirati Therapeutics, Inc.
Treatment of diffuse large B-cell lymphoma
Mirati Therapeutics, Inc.
Treatment of growth hormone deficiency
Treatment of pediatric multiple sclerosis (0 through 16 years of age).
Nutra Pharma Corporation
modified human papillomavirus capsid protein conjugated to a near infrared dye
modified mRNA encoding UGT1A1 protein, formulated in lipid nanoparticles
Treatment of Crigler-Najjar syndrome
Alexion Pharmaceuticals, Inc.
modified recombinant human C-type natriuretic peptide (CNP)
BioMarin Pharmaceutical, Inc.
modified recombinant mitochondrial transcription factor A (TFAM) containing the mitochondrial transduction domain
Treatment of inherited mitochondrial respiratory chain disease
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 206 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1759
mogamulizumab n/a 11/2/20101760
mogamulizumab n/a 11/2/20101761
mogamulizumab n/a 7/14/20111762
monarsen n/a 11/14/2003 Treatment of myasthenia gravis Bioline Rx, Ltd.1763
mongersen n/a 7/20/2015 Celgene Corporation1764
monoclonal antibody 11-1F4 n/a 12/7/2009 Alan Solomon, M.D.1765
monoclonal antibody 11-1F4 n/a 12/11/2009 Alan Solomon, M.D.1766
monoclonal antibody 3F8 n/a 10/16/2008 Treatment of neuroblastoma1767
n/a 8/25/2014 LeafBio, Inc.
Treatment of peripheral T-cell lymphoma.
Kyowa Kirin Pharmaceutical Development, Inc.
Treatment of patients with cutaneous T-cell lymphoma.
Kyowa Kirin Pharmaceutical Development Inc.
Treatment of adult T-cell leukemia/lymphoma (ATLL).
Kyowa Kirin Pharmaceutical Development, Inc.
Treatment of pediatric Crohn's disease
For use as a radioimmaging agent in amyloidosis
For use as a therapeutic agent for patients AL amyloidosis.
United Therapeutics Corporation
monoclonal antibody consisting of three mouse/human chimeric IgG1 monoclonal antibodies (c2G4, c4G7, and c13C6) that target Ebola virus
Treatment of Ebola virus infection
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 207 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1768
n/a 11/4/20131769
n/a 1/7/1993 Treatment of lupus nephritis.1770
monoclonal antibody Hu3F8 n/a 1/10/2014 Treatment of osteosarcoma1771
n/a 10/29/2015 ChemomAb, Ltd.1772
n/a 1/6/2015 Treatment of systemic sclerosis ChemomAb, Ltd.1773
Monoclonal antibody-B43.13 Ovarex Mab-B43.13 11/25/19961774
Monolaurin Glylorin 4/29/1993 Glaxo Wellcome Inc.1775
U-Cord-Cell(R) 9/15/20151776
motexafin gadolinium Xcytrin 1/27/2003 Pharmacyclics, LLC1777
n/a 3/24/2015
monoclonal antibody directed at hepatitis C virus E2 glycoprotein
Prevention of Hepatitis C recurrence in patients receiving liver transplantation
MassBiologics-University of MA Medical School
Monoclonal antibody for immunization against lupus nephritis
VivoRx Autoimmune, Inc.
Memorial Sloan-Kettering Cancer Center
monoclonal antibody targeting eotaxin-2
Treatment of idiopathic pulmonary fibrosis.
monoclonal antibody targeting eotaxin-2
Treatment of epithelial ovarian cancer.
Quest PharmaTech, Inc.
Treatment of congenital primary ichthyosis.
Mononuclear enriched fraction of human umbilical cord blood
Treatment of amyotrophic lateral sclerosis (ALS).
Saneron CCEL Therapeutics, Inc.
For use in conjunction with whole brain radiation for the treatment of brain metastases arising from solid tumors
mouse-human chimeric antibody of the IgG1 isotype, directed against human CD37
Treatment of chronic lymphocytic leukemia (CLL).
Boehringer Ingelheim Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 208 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1778
n/a 9/20/2012 Treatment of neuroblastoma. APEIRON Biologics AG1779
moxetumomab pasudotox n/a 11/15/2007 Treatment of hairy cell leukemia MedImmune, LLC1780
moxetumomab pasudotox n/a 6/28/2013 MedImmune, LLC1781
moxetumomab pasudotox n/a 11/15/2007 MedImmune, LLC1782
moxidectin n/a 9/29/20101783
mPEG-r-crisantaspase Asparec(Tm) 2/1/20101784
n/a 11/23/2016 PhaseRx, Inc.1785
MTC-DOX for Injection n/a 1/3/2001 FeRx Incorporated1786
n/a 10/16/2013 Celgene Corporation1787
n/a 12/7/2015 Treatment of ovarian cancer. Tapimmune, Inc.
mouse-human chimeric monoclonal anti-GD2 IgG1 antibody
Treatment of acute lymphoblastic leukemia
Treatment of CD22-positive chronic lymphocytic leukemia
Treatment of onchocerciasis volvulus in children and adults.
Medicines Development Limited
Treatment of acute lymphoblastic leukemia.
Jazz Pharmaceuticals, Inc.
mRNA encoding human ornithine transcarbamylase
Treatment of ornithine transcarbamylase deficiency
Treatment of hepatocellular carcinoma
mTOR kinase inhibitor (CC-223)
Treatment of hepatocellular carcinoma
multi-epitope folate receptor alpha peptide vaccine with GM-CSF adjuvant (TPIV200)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 209 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1788
n/a 7/27/20041789
Multi-peptide cancer vaccine n/a 6/23/2013 Treatment of multiple myeloma OncoPep, Inc.1790
n/a 12/12/19891791
Multistem(r) n/a 9/15/2010 Athersys, Inc.1792
Oncolym 11/27/19981793
Theragyn 3/22/1999 Antisoma plc1794
Begedina 2/18/2011 Adienne S.A.
Multi-ligand somatostatin analog
Treatment of patients with functional gastroenteropancreatic (GEP) neuroendocrine tumors (specifically, carcinoid, insulinoma, gastrinoma, somastatinoma, GRFoma, VIPoma and glucagonoma.
Novartis Pharmaceuticals Corporation
Multi-vitamin infusion (neonatal formula)
For establishment and maintenance of total parenteral nutrition in very low birth weight infants.
Astra Pharmaceuticals, L.P.
Prophylaxis of graft vs host disease
Murine MAb (Lym-1) and Iodine 131-I radiolabeled murine MAb (Lym-1) to human B-cell lymphoma
Treatment of B-cell non-Hodgkin's lymphoma.
Peregrine Pharmaceuticals, Inc.
Murine MAb to polymorphic epithelial mucin, human milk fat globule 1
Adjuvant treatment of ovarian cancer.
murine monoclonal antibody against CD26
Treatment of graft versus host disease
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 210 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1795
n/a 9/23/2014 Treatment of Neuroblastoma1796
Mx-dnG1 Rexin-G 6/24/2008 Treatment of osteosarcoma.1797
Mx-dnG1 Rexin-G(R) 6/24/2008 Treatment of soft tissue sarcoma1798
Rexin-G 8/15/2003 Treatment of pancreatic cancer1799
n/a 10/11/1995 Statens Seruminstitut1800
mycobacterium vaccae n/a 8/20/2010 Treatment of tuberculosis1801
myo-inositol n/a 4/7/2005 Abbott Nutrition1802
n/a 10/29/2015 Treatment of malaria.
MV-NB-02, its bivalent ganglioside vaccine consisting of GD2-lactone and GD3-lactone each covalently conjugated to keyhole hemocyanin
MabVax Therapeutics, Inc.
Epeius Biotechnologies Corporation
Epeius Biotechnologies Corporation
Mx-dnG1 or Rexin-G retroviral vector
Epeius Biotechnologies Corporation
Mycobacterium avium sensitin RS-10
For use in the diagnosis of invasive Mycobacterium avium disease in immunocompetent individuals.
Immodulon Therapeutics Ltd
Prevention of retinopathy of prematurity in preterm infants at risk for developing retinopathy of prematurity
N'-(7-Chloroquinolin-4-yl)-N,N-diethyl-propane-1,3-diamine
Donald J. Krogstad, MD
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 211 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1803
n/a 4/6/2012 Treatment of mercury toxicity CTI Science Limited1804
n/a 11/17/20141805
n/a 11/17/2014 Treatment of optic neuritis Bionure Farma SL1806
n/a 8/8/20071807
n/a 2/1/2008
N,N'-bis(2-mercaptoethyl)isophthalamide (NBMI)
N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide
Treatment of Duchenne muscular dystrophy
Catabasis Pharmaceuticals, Inc.
N-(2-amino-2-oxoethyl)-2-(2-((4-fluorophenethyl)amino)-N-isobutylacetamido)-N-(3-(2-oxopyrrolidin-1-yl)propyl)acetamide N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)propyl}-carbamoyl}-methyl)-2-[2-(2-fluorophenyl)-ethylamino]-N-isobutyl-acetamide
N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide dihydrobromide
Treatment of Hodgkin's lymphoma
Mirati Therapeutics, Inc.
N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide dihydrobromide
Treatment of acute myeloid leukemia
Mirati Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 212 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1808n/a 10/15/2014 Treatment of AL Amyloidosis ProteoTech, Inc.
1809
n/a 11/26/2013 Treatment of gliomas1810
n/a 7/6/2012 BioLineRx, Ltd.1811
n/a 2/18/2016 Treatment of glioma.1812
n/a 10/21/2014 Treatment of ovarian cancer Amgen, Inc.1813
n/a 3/18/2009
N-(3,4-dihydroxyphenyl)-3,4-dihydroxybenzamide
N-(3-fluorobenzyl)-2-(5-(4-morpholinophenyl) pyridin-2-yl) Acetamide Besylate
Kinex Pharmaceuticals, Inc.
N-(4-Fluoro-benzoyl)-L-arginyl-L-arginyl-[L-3-(naphthyl-alanyl]-L-cysteinyl-L-tyrosyl-L-citrullinyl-L-lysyl-D-lysyl-L-prolyl-L-trosyl-L-arginyl-L-citrullinyl-L-cysteinyl-L-aginine amide, cyclic (4-13)-disulfide
For use to mobilize hematopoietic stem cells (HSCs) from the marrow to peripheral blood for collection for autologous or allogeneic transplantation
N-(4-Methoxyphenyl)-N,2,6-trimethylfuro[2,3-d] pyrimidin-4-amine
Flag Therapeutics, Inc.
N-(4-{3-(2-aminopyrimidin-4-yl)pyridin-2-yl]oxy}phenyl)-4-(4-methylthiophen-2-yl)phthalazin-1-amine bis-mesylate dihydrate
N-(5-tert-Butylisoxazol-3-yl)-N'-{4-[7-(2-(morpholin-4-yl)ethoxy) imidazo[2,1-b][1,3]benzothiazol-2-yl]phenyl}urea di-hydrochloride salt
Treatment of acute myeloid leukemia
Daiichi Sankyo Pharma Development
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 213 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1814
n/a 11/17/20141815
n/a 8/5/2010 Treatment of myelofibrosis Gilead Sciences, Inc.1816
n/a 10/17/2005 Treatment of multiple myeloma. AB Science1817
N-acetyl cysteine amide n/a 12/31/20131818
n/a 2/6/2006 ExSAR Corporation1819
N-acetyl-procainamide n/a 12/10/1996 NAPA of the Bahamas1820
N-acetylcysteinate Lysine 12/27/2000
N-(9-methoxynonyl)-1-deoxynojirimycin hydrochloride
Treatment of acute dengue illness (inclusive of acute dengue illness, dengue fever, dengue hemorrhagic fever, and dengue shock syndrome)
Emergent Virology, LLC
N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl(phenyl]amino}pyrimidin-4-yl)benzamide
N-(methyl-diazacyclohexyl-methylbenzamide)-azaphenyl-aminothiopyrrole) mesylate
Treatment of retinitis pigmentosa
Nacuity Pharmaceuticals, Inc.
N-acetyl-glucosamine thiazoline
Treatment of adult Tay-Sachs disease
Prevention of life-threatening ventricular arrhythmias in patients with documented procainamide-induced lupus.
Nacystelyn Dry Powder Inhaler
For the management of cystic fibrosis
Galephar Pharmaceutical Research, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 214 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1821
N-acetylcysteine n/a 7/19/20121822
N-acetylcysteine n/a 9/9/2002 Treatment of acute liver failure1823
n/a 8/31/20151824
n/a 4/5/20101825
n/a 10/16/2007 Treatment of tuberculosis. Sequella, Inc.1826
n/a 1/13/2012 Treatment of meningioma1827
n/a 1/13/20121828
N-Methanocarbathymidine n/a 10/15/2014 Treatment of neonatal herpes
Prevention of ototoxicity caused by platinum-based chemotherapeutic agents used to treat pediatric cancers
Edward A. Neuwelt, MD
Cumberland Pharmaceuticals, Inc.
N-acetylcysteine and sodium thiosulfate
Prevention of platinum-induced toxicities in pediatric patients (0 through 16 years of age).
Edward A. Neuwelt, MD
N-Acetylmannosamine (ManNAc)
Treatment of hereditary inclusion body myopathy type 2
Escala Therapeutics, Inc.
N-adamantanyl-N'-Geranyl-ethylenediamine
N-Hydroxy-4-(3-methyl-2-(S)phenyl-butyrylamino)benzamide
Arno Therapeutics, Inc.
N-Hydroxy-4-(3-methyl-2-(S)phenyl-butyrylamino)benzamide
Treatment of Schwannoma of the central nervous system
Arno Therapeutics, Inc.
N & N Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 215 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1829
n/a 7/18/2013 Treatment of mesothelioma Verastem, Inc.1830
N-t-butylhydroxylamine n/a 5/12/2015 Andrew Lim1831
n/a 3/21/2013 Treatment of polycythemia vera1832
n/a 5/18/20091833
n/a 1/29/2010 Treatment of pancreatic cancer EMD Serono, Inc.
N-methyl-4-({4-[({3-methyl(methylsulfonyl)amino]pyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride
Treatment of infantile neuronal ceroid lipofuscinosis.
N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate
sanofi-aventis U.S. LLC
N-tert-butyl-3-[5-methyl-2-{[4-(2-pryyolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl-amino] benzenesulfonamide dihydrochloride monohydrate
Treatment of secondary and primary myelofibrosis
Sanofi-Aventis US, LLC a Sanofi company
N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino] isonicotinamide hydrochloride
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 216 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1834
n/a 1/12/2011 EMD Serono, Inc.1835
n/a 1/7/20151836
n/a 12/27/2006 CytRx Corporation1837
n/a 5/25/1995 Treatment of malignant glioma. Sugen, Inc.1838
n/a 3/12/1996 Treatment of ovarian cancer. Sugen, Inc.
N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino] isonicotinamide hydrochloride
Treatment of acute myeloid leukemia (AML).
N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt
For the treatment of Cushing's Syndrome
Millendo Therapeutics, Inc.
N-[3-(4',5'-bipyrimidin-2-ylamino)-4-methylphenyl]-4-{[(3S)-3-(dimethylamino)pyrrolidin-1-yl]methyl}-3-(trifluoromethyl)benzamide
Treatment of Philadelphia chromosome-positive chronic myelogenous leukemia.
N-[4-(trifluoromethyl)phenyl] 5 methylisoxazole-4-carboxamide
N-[4-(trifluoromethyl)phenyl]-5-methylisoxazole-4-carboxamide
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 217 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1839
n/a 2/3/2015 Ignyta, Inc.1840
n/a 2/12/2015 Ignyta, Inc.1841
n/a 2/4/2011 Treatment of tuberculosis. Sequella, Inc.1842
n/a 8/7/2014 Treatment of pancreatic cancer Sun BioPharma, Inc.1843
n/a 12/7/2000 For pancreatic cancer1844
n/a 2/27/2008 Treatment of multiple myeloma1845
nabumetone n/a 5/5/2008 Cook Pharma
N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide
Treatment of TrkA-positive, TrkB-positive, TrkC-positive ROS1-positive or ALK-positive non-small cell lung cancer
N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide
Treatment of TrkA-positive, TrkB-positive, TrkC-positive, ROS1-positive, or ALK-positive colorectal cancer
N-{[(5S)-3-(3-fluoro-4-thiomorpholin-4-ylphenyl)-2-oxo-1,3-oxazolidin-5-yl]methyl}acetamide
N1,N14,-diethyl-3,12,-dihydroxyhomospermine
N2'-deacetyl-N2'-(3-mercapto-1-oxopropyl)-Maystansine-Conjugated Humanized C242 Monoclonal Antibody
SmithKline Beecham Pharmaceuticals
N2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 Monoclonal Antibody
Biotest Pharmaceuticals Corporation
Treatment of pediatric juvenile rheumatoid arthritis
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 218 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1846
naloxone n/a 11/23/2010 Elorac, Inc.1847
naltrexone n/a 1/19/2010 TNI BioTech, Inc.1848
naltrexone n/a 2/23/20151849
naltrexone n/a 1/13/20151850
n/a 4/14/20141851
n/a 10/28/2015 Treatment of pancreatic cancer1852
n/a 7/9/20151853
n/a 5/23/2016 Treatment of ovarian cancer. PeptiMed1854
NanoDTPA(tm) n/a 6/17/2011
Topical treatment of pruritus associated with mycosis fungoides
Treatment of Crohn's disease in pediatric patients
Treatment of postherpetic neuralgia
Allodynic Therapeutics LLC
Treatment of autoimmune hepatitis
TaiwanJ Pharmaceuticals Co., Ltd.
naltrexone/clonidine combination
Treatment of postherpetic neuralgia
Allodynic Therapeutics LLC
nano-diamino-tetraiodothyroacetic acid
NanoPharmaceuticals, LLC
nano-diamino-tetraiodothyroacetic acid
Treatment of glioblastoma multiforme (GBM)
NanoPharmaceuticals, LLC
Nanocomplex consisting of a small interfering RNA that targets the human EV11 oncogenic transcription factor and an elastin-like polypeptide drug carrier.
Treatment of known or suspected cases of internal contamination with plutonium, americium, or curium
Nanotherapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 219 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1855
Edroma 11/29/2016 Treatment of Glioma1856 nanoparticulate paclitaxel Nanotax 10/6/2015 Treatment of ovarian cancer NanOlogy, LLC1857
napabucasin n/a 10/25/2016 Treatment of pancreatic cancer.1858
napabucasin n/a 6/20/20161859
naproxcinod n/a 3/16/2015 Nicox SA1860
n/a 11/18/2002 Treatment of polycythemia vera1861
n/a 1/18/2000 Treatment of Behcet's disease1862
n/a 8/10/20001863
NDX-peptides n/a 12/14/20101864
Nebacumab Centoxin 10/1/1986 Centocor, Inc.1865
nelarabine n/a 9/2/1999
nanoparticles containing Zoledronic acid
Laboratorio Italiano Biochimico Farmaceutico Lisapharma SpA
Boston Biomedical, Inc.
Treatment of gastric cancer including gastroesophageal junction cancer.
Boston Biomedical, Inc.
Treatment of Duchenne muscular dystrophy.
natural human lymphoblastoid interferon-alpha
Amarillo Biosciences, Inc.
Natural human lymphoblastoid interferon-alpha
Amarillo Biosciences, Inc.
Natural human lymphoblastoid interferon-alpha
Treatment of papillomavirus warts in the oral cavity of HIV positive patients.
Amarillo Biosciences, Inc.
Treatment of amyotrophic lateral sclerosis
NeoDiagnostic Research Ltd
Treatment of patients with gram-negative bacteremia which has progressed to endotoxin shock.
Treatment of chronic lymphocytic leukemia.
Novartis Pharmaceuticals Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 220 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1866
neostigmine n/a 3/18/20131867
neostigmine methylsulfate n/a 2/17/2012 Treatment of Myasthenia Gravis1868
neridronate n/a 3/25/2013 Grunenthal USA, Inc.1869
Neurotrophin-1 n/a 9/13/19941870
Cylexin 12/22/1993 Cytel Corporation1871
nevirapine n/a 11/25/20091872
n/a 11/9/2016 Gamida Cell Ltd.1873
Nifedipine n/a 6/13/1991 Treatment of interstitial cystitis.
Treatment of acute colonic pseudo-obstruction
Luitpold Pharmaceuticals, Inc.
Luitpold Pharmaceuticals, Inc.
Treatment of complex regional pain syndrome (CRPS-1, CRPS-II, CRPS-NOS)
Treatment of motor neuron disease/amyotrophic lateral sclerosis.
Ericsson, Arthur Dale, M.D.
Neutrophil-endothelial interaction inhibitor
Treatment of post-ischemic pulmonary reperfusion edema following surgical treatment for chronic thromboembolic pulmonary hypertension.
Prevention of HIV infection in pediatric patients under the age of 16 years
Auritec Pharmaceuticals
NiCord (cord blood-derived exvivo expanded CD34+ cells and the non-expanded cell fraction of the same cord blood unit)
Treatment of chronic myelogenous leukemia (CML)
Fleischmann, Jonathan M.D.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 221 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1874
nifurtimox Lampit 8/5/20101875
nifurtimox n/a 12/31/2013 Treatment of Chagas disease1876
Nikkomycin Z n/a 2/14/2006 Treatment of coccidioidomycosis1877
nimotuzumab n/a 9/8/2015 Treatment of pancreatic cancer. InnoCIMab Pte Ltd1878 Nimotuzumab n/a 11/17/2004 Treatment of glioma InnoKeys PTE Ltd.1879
n/a 9/3/2004 The Vaccine Company1880
n/a 9/3/2004 The Vaccine Company1881
n/a 9/3/2004 The Vaccine Company1882
nintedanib n/a 7/6/20161883
niprisan Hemoxin 8/15/2003 Treatment of sickle cell disease1884 niraparib n/a 4/30/2010 Treatment of ovarian cancer TESARO, Inc.1885
nitazoxanide Cryptaz 10/23/2001
Treatment of Chagas disease (American Typanosomiasis)caused by T. cruzi
Bayer HealthCare Pharmaceuticals, Inc.
MetronomX Therapeutics, LLC
Valley Fever Center for Excellence (1-111 INF)
Nine amino acid polypeptide derived from proteinase 3
Treatment of myelodysplastic syndromes requiring therapy
Nine amino acid polypeptide derived from proteinase 3
Treatment of acute myelogenous leukemia
Nine amino acid polypeptide derived from proteinase 3
Treatment of chronic myelogenous leukemia.
Treatment of systemic sclerosis (including the associated interstitial lung disease).
Boehringer Ingelheim Pharmaceuticals, Inc.
Xechem International, Inc.
Treatment for intestinal amebiasis
Romark Laboratories, L.C.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 222 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1886Nitisinone Orfadin 10/19/2001 Treatment of alkaptonuria Swedish Orphan AB
1887
Nitric oxide Inomax 9/27/2004 INO Therapeutics1888 Nitric oxide n/a 9/11/2013 Treatment of cystic fibrosis Novoteris, LLC1889
nitric oxide Inomax (R) 9/5/2008 INO Therapeutics1890
nitric oxide Inomax 12/28/20111891
Nitric oxide n/a 2/16/2005 Diagnosis of sarcoidosis1892
nitric oxide n/a 9/23/2014 Treatment of cystic fibrosis1893
Nitric oxide n/a 7/10/1995 INO Therapeutics, Inc.1894
nitric oxide n/a 6/18/2012 GeNO, LLC1895
Nitroprusside n/a 2/21/20011896
nivolumab Opdivo(R) 9/2/2015
To reduce the risk of chronic lung disease in premature neonates
Use in combination with a drug delivery device for acute treatment of sickle cell vaso-occlusive crisis (pain crises)
Treatment of pulmonary arterial hypertension
Bellerophon Therapeutics
SensorMedics Corporation
Advanced Inhalation Therapies Ltd.
Treatment of acute respiratory distress syndrome in adults.
Treatment of persistent pulmonary hypertension in newborns
Treatment and prevention of cerebral vasospasm following subarachnoid hemorrhage.
Thomas, MD, Jeffrey Evan
Treatment of hepatocellular carcinoma.
Bristol-Myers Squibb Company
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 223 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1897nivolumab n/a 9/15/2015
1898nivolumab n/a 8/27/2015 Treatment of glioblastoma
1899nivolumab Opdivo 8/22/2016 Treatment of esophageal cancer
1900
n/a 12/15/20141901
At001 12/3/20141902
n/a 5/24/20161903
n/a 12/8/2015 REGENXBIO, Inc.
Treatment of small cell lung cancer.
Bristol-Myers Squibb Company
Bristol-Myers Squibb Co
Bristol-Myers Squibb Co.
non-peptide small molecule orally available agonist of the parathyroid hormone receptor type I
Treatment of hypoparathyroidism
Chugai Pharma USA, LLC
non-replicating adeno-associated viral vector, serotype 8, expressing human myotubularin gene
Treatment of x-linked myotubular myopathy
Audentes Therapeutics, Inc.
non-replicating recombinant adeno-associated viral vector, serotype 8, expressing the 1A1 isoform of the bilirubin-uridine diphosphate glucuronosyltransferase gene
Treatment of Crigler Najjar syndrome.
Audentes Therapeutics, Inc.
non-replicating recombinant adeno-associated virus (AAV) vector of serotype 9 expressing human iduronate-2-sulfatase
Treatment of Mucopolysaccharidosis Type II
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 224 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1904
n/a 10/20/20141905
n/a 12/31/2013 US Biotest, Inc.1906
n/a 1/31/2014 US Biotest, Inc.1907
noscapine n/a 12/29/2015 KineMed Inc.1908
n/a 9/10/2009 Antisoma, Inc.1909
n/a 8/17/2004 Treatment of pancreatic cancer1910
n/a 7/28/2005 Antisoma Inc.1911
nusinersen n/a 4/18/2011 Biogen, Inc.1912
n/a 1/15/2013 Vudbenk Life Sciences
non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein
Treatment of retinitis pigmentosa
RetroSense Therapeutics, LLC
NorLeu3-Angiontensin(1-7)) [NorLeu3-A(1-7)]
Treatment of leakage from the surgical site following penetrating keratoplasty
NorLeu3-Angiotensin(1-7) [NorLeu3-A(1-7)]
Treatment of dermal injury due to nuclear/radiation incident
Treatment of amyotrophic lateral sclerosis.
Nucleic acid aptamer binding to tumor cell nucleolin
Treatment of acute myeloid leukemia
Nucleic acid aptamer binding to tumor cell nucleolin
Antisoma Research Ltd.
Nucleic acid aptamer binding to tumor cell nucleon
Treatment of renal cell carcinoma
Treatment of spinal muscular atrophy
O-(3-piperidino-2-hydroxyl-1-propyl)-nicotinic acid amidoxime hydrochloride
Treatment of Duchenne Muscular Dystrophy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 225 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1913
n/a 3/16/2012 JSK Therapeutics, Inc.1914
n/a 12/13/2013 Treatment of multiple myeloma JSK Therapeutics, Inc.1915
obinutuzumab n/a 2/17/2012 Genentech, Inc.1916
oblimersen Genasense 8/28/2001 Genta, Inc.1917
oblimersen Genasense 8/28/2001 Treatment of multiple myeloma Genta, Inc.1918
oblimersen Genasense 8/28/2001 Genta, Inc.1919
oblimersen Genasense 7/31/2000 Genta, Inc.1920
octreotide Sandostatin Lar 8/5/20101921
octreotide (oral) Octreolin(Tm) 6/17/2010 Chiasma, Inc.1922
n/a 12/7/2009 Treatment of acromegaly1923
oglufanide disodium n/a 9/24/2001 Treatment of ovarian cancer
O2-(2,4-dinitrophenyl) 1-[(4-ethoxycarbonyl)piperazin-1-yl]diazen-1-ium-1,2-diolate
Treatment of acute myeloid leukemia
O2-(2,4-dinitrophenyl) 1-[(4-ethoxycarbonyl)piperazin-1yl]diazen-1-ium-1,2-diolate
Treatment of diffuse large B cell lymphoma
Treatment of acute myelocytic leukemia
Treatment of chronic lymphocytic leukemia
Treatment of advanced malignant melanoma (Stages II,III, IV).
Treatment of neuroendocrine tumors
Novartis Pharmaceuticals Corporation
For the oral treatment of acromegaly
octreotide acetate subcutaneous implant
Endo Pharmaceuticals Solutions, Inc.
Implicit Bioscience Pty Ltd
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 226 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1924
olaptesed pegol n/a 8/19/2014 Noxxon Pharma AG1925
oleylphosphocholine n/a 10/25/2013 Treatment of leishmaniasis1926
n/a 2/2/20161927
n/a 2/27/20141928
olipudase alfa n/a 8/3/2000 Genzyme Corporation1929
omacetaine mepesuccinate n/a 1/12/20091930
Technivie® Tablets 2/24/2016 AbbVie Inc.1931
Viekira Pak 7/16/2015 Abbvie, Inc.1932
n/a 3/3/2011 Treatment of soft tissue sarcoma1933
n/a 11/22/1995
Treatment of glioblastoma in conjunction with radiotherapy.
Dafra Pharma International nv
oligodendrocyte progenitor cells
Treatment of acute spinal cord injury
Asterias Biotherapeutics
oligopeptide containing 6 amino acids (H-Phe-Ser-Arg-Tyr-Ala-Arg-OH)
Treatment of amyotrophic lateral sclerosis
Genervon Biopharmaceuticals, LLC
Treatment of acid sphingomyelinase deficiency (Niemann-Pick disease)
Treatment of myelodysplastic syndromes
IVAX International GmbH
Ombitasvir/Paritaprevir/Ritonavir
Treatment of pediatric patients with chronic hepatitis C virus infection
ombitasvir/paritaprevir/ritonavir and dasabuvir
Treatment of pediatric patients with hepatitis C virus infection (0 through 16 years of age)
ombrabulin; N-{2-methoxy-5-[(Z)-2-(3,4,5-trimethozyphenyl)vinyl]phenyl}-L-serinamide hydrochloride
Sanofi-Aventis U.S., Inc.
Omega-3 (n-3) polyunsaturated fatty acid with all double bonds in the cis configuration
Prevention of organ graft rejection.
Research Triangle Pharmaceuticals
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 227 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1934Omacor 5/4/2000 Treatment of IgA nephropathy. Pronova Biocare, AS
1935
Omegaven emulsion Omegaven 2/27/20081936
n/a 2/10/2015 Treatment of esophageal ulcers1937
omigapil n/a 6/24/20081938
n/a 12/22/2014 Oncos Therapeutics1939 oncophage n/a 4/14/2009 Treatment of glioma Agenus, Inc.1940
n/a 10/14/20151941
opioid growth factor n/a 4/16/20131942
opium tincture n/a 12/2/2011
Omega-3 (n-3) polyunsaturated fatty acids
Treatment of parenteral nutrition-associated liver disease
Fresenius Kabi Deutschland GmbH
omeprazole-lansoprazole with buffer
Effexus Pharmaceuticals, LLC
Treatment of congenital muscular dystrophy.
Santhera Pharmaceuticals Limited
Oncolytic adenovirus coding for granulocyte macrophage colony stimulating factor (ONCOS-102)
Treatment of malignant mesothelioma
ondansetron inhalation powder
Prevention of chemotherapy-induced nausea and vomiting due to highly emetogenic chemotherapy in pediatric patients (0 through 16 years of age) .
Luxena Pharmaceuticals, Inc.
Treatment of liver and intrahepatic bile duct cancer
Primocure Pharma, Inc.
Treatment of chronic diarrhea in short bowel syndrome patients with an inadequate response to anti-diarrheal treatment
Valeant Pharmaceuticals International, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 228 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1943oprozomib n/a 8/25/2014
1944oprozomib n/a 10/28/2014 Treatment of multiple myeloma
1945
n/a 8/20/2012 Treatment of narcolepsy1946
oral testosterone n/a 6/2/2016 TesoRx Pharma, LLC1947
oral unfractionated heparin n/a 1/29/2004 Treatment of sickle cell disease TRF Technologies, Inc.1948
osilodrostat n/a 9/13/2013 Treatment of Cushing's disease1949
otelixizumab n/a 2/6/2006 GlaxoSmithKline1950
n/a 12/23/20141951 Oxaliplatin n/a 10/6/1992 Treatment of ovarian cancer. Debio Pharm S.A.1952 oxaloacetate n/a 7/24/2012 Treatment of gliomas Terra Biological LLC1953
Oxaloacetic acid n/a 11/18/2015 Terra Biological LLC1954
Oxalobacter formigenes n/a 3/29/2006 OxThera, Inc.
Treatment of Waldenstrom's macroglobulinemia
Onyx Therapeutics, Inc.
Onyx Therapeutics, Inc.
optically pure phenylalanine derivative
Jazz Pharmaceuticals International III Limited
Treatment of constitutional delay of growth and puberty in adolescent boys 14 to 17 years of age.
Novartis Pharmaceuticals corporation
Treatment of new-onset type I diabetes mellitus
OTL38; conjugate of pteroi acid and S0456 near infrared dye
Detecting folic receptor alpha positive lesions in ovarian cancer patients (diagnostic for the management of ovarian cancer)
On Target Laboratories, LLC
Treatment of amyotrophic lateral sclerosis
Treatment of primary hyperoxaluria
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 229 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1955
Oxandrolone Oxandrin 4/22/19971956
Oxandrolone Oxandrin 9/6/19911957
oxfendazole n/a 7/14/20141958
Oxymorphone Numorphan H.P. 3/19/19851959
Oxypurinol n/a 11/9/19981960
oxytocin n/a 11/24/2014 Pr Maithe Tauber1961
ozanimod n/a 12/29/20151962
n/a 1/9/2013 Lentigen Corporation1963
n/a 5/27/2014 Array BioPharma, Inc.
Treatment of patients with Duchenne's muscular dystrophy and Becker's muscular dystrophy.
Savient Pharmaceuticals, Inc.
Adjunctive therapy for AIDS patients suffering from HIV-wasting syndrome.
Bio-Technology General Corp.
Treatment of cysticercosis (including neurocysticercosis)
Robert H. Gilman, MD, DTMH and
For relief of severe intractable pain in narcotic-tolerant patients.
DuPont Merck Pharmaceutical Company
Treatment of hyperuricemia in patients intolerant to allopurinol.
Cardiome Pharma Corp.
Treatment of Prader-Willi syndrome
Treatment of pediatric (0 through 16 years of age) ulcerative colitis
Receptos Services, LLC
P140K MGMT transduced human CD34 cells
For bone marrow protection in the treatment of glioblastoma multiforme
p38 mitogen-activated kinase inhibitor
Treatment of dilated cardiomyopathy, including dilated cardiomyopathy secondary to lamin A/C gene mutations
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 230 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1964
Paclitaxel Paxene 4/15/19971965
paclitaxel n/a 5/1/2009 Treatment of pancreatic cancer1966
paclitaxel aqueous gel Oncogel(Tm) 2/1/2008 Treatment of esophageal cancer1967
paclitaxel aqueous gel Oncogel (Tm) 12/23/2008 Treatment of brain cancer. BTG International Inc.1968
paclitaxel nanoparticles n/a 1/2/2013 Treatment of pancreatic cancer CIRJ Co., Ltd.1969
paclitaxel poliglumex Opaxio 9/20/20121970
paclitaxel, micellar Paclical 4/3/2009 Treatment of ovarian cancer1971
paclitaxel-coated buttress n/a 7/15/2014 AcuityBio, Inc.1972
pacritinib n/a 3/13/20081973
pafuramidine maleate n/a 11/17/2006
Treatment of AIDS-related Kaposi's sarcoma.
Baker Norton Pharmaceuticals, Inc.
SynCore Biotechnology Co., Ltd.
BTG International, Inc.
Treatment of glioblastoma multiforme
CTI BioPharma Corporation
Oasmia Pharmaceutical AB
For use as local administration at the time of curative surgery in the treatment of stage 0 to III non-small cell lung cancer.
Treatment of primary myelofibrosis (MF), post-polycythemia vera MF, and post-essential thrombocythemia MF
CTI BioPharma Corporation
Treatment of pneumocystis jiroveci pneumonia
Immtech Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 231 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1974
Pafuramidine maleate n/a 5/14/2007 Treatment of malaria1975
Pafuramidine maleate n/a 8/31/20071976
palovarotene n/a 7/21/20141977
pantothenate phosphate n/a 5/4/2015 Retrophin, Inc.1978
Wobe-Mugos 12/21/1998 Treatment of multiple myeloma.1979
paquinimod n/a 1/17/2014 Treatment of systemic sclerosis Active Biotech AB1980
paromomycin n/a 2/11/20151981
n/a 1/18/20081982
n/a 10/19/2016 Serodus ASA
Immtech Pharmaceuticsl, Inc.
Treatment of human African trypanosomiasis (sleeping sickness)
Immtech Pharmaceuticals, Inc.
Treatment of fibrodysplasia ossificans progressiva
Clementia Pharmaceuticals, Inc.
Treatment of pantothenate kinase associated neurodegeneration (PKAN).
Papain, trypsin, and chymotrypsin
Marlyn Nutraceuticals, Inc.
Treatment of cutaneous leishmaniasis (Old World and New World)
The Surgeon General, Dept. of the Army
paromomycin sulfate/gentamicin sulfate
Treatment of all uncomplicated cutaneous leishmaniasis (both New World and Old World)
Office of the Surgeon General
Partial agonist at the opioid receptor-like 1 receptor with amino acid sequence Ac-Arg-Tyr-Tyr-Arg-Trp-Lys-Lys-Lys-Lys-Lys-Lys-Lys-NH2
Treatment of pulmonary arterial hypertension
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 232 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1983
Medi-491 5/7/1999 MedImmune, Inc.1984
pazopanib n/a 5/6/2013 Treatment of ovarian cancer.1985
n/a 4/2/2015 Treatment of angiosarcoma1986
PEG-glucocerebrosidase Lysodase 12/9/19921987
PEG-interleukin-2 n/a 2/1/1990 Chiron Corporation1988 pegargiminase n/a 7/21/2014 Treatment of mesothelioma. Polaris Group1989
Peginterferon alfa-2a Pegasys 9/30/19991990
Peginterferon alfa-2a Pegasys 7/13/19981991
pegsitacase Uricase-Peg 20 12/3/2009 EnzymeRx, LLC1992
pegylated arginine deiminase Hepacid 3/26/1999
Parvovirus B19 (recombinant VP1 and VP2; S.frugiperda cells) vaccine
Prevention of transient aplastic crisis in patients with sickle cell anemia.
Novartis Pharmaceuticals Corp.
PEG-b-PLA micelles containing (1) paclitaxel, (2) sirolimus (rapamycin) and (3) tanespimycin (17-AAG)(also known as: triolimus)
Co-D Therapeutics, Inc.
For use as chronic enzyme replacement therapy in patients with Gaucher's disease who are deficient in glucocerebrosidase.
National Institute of Mental Health, NIH
Treatment of primary immunodeficiencies associated with T-cell defects.
Treatment of chronic myelogenous leukemia.
Hoffman-La Roche Inc.
Treatment of renal cell carcinoma.
Hoffman-La Roche Inc.
Treatment of Lesch-Nyhan syndrome.
Treatment of hepatocellular carcinoma
Polaris Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 233 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
1993
Pegylated arginine deiminase Melanocid 4/12/19991994
pegylated carboxyhemoglobin n/a 10/21/2010 Sangart, Inc.1995
Sanguinate 1/15/20151996
n/a 4/30/2014 PharmaEssentia1997
n/a 3/16/2015 AERase, Inc.1998
n/a 4/10/2014 Treatment of myelofibrosis1999
n/a 4/11/20142000
n/a 4/2/2012 Treatment of polycythemia vera2001
n/a 10/25/20132002
n/a 10/1/2014 Treatment of pancreatic cancer
Treatment of invasive malignant melanoma.
Polaris Pharmaceuticals, Inc.
Treatment of acute painful sickling crises in patients with sickle cell disease
pegylated carboxyhemoglobin bovine
Treatment of the comorbidities associated with Sickle Cell Disease
Prolong Pharmaceuticals, LLC
pegylated granulocyte colony stimulating factor
Treatment of patients at risk of developing myelosuppression following a radiological or nuclear incident
pegylated human recombinant arginase I
Treatment of arginase I deficiency (hyperargininemia).
pegylated proline interferon alpha-2b
PharmaEssentia, Corporation
pegylated proline interferon alpha-2b
Treatment of essential thrombocythemia
PharmaEssentia, Corporation
pegylated proline-interferon alpha-2b; PEG-P-IFNa-2b
PharmaEssentia Corporation
PEGylated recombinant anti-Pseudomonas aeruginosa PcrV Fab' antibody
Treatment of cystic fibrosis patients with Pseudomonas aeruginosa lung infections
KaloBios Pharmaceuticals, Inc.
PEGylated recombinant human hyaluronidase PH20
Halozyme Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 234 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2003n/a 7/18/2016 Treatment of pancreatic cancer
2004pelareorep Reolysin 2/24/2015
2005pelareorep Reolysin(R) 2/10/2015 Treatment of ovarian cancer
2006pelareorep Reolysin 4/15/2015 Treatment of malignant glioma
2007pelareorep Reolysin 5/4/2015 Treatment of gastric cancer
2008pelareorep Reolysin(R) 2/24/2015
2009pelareorep Reolysin(R) 2/11/2015 Treatment of pancreatic cancer
2010
Peldesine n/a 10/5/19932011
pembrolizumab Keytruda 6/16/20152012
pembrolizumab Keytruda 12/30/20152013
pembrolizumab Ketruda 1/14/20162014
pembrolizumab Keytruda 2/17/2016 Treatment of multiple myeloma.2015
pembrolizumab Keytruda 10/11/2016
Pegylated recombinant human interleukin-10
ARMO BioSciences, Inc.
Treatment of fallopian tube cancer
Oncolytics Biotech, Inc.
Oncolytics Biotech, Inc.
Oncolytics Biotech, Inc.
Oncolytics Biotech, Inc.
Treatment of primary peritoneal cancer
Oncolytics Biotech, Inc.
Oncolytics Biotech, Inc.
Treatment of cutaneous T-cell lymphoma.
BioCryst Pharmaceuticals, Inc.
Treatment of gastric cancer, including gastroesophageal junction adenocarcinoma
Merck, Sharp & Dohme
Treatment of Hodgkin lymphoma.
Merck, Sharp & Dohme Corp.
Treatment of primary mediastinal B cell lymphoma.
Merck, Sharp & Dohme Corp.
Merck, Sharp & Dohme Corp.
Treatment of nasopharyngeal carcinoma
Merck, Sharp & Dohme Corp., a subsidiary of Merck & Co.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 235 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2016pentagastrin n/a 5/29/2009
2017Pentamidine isethionate n/a 10/29/1984 Aventis Behring L.L.C.
2018
Pneumopent 10/5/1987 Fisons Corporation2019
n/a 1/13/2015 CereNOva, LLC2020
Cylexin 7/18/1997 Cytel Corporation2021
pentetate trisodium 4/12/20042022
pentetrazol n/a 9/9/20152023
pentosan polysulfate sodium n/a 9/16/2011 Treatment of sickle cell disease.2024
pentosan polysulfate sodium n/a 11/21/2008 Treatment of sickle cell disease. TRF Pharma, Inc.
To aid in the diagnosis of medullary thyroid carcinoma
Medical Defense Technologies, LLC
Treatment of Pneumocystis carinii pneumonia.
Pentamidine isethionate (inhalation)
Prevention of Pneumocystis carinii pneumonia in patients at high risk of developing this disease
pentapeptide with sequence Ac-VSRRR-NH2
Treatment of intracerebral hemorrhage
Pentasaccharide ethyl glycoside consisting of one alpha-D-sialylosyl residue as a sodium salt, two beta-D-galactopyranosyl residues, one 2-acetamido-beta-D-glucopyranosyl unit, and one alpha-L-fucopyranosyl unit
Treatment of neonates and infants undergoing cardiopulmonary bypass during surgical repair of congenital heart lesions.
Diethylenetriaminepentaacetate
Treatment of patients with known or suspected internal contamination with plutonium, americium, or curium.
Heyl Chemisch-Pharmazeutische Fabrik GMBH & Co. KG
Treatment of idiopathic hypersomnia.
Balance Therapeutics, Inc.
Vanguard Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 236 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2025
pentosan polysulfate sodium Lysosan (Tm) 1/5/20152026
Pentostatin Nipent 1/29/1991 SuperGen, Inc.2027
Pentostatin Nipent 3/27/1998 SuperGen, Inc.2028
Pentostatin Nipent 11/24/1999 SuperGen, Inc.2029
pentoxifylline n/a 6/14/2012 Treatment of Behcet's disease2030
n/a 4/27/2006 Treatment of systemic sclerosis Digna Biotech, S.L.2031
n/a 4/26/2006 Digna Biotech, S.L.2032
n/a 6/29/2016 Treatment of pancreatic cancer.2033
n/a 9/15/2010 Rose Pharmaceuticals2034
peptide YY n/a 4/18/2011
Treatment of mucopolysaccharidosis (MPS) type VI
Plexcera Therapeutics, LLC
Treatment of patients with chronic lymphocytic leukemia.
Treatment of cutaneous T-cell lymphoma.
Treatment of peripheral T-cell lymphomas.
Keck Graduate Institute of Applied Life Sciences
Peptide 144 TGF beta-1 inhibitor
Peptide 144 TGF beta-1-inhibitor
Treatment of localized scleroderma
Peptide consisting of L-amino acids with the sequence H-lys-glu-phe-leu-his-pro-ser-lys-val-asp-leu-pro-arg-OH
Soricimed Biopharma, Inc.
Peptide that inhibits mechanosensitive ion channel (MSC) activity
Treatment of Duchenne Muscular Dystrophy (DMD.)
Treatment of hepatocellular carcinoma.
Mondobiotech Laboratories AG
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 237 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2035
n/a 2/20/2009 VAXON-Biotech2036
n/a 9/3/20092037
n/a 2/4/2011 Treatment of myasthenia gravis CuraVac Europe SPRL2038
n/a 5/14/20072039
perampanel Fycompa 12/7/2012 Eisai, Inc.2040
peretinoin n/a 9/23/2011
peptide-based vaccine targeting telomerase reverse transcriptase
Treatment of telomerase reverse transcriptase (TERT) positive non-small cell lung cancer in HLA-A*0201 positive patients
peptide-conjugated phosporodiamidate morpholino oligomer (PPMO)
Treatment of Duchenne muscular dystrophy in patients with a deletion, duplication or frame shift mutation correctable by skipping of exon 50 of the dystrophin gene to allow restoration of the reading frame (including mutations within exons 51, 51-53, or 51-55).
Sarepta Therapeutics, Inc.
peptides mimicking antigen receptors on autoimmune B cells and autoimmune T cells associated with myasthenia gravis
Peptidomimetic analog of hexarelin
Diagnosis of growth hormone deficiency
Aeterna Zentaris GmbH
Treatment of Lennox-Gastaut Syndrome
Treatment of hepatocellular carcinoma.
Kowa Pharmaceutical Europe Co. Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 238 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2041
Perflubron Liquivent(Tm) 4/26/20012042
perhexiline maleate n/a 6/14/20122043
perifosine n/a 7/9/2010 Treatment of neuroblastoma2044
perillyl alcohol n/a 4/18/2011 Treatment of glioma2045 pertuzumab n/a 6/8/2009 Treatment of ovarian cancer Genentech, Inc.2046
pertuzumab n/a 7/12/2013 Genentech, Inc.2047
pexastimogene devacirepvec n/a 5/6/20132048
n/a 7/5/20122049
PGC-C12E-terlipressin n/a 12/27/20122050
n/a 10/23/2013
Treatment of acute respiratory distress disease (ARDS) in adults
PFC Development/New Alliance Pharmaceutical
Treatment of moderate to severe symptomatic hypertrophic cardiomyopathy
Heart Metabolics, Limited.
Aeterna Zentaris GmbH
NeOnc Technologies, Inc.
Treatment of gastric cancer, including gastroesophageal junction cancer
Treatment of hepatocellular carcinoma
SillaJen Biotherapeutics, Inc.
PG2 (Astragalus polysaccharides extracted and purified from the dry root of Astragalus membranaceus)
Treatment of idiopathic thrombocytopenic purpura
PhytoHealth Corporation
Treatment of ascites due to all etiologies except for cancer
PharmaIN Corporation
phenobarbitol sodium injection
Treatment of hypoxic-ischemic encephalopathy to prevent siezures in neonates
Fera Pharmaceuticals, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 239 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2051
n/a 3/16/20152052
Phenylacetate n/a 3/6/19982053
Phenylalanine ammonia-lyase Phenylase. Ravpal-Peg 3/8/19952054
Phenylbutyrate n/a 1/19/20002055
phenylephrine n/a 2/14/2002 S.L.A. Pharma2056
phenylephrine n/a 1/31/2012 Treatment of Tetralogy of Fallot2057
n/a 11/2/20102058
n/a 8/23/2016 TG Therapeutics, Inc.2059
n/a 9/22/2008 Treatment of Ewing's sarcoma2060
picibanil n/a 3/17/2011 Richard Smith, M.D.
phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis (1,1-dimethyethyl) monohydrochloride
Treatment of Huntington's disease.
Ipsen Biopharmaceuticals, Inc.
For use as an adjunct to surgery, radiation therapy and chemotherapy for the treatment of patients with primary or recurrent malignant glioma.
Elan Drug Delivery, Inc.
Treatment of hyperphenylalaninemia
BioMarin Pharmaceutical
Treatment of acute promyelocytic leukemia.
Elan Drug Delivery, Inc.
Treatment of ileal pouch anal anastomosis related fecal incontinence
Luitpold Pharmaceuticals, Inc.
Phosphatidylinositol 3-Kinase "PI3K" Inhibitor
Treatment of chronic lymphocytic leukemia (CLL)
Semafore Pharmaceuticals
phosphoinositide-3-kinase delta inhibitor
Treatment of chronic lymphocytic leukemia
phosphorothioate antisense olignucleotide against EWS-Fli-1
The Cure Our Children Foundation
Treatment of lymphatic malformations
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 240 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2061
picibanil n/a 5/15/20092062
Picoplatin n/a 11/2/20052063
pirfenidone n/a 7/31/20142064
pirfenidone n/a 11/19/2013 Genentech, Inc.2065
n/a 6/30/2014 Treatment of cystic fibrosis2066
n/a 3/29/2005 Treatment of cystic fibrosis2067
plasmin (human) n/a 3/30/20092068
plasminogen (human) n/a 3/5/20132069
pleconaril n/a 12/22/2014
Treatment of patients with lymphatic malformations
Royds Pharmaceuticals, Inc.
Treatment of small cell lung cancer
Poniard Pharmaceuticals
Treatment of idiopathic pulmonary fibrosis
Genoa Pharmaceuticals, Inc.
Treatment of systemic sclerosis (including the associated interstitial lung disease)
plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a nonviral cationic lipid based gene transfer agent
Imperial Innovations Limited
Plasmid DNA vector expressing cystic fibrosis transmembrane gene
Copernicus Therapeutics, Inc.
Treatment of acute peripheral arterial occlusion
Grifols Therapeutics, Inc.
Treatment of hypoplasminogenemia, or type I plasminogen deficiency
ProMetic BioTherapeutics, Inc
Treatment of symptomatic enteroviral infection in the neonate
AntiVirus Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 241 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2070Plitidepsin Aplidin 9/30/2004 Treatment of multiple myeloma PharmaMar USA, Inc.
2071polidocanol n/a 2/19/2015 Provensis Ltd
2072
Poloxamer 188 n/a 8/5/1997 CytRx Corporation2073
Poloxamer 188 Florcor 2/22/1990 CytRx Corporation2074
poloxamer 188 n/a 11/8/20132075
poloxamer 188 (purified) n/a 6/27/19892076
Poloxamer 331 Protox 3/21/1991 CytRx Corporation2077
poloxamer-188 NF n/a 1/19/20102078
Poly I: poly C12U Ampligen 12/9/19932079
Poly I: poly C12U Ampligen 12/9/19932080
Poly I: poly C12U Ampligen 5/20/1991
Treatment of congenital venous malformations
Treatment of vasospasm in subarachnoid hemorrhage patients following surgical repair of a ruptured cerebral aneurysm.
Treatment of severe burns requiring hospitalization.
Treatment of Acute Limb Ischemia
Mast Therapeutics Inc.
Treatment of sickle cell disease (this includes the treatment and prevention of complications of sickle cell disease)
Mast Therapeutics Inc.
Initial therapy of toxoplasmosis in patients with AIDS.
Treatment of Duchenne muscular dystrophy
Phrixus Pharmaceuticals, Inc.
Treatment of invasive metastatic melanoma (stage IIb, III, IV).
Hemispherx Biopharma, Inc.
Treatment of chronic fatigue syndrome
Hemispherx Biopharma, Inc.
Treatment of renal cell carcinoma.
Hemispherx Biopharma, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 242 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2081
n/a 10/25/20132082
n/a 9/13/2016 Treatment of malaria2083
poly-CD-PEG-camptothecin n/a 5/21/2015 Treatment of ovarian cancer. Cerulean Pharma, Inc.2084
Poly-ICLC Hiltonol 3/17/1997 Oncovir2085
n/a 4/5/2016 TGV-Inhalonix, Inc.2086
n/a 3/17/2015 Treatment of homocystinuria2087
Zurase 9/14/1999 EnzymeRx, LLC
poly(lactide-co-glycolide) carboxylated microparticle
Treatment of acute encephalitis syndrome
Cour Pharmaceutical Development Company, Inc.
poly(lactide-co-glycolide) carboxylated microparticle
Cour Pharmaceutical Development Company, Inc.
Treatment of primary brain tumors.
poly-N,N'-hexamethyleneguanidine-poly-N1,N4-hexamethyleneaminoguanidine
Treatment of pulmonary infections in patients with cystic fibrosis including infections due to pseudomonas aeruginosa,Burkholderia cepacia and other bacteria
polyethylene glycol modified recombinant C-terminal truncate of human cystathionine beta-synthase
Orphan Technologies Limited
Polyethylene glycol-modified uricase
Prophylaxis of hyperuricemia in cancer patients prone to develop tumor lysis syndrome during chemotherapy.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 243 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2088
Zurase 12/21/1998 EnzymeRx, LLC2089
n/a 6/6/2005 EnzymeRx, LLC2090
polyinosinic-polycytidilic acid Poly-Iclc 8/2/2002 Oncovir2091
polyinosinic-polycytidilic acid Poly-Iclc 3/3/2003 Oncovir2092
Hiltonol 11/19/2002 Oncovir2093
Polymeric oxygen n/a 3/25/1992 Treatment of sickle cell anemia. Capmed USA2094
polyphenon E n/a 7/17/2008 Mitsui Norin Co., Ltd2095
Ampligen 7/19/1988 Treatment of AIDS2096
n/a 6/9/2006 Polynoma LLC
Polyethylene glycol-modified uricase
Treatment of tumor lysis syndrome in cancer patients undergoing chemotherapy.
Polyethylene glycol-modified uricase
Treatment of hyperuricemia in patients with gout refractory to conventional therapy or in whom conventional therapy is contraindicated
As an adjuvant to smallpox vaccination
Treatment of flavivirus infections including those due to West Nile, Japanese encephalitis, dengue, St. Louis encephalitis, yellow fever, Murray valley, and Banzai viruses
polyinosinic-polycytidilic acid (Poly-ICLC)
Treatment for orthopox virus infections
Treatment of chronic lymphocytic leukemia
Polyribonucleotide; Polyribo-inosinic/-cyclidylic/-uridylic acid
Hemispherx Biopharma, Inc.
Polyvalent, shed-antigen melanoma vaccine
Treatment of stage IIb to stage IV melanoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 244 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2097
pomalidomide n/a 9/21/2010 Celgene Corporation2098
ponatinib Iclusig(R) 3/12/20152099
Neurocell-Pd 12/17/1996 Diacrin/Genzyme LLC2100
Neurocell-Pd 12/17/1996 Diacrin/Genzyme LLC2101
Neurocell-Hd 12/10/1996 Diacrin/Genzyme LLC2102
Neurocell-Hd 12/10/1996 Diacrin/Genzyme LLC
Treatment of persons with myeloproliferative neoplasm-associated myelofibrosis and anemia who are red blood cell tranfusion dependent.
Treatment of gastrointestinal stromal tumors (GIST).
ARIAD Pharmaceuticals, Inc.
Porcine fetal neural dopaminergic cells and/or precursors aseptically prepared and coated with anti-MHC-1 Ab for intracerebral implantation
Treatment of Hoehn and Yahr stage 4 and 5 Parkinson's disease.
Porcine fetal neural dopaminergic cells and/or precursors aseptically prepared for intracerebral implantation.
Treatment of Hoehn and Yahr stage 4 and 5 Parkinson's disease.
Porcine fetal neural gabaergic cells and/or precursors aseptically prepared and coated with anti-MHC-1 Ab for intracerebral implantation
Treatment of Huntington's disease.
Porcine fetal neural gabaergic cells and/or precursors aseptically prepared for intracerebral implantation for Huntington's disease.
Treatment of Huntington's disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 245 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2103porcine GM1 ganglioside n/a 12/3/2012
2104
N-Graft 6/24/19972105
Porfimer sodium Photofrin 11/15/19892106
Porfimer sodium Photofrin 11/18/20042107
Porfiromycin Promycin 3/13/1997 Treatment of cervical cancer.2108
Porfiromycin Promycin 9/19/19952109
Posaconazole Noxafil 10/4/20162110
Posaconazole Posoril 7/16/2004 Treatment of zygomycosis2111
n/a 1/8/20072112
pracinostat n/a 2/27/2014 MEI Pharma Inc.
Treatment of acute spinal cord injury
TRB Chemedica International S.A.
Porcine Sertoli cells aseptically prepared for intracerebral co-implantation with fetal neural tissue
Treatment of Hoehn and Yahr stage four and five Parkinson's disease.
Titan Pharmaceuticals, Inc.
For the photodynamic therapy of patients with transitional cell carcinoma in situ of the urinary bladder.
QLT Phototherapeutics, Inc.
Treatment of cholangiocarcinoma
Concordia Laboratories, Inc.
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of head and neck cancer.
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of invasive aspergillosis
Merck, Sharp & Dohme Corp.
Schering-Plough Corporation
potassium sodium aluminosilicate
For treatment of poisoning by or exposure to cesium.
Castle Creek Pharmaceuticals, LLC
Treatment of acute myeloid leulemia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 246 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2113
pralatrexate n/a 5/3/20102114
pralatrexate n/a 10/20/20082115
pralatrexate n/a 10/20/20082116
pralmorelin hydrochloride n/a 10/18/20122117
pramipexole Mirapex 1/31/20082118
prasugrel hydrochloride Effient 5/26/2015 Treatment of sickle cell disease Eli Lilly2119
Primaquine phosphate n/a 7/23/1993 Sanofi Winthrop, Inc.2120
pritumumab n/a 10/28/2014 Treatment of glioma Nascent Biotech, Inc.2121
pritumumab n/a 4/6/2016 Treatment of pancreatic cancer. Nascent Biotech, Inc.2122
Procarbazine HCl Matulane 8/8/2006 Treatment of malignant glioma
Treatment of advanced or metastatic transitional cell carcinoma of the urinary bladder
Allos Therapeutics, Inc.
Treatment of follicular lymphoma
Allos Therapeutics, Inc.
Treatment of diffuse large B-cell lymphoma
Allos Therapeutics, Inc.
As a diagnostic agent for the detection of growth hormone deficiency
Sella Pharmaceuticals, Inc.
Treatment of Tourette's syndrome in pediatric patients
Boehringer-Ingelheim Pharmaceuticals, Inc.
For use in combination with clindamycin hydrochloride in the treatment of Pneumocystis carinii pneumonia associated with AIDS.
Sigma-Tau Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 247 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2123n/a 7/23/2015 Treatment of malignant glioma
2124profimer sodium Photofrin(R) 12/2/2011
2125
progesterone n/a 12/22/19942126
prolactin receptor antagonist Prolanta 4/15/2013 Treatment of ovarian cancer Oncolix, Inc.2127
n/a 12/9/20152128
propofol hemisuccinate n/a 8/19/2016 EpaleX Corporation2129
propranolol and etodolac n/a 4/27/2015 Treatment of pancreatic cancer.2130
propranolol and etodolac n/a 7/8/2015 Treatment of malignant glioma.2131
n/a 2/24/20152132
Circulase 6/12/19982133 Protaxel n/a 5/21/2003 Treatment of ovarian cancer Biophysica, Inc.
procaspase-activating compound 1
Vanquish Oncology, Inc.
Treatment of malignant mesothelioma
Concordia Laboratories, Inc.
Establishment and maintenance of pregnancy in women undergoing in vitro fertilization or embryo transfer procedures.
Watson Laboratories, Inc.
propagermanium and irbesartan
Treatment of focal segmental glomerulosclerosis.
Dimerix Bioscience Ltd.
Treatment of acute repetitive seizures
Vicus Therapeutics, LLC
Vicus Therapeutics, LLC
propranolol hydrochloride and etodolac
Treatment of hepatocellular carcinoma
Vicus Therapeutics, LLC
Prostaglandin E1 enol ester (AS-013)
Treatment of Fontaine Stage IV chronic critical limb ischemia.
LTT Baio-Pharma Co., Ltd
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 248 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2134
Protein C concentrate 6/19/19922135
n/a 9/19/2016 Fate Therapeutics2136
n/a 9/1/2015 TxCell SA2137
purified bovine type collagen n/a 4/27/2009 ImmuneWorks, Inc.2138
Immudyn 9/22/19972139
Purified type II collagen Colloral 2/9/1995 AutoImmune, Inc.2140
Imt504 11/24/2014
Protein C Concentrate (Human) Vapor Heated, Immuno
For replacement therapy in patients with congenital or acquired protein C deficiency for the prevention and treatment of warfarin-induced skin necrosis during oral anticoagulation.
Immuno Clinical Research Corp.
ProTmune (ex vivo Programmed Mobilized Peripheral Blood)
Prevention of graft-versus-host disease in patients undergoing allogenic hematopoietic cell transplantation
purified autologous type 1 regulatory T lymphocytes specific for human type II collagen
Treatment of chronic non-infectious uveitis
Treatment of idiopathic pulmonary fibrosis
Purified extract of Pseudomonas aeruginosa
Treatment of immune thrombocytopenia purpura where it is required to increase platelet counts.
Able Laboratories, Inc.
Treatment of juvenile rheumatoid arthritis.
PyNTTTTGT class of oligodeoxynucleotide with a 24-base single chain composed of nucleotide sequence 5'TCATCATTTTGTCATTTTGTCATT 3'
Treatment of rabies virus infections
Mid-Atlantic BioTherapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 249 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2141
pyridoxine; vitamin B6 n/a 3/3/20112142
pyrimethamine n/a 8/16/2011 ExSAR Corporation2143
Pyruvate n/a 2/21/2001 Cellular Sciences, Inc2144
pyrvinium n/a 1/5/20152145
quinacrine n/a 9/28/20122146
quinine sulfate n/a 12/2/20082147
R-(-)-gossypol n/a 10/24/20062148
n/a 9/10/2009 Treatment of gastric carcinoids Trio Medicines Ltd.2149
n/a 11/28/20082150
rAAV2-CB-hRPE65 n/a 2/11/2005
Treatment of pyridoxine dependent seizures.
NBI Pharmaceuticals, Inc.
Treatment of GM-2 gangliosidoses (Tay-Sachs disease and Sandhoff disease).
Treatment of interstitial lung disease.
Treatment of familial adenomatous polyposis
StemSynergy Therapeutics, Inc.
Treatment of hepatocellular carcinoma
Cleveland BioLabs, Inc & Incuron, LLC Joint Ventur
Treatment of malaria excluding Plasmodium faliparum
Zydus Pharmaceuticals, Inc.
Treatment of chronic lymphocytic leukemia.
Ascenta Therapeutics, Inc.
R-1-[2,3-dihydro-2-oxo-1-pivaloylmethyl-5-(2-pyridyl)-1 H -1,4-benzodiazepine -3-yl]-3-(3-methylaminophenyl)urea
R-4-amino-3-(4-chlorophenyl)butanoic acid
Treatment of the behavorial abnormalities associated with fragile X syndrome
Seaside Therapeutics, Inc.
Treatment of type II Leber's Congential Amaurosis
Applied Genetic Technologies Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 250 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2151
Thymoglobulin 9/26/20062152
Galiomedix(Tm) 12/31/2013 RadioMedix, Inc.2153
raloxifene hydrochloride Evista 8/20/20102154
ramucirumab n/a 11/4/2011 Eli Lilly and Company2155
ranagengliotucel-L Glionix(Tm) 5/29/2009 Treatment of astrocytic tumors NovaRx Corporation2156
ranprinase Onconase 1/25/2007 Alfacell Corporation2157
Rapamycin n/a 3/20/2007 OncoImmune, Inc.2158
rapamycin n/a 2/24/2016 DSLP2159
rapamycin n/a 10/20/2016 Lam Therapeutics2160
n/a 2/6/20142161
n/a 6/19/2014 Treatment of pancreatic cancer.
Rabbit anti-human thymocyte globulin (rATG)
Induction treatment to prevent rejection and to minimize maintenance immunosupression in pediatric liver transplant recipients
Children's Hospital of Pittsburgh
radiolabeled somastatin analog
Diagnostic for the management of neuroendocrine tumors
Treatment of hereditary hemorrhagic telangiectasia
Consejo Superior de Investigaciones Cientificas
Treatment of hepatocellular carcinoma
Treatment of malignant mesothelioma
Treatment of tuberous sclerosis complex
Treatment of facial angiofibromas (FA) associated with tuberous sclerosis complex (TSC).
Treatment of pulmonary arterial hypertention
Re188 P2045 somatostatin analog
Treatment of small cell lung cancer
Andarix Pharmaceuticals
Re188 P2045 somatostatin peptide analogue
Andarix Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 251 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2162recilisib sodium Ex-Rad(R) 6/1/2012
2163recilisib sodium Ex-Rad(R) 9/4/2012
2164
n/a 4/30/20142165
n/a 4/29/2014 Abeona Therapeutics2166
n/a 3/3/2015 Pfizer, Inc.2167
n/a 3/25/20132168
n/a 5/11/20092169
Raav-Aat 1/27/20032170
n/a 8/25/2009 Neurologix, Inc.
Prevention of acute radiation syndrome
Onconova Therapeutics, Inc.
Treatment of acute radiation syndrome
Onconova Therapeutics, Inc.
Recombinant AAV9 expressing human alpha-N-acetylglucosaminidase
Mucopolysaccharidosis III-B (Sanfilippo Syndrome Type B)
Abeona Therapeutics LLC
recombinant AAV9 expressing human sulfoglucosamine sulfohydrolase
Treatment of mucopolysaccharidosis type III-A (Sanfilippo Syndrome Type A)
recombinant activated Factor X variant
Treatment of intracerebral hemorrhage
recombinant adeno- associated virus vector AAV2/rh8 expressing human B-hexosaminidase A and B subunits
For the treatment of Sandhoff disease
Nat'l Tay-Sachs & Allied Diseases Association
Recombinant adeno-associated virus (serotype 2) (rAAV2) gene transfer agent expressing RPE65
Treatment of Leber's congential amaurosis
AmpliPhi Biosciences Corporation
recombinant adeno-associated virus alpha 1-antitrypsin vector
Treatment of alpha1-antitrypsin deficiency
Applied Genetic Technologies Corp.
recombinant adeno-associated virus encoded gene for X-linked mammalian inhibitor of apoptosis protein (XIAP)
Treatment of Huntington's disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
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Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2171
n/a 6/24/20082172
n/a 9/28/20162173
n/a 10/29/20152174
n/a 12/29/20152175
n/a 6/16/2015
Recombinant adeno-associated virus retinal pigment epithelium gene vector AAV2-hRPE65v2
Treatment of Leber congenital amaurosis due to RPE65 mutations.
Spark Therapeutics, Inc.
recombinant adeno-associated virus serotype 8 vector encoding human glucose-6-phosphatase-alpha (G6Pase or G6PC)
Treatment of glycogen storage disease type Ia (von Gierke Disease)
Dimension Therapeutics
recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP
Treatment of Angelman Syndrome.
Agilis Biotherapeutics, LLC
recombinant adeno-associated virus serotype AAV8 vector encoding human ornithine transcarbamylase
Treatment of ornithine transcarbamylase deficiency
Dimension Therapeutics
recombinant adeno-associated virus serotype rhesus 74 (rh74) expressing the human CT GalNac transferase (GALGT2) gene, also called the Sda or Cad GAlNac transferase or b4Galnt2, under control of the muscle creatine kinase (MCK) promoter
Treatment of Duchenne muscular dystrophy
Nationwide Children's Hospital
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
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Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2176
n/a 9/19/20162177
n/a 11/16/20152178
n/a 2/4/20112179
n/a 9/1/20162180
n/a 9/1/2016
recombinant adeno-associated virus serotype rhesus 74 expressing the human dysferin gene (AAVrh74.MHCK7.DYSF.DV)
Treatment of Limb Girdle Muscular Dystrophy Type 2B
Nationwide Children's Hospital
recombinant adeno-associated virus vector expressing the cyclic nucleotide gated channel alpha subunit (CNGA3)
Treatment of achromatopsia caused by mutations in the CNGA3 gene
Applied Genetic Technologies Corp.
recombinant adeno-associated virus vector expressing the cyclic nucleotide gated channel beta subunit (rAAV-CNGB3)
Treatment of achromatopsia caused by mutations in the CNGB3 gene.
Applied Genetic Technologies Corporation
recombinant adeno-associated virus vector serotype 9 expressing human iduronate-2-sulfatase
Treatment of mucopolysaccharidosis II, also called Hunter syndrome A.
Research Institute at Nationwide Children's Hospital
recombinant adeno-associated virus vector serotype 9 expressing human N-Sulfoglucosamine Sulfohydrolase
Treatment of mucopolysaccharidosis type III A, also called Sanfilippo syndrome A
Research Institute at Nationwide Children's Hospital
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 254 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2181
n/a 3/25/2013 Treatment of Tay-Sachs disease2182
n/a 8/20/2009 Treatment of Hodgkin lymphoma Affimed GmbH2183
n/a 4/26/2006 Savara Inc.2184
Cethrin 11/18/20052185
n/a 7/29/2008 Baxalta US Inc.2186
n/a 7/29/2008 Baxalta US Inc.2187
recombinant DNA plasmid n/a 1/31/2014 Scancell Ltd.2188
recombinant elafin n/a 3/18/2013 Proteo Biotech AG
recombinant adenovirus vector AAV2/rh8 expressing human B-hexosaminidase A & B subunits
Na't Tay-Sachs & Allied Diseases Association
Recombinant antibody construct against human CD30 and CD16A
Recombinant coagulation factor VIIa
Treatment of diffuse alveolar hemorrhage
recombinant deriative of C3 transferase
Treatment of acute spinal cord injury
Vertex Pharmaceuticals, Inc.
Recombinant disintegrin and metalloprotease with thrombospondin type 1 motifs
Prevention of thrombotic thrombocytopenic purpura including its congential, acquired idiopathic and secondary forms.
Recombinant disintegrin and metalloprotease with thrombospondin type 1 motifs
Treatment of thrombotic thrombocytopenic purpura including its congential, acquired idiopathic, and secondary forms.
Treatment of stage IIb, IIc, III and IV melanoma.
Prevention of inflammatory complications of transthoracic esophagectomy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 255 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2189
n/a 8/18/2005 Henogen S.A.2190
Valortim 2/16/2006 Treatment of anthrax infection PharmAthene, Inc.2191
Recombinant Fusion Protein n/a 7/24/2012 Apogenix GmbH2192
n/a 10/7/20152193
n/a 3/18/2013 Celgene Corporation2194
n/a 3/11/2013 Treatment of B-thalassemia Celgene Corporation
Recombinant Epstein-Barr virus gp350 glycoprotein vaccine
Prevention of post-transplantation lymphoproliferative disorders in pediatric recipients of solid-organ transplantation
Recombinant fully human monoclonal antibody to anthrax protective antigen
Treatment of Myelodysplastic Syndrome
recombinant fusion protein comprising exenatide and XTEN
Treatment of short bowel syndrome.
NAIA Pharmaceuticals, Inc.
recombinant fusion protein consisiting of a modified form of extracellular domain of human Activin receptor IIB
Treatment of myelodysplastic syndrome
recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB (ActRIIB) linked to a human IgG1 Fc domain
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 256 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2195
n/a 5/6/2013 CSL Behring2196
n/a 3/19/20012197
Proxinium 1/28/2005 Viventia Biotech, Inc.2198
n/a 10/13/2009 Apogenix GmbH2199
n/a 8/20/2010 Treatment of Pompe disease.2200
n/a 11/9/2016 Genexine, Inc.2201
Plexcerase(Tm) 12/24/2013 Treatment of Faber disease2202
n/a 5/13/2015 Treatment of hypophosphatasia. AM-Pharma BV
recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP)
Treatment of congenital factor VII deficiency which includes treatment and prophylaxis of bleeding episodes in patients with congenital factor VII deficiency
Recombinant fusion protein of Mycobacterium bovis BCG Hsp65 and HPV16 E7
Treatment of recurrent respiratory papillomatosis (RRP)
StressGen Biotechnologies, Inc. is now Nventa
recombinant fusion protein with a truncated form of the cytotoxic protein Pseudomonas exotoxin
Treatment of Ep-CAM-positive squamous cell carcinoma of the head and neck
Recombinant fusion protein-extracellular portion of CD95 fused to the Fc part of human IgG1
Treatment of glioblastoma multiforme
recombinant glycosylated independent lysosomal targeting (GILT) tagged human acid alpha glucosidase
BioMarin Pharmaceutical, Inc.
recombinant growth hormone fused to hyFc
Treatment of growth hormone deficiency.
recombinant human acid ceramidase
Enzyvant Sciences Ltd.
recombinant human alkaline phosphatase
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 257 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2203
n/a 4/28/20052204
n/a 11/20/2001 Treatment of cystic fibrosis AiroMedica LLC2205
n/a 3/6/1998 Treatment of cystic fibrosis.2206
n/a 8/28/2001 AiroMedica LLC2207
n/a 2/22/2001 Treatment of myasthenia gravis2208
n/a 11/19/2013 Treatment of Pompe Disease2209
n/a 2/2/2006 Chiesi USA, Inc.2210
n/a 4/15/20132211
n/a 7/24/2012 Pfizer, Inc.
Recombinant human alpha 1-antitrypsin
Prevention of bronchopulmonary dysplasia
Arriva Pharmaceuticals, Inc.
recombinant human alpha 1-antitrypsin (rAAT)
recombinant human alpha-1 antitrypsin
PPL Therapeutics (Scotland) Limited
recombinant human alpha-1 antitrypsin (rAAT)
To delay progression of chronic obstructive pulmonary disease resulting from AAT deficiency-mediated emphysema and bronchiectasis
Recombinant Human Alpha-Fetoprotein (rhAFP)
Alpha Cancer Technologies, Inc.
recombinant human alpha-glucosidase conjugated with synthetic bis-mannose-6-phosphate-Man6 glycan
Genzyme, a Sanofi Company
Recombinant human alpha-mannosidase
Treatment of alpha-mannosidosis
recombinant human alpha-N-acetylglucosaminidase
Treatment of mucopolysaccharidosis IIIB (Sanfilippo B syndrome)
Alexion Pharmaceuticals, Inc.
recombinant human anti-GDF-8 monoclonal antibody
Treatment of Duchenne Muscular Dystrophy.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 258 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2212
n/a 4/6/2000 AT III LLC2213
n/a 2/16/20122214
recombinant human biglycan n/a 7/13/20162215
n/a 6/9/2006 Pharming Group N.V.2216
n/a 6/9/20062217
n/a 2/23/1999 Pharming Group N.V.2218
n/a 6/9/2015 OncoImmune, Inc.2219
n/a 8/30/1990 Genentech, Inc.2220
n/a 6/8/2016 Herantis Pharma Plc
Recombinant human antithrombin III
Treatment of antithrombin III dependent heparin resistance requiring anticoagulation.
recombinant human beta-glucuronidase
Treatment of mucopolysaccharidosis VII (MPS VII, Sly Syndrome)
Ultragenyx Pharmaceutical, Inc.
Treatment of Duchenne Muscular Dystrophy
Tivorsan Pharmaceuticals, Inc.
Recombinant human C1 inhibitor
Prevention and/or treatment of delayed graft function after solid organ transplantation
Recombinant human C1 inhibitor
Treatment of capillary leakage syndrome
Pharming Technologies B.V.
Recombinant human C1-esterase inhibitor
Prophylactic treatment of angioedema caused by hereditary or acquired C1-esterase inhibitor deficiency.
recombinant human CD24FcIg (humanized fusion protein consisting of the extracellular domain of CD24 linked to IgG1 Fc domain
Prevention of graft-versus-host disease
Recombinant human CD4 immunoglobulin G
Treatment of AIDS resulting from infection with HIV-1.
recombinant human cerebral dopamine neurotrophic factor
Treatment of amyotrophic lateral sclerosis
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 259 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2221
n/a 7/13/19982222
n/a 3/17/20142223
n/a 8/18/2014 SciVac Ltd.2224
n/a 8/18/2014 SciVac Ltd.2225
n/a 2/21/2002 EntreMed, Inc.2226
n/a 8/13/2001 EntreMed, Inc.2227
n/a 5/26/2011 Treatment of mutliple myeloma2228
n/a 11/30/2012 Pfizer, Inc.2229
n/a 10/4/2007
Recombinant human Clara Cell 10kDa protein
Prevention of neonatal bronchopulmonary dysplasia in premature neonates with respiratory distress syndrome.
Therabron Therapeutics, Inc.
recombinant human collagen alpha-1 (VII) chain homo-trimer (rC7)
Treatment of dystrophic epidermolysis bullosa
Shire Human Genetic Therapies, Inc.
recombinant human deoxyribonuclease I
Treatment of graft-vs-host disease
recombinant human deoxyribonuclease I (DNase I)
Prevention of graft-vs-host disease
recombinant human endostatin protein
Treatment of metastatic melanoma
Recombinant human endostatin protein
Treatment of neuroendocrine tumors
recombinant human erythropoietin (rHuEPO)
XTL Biopharmaceuticals, Ltd
Recombinant Human Factor VIIa Variant
Routine prophylaxis to prevent bleeding episodes in patients with hemophilia A and B patients with inhibitors
Recombinant human fibrinogen
Treatment of bleeding in patients deficient in fibrinogen
Pharming Technologies B.V.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 260 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2230
n/a 1/27/2015 TargaZyme, Inc.2231
Galaczym 12/12/2011 ACE BioSciences A/S2232
Recombinant human gelsolin n/a 3/6/19952233
Recombinant human gelsolin n/a 1/12/19942234
n/a 3/22/20102235
n/a 10/31/2012 Savara Inc.2236
n/a 12/3/2012 Jade Therapeutics LLC2237
Tbd 9/20/2000
recombinant human fucosyltransferase VI [ FTVI ] + GDP-fucose
To increase the activity of regulatory T-cells (Tregs) used in the prevention of graft versus host disease in patients undergoing hematopoietic stem cell transplantation
recombinant human galactocerebrosidase (rhGALC);
Treatment of globoid cell leukodystrophy (Krabbe Disease)
Treatment of acute and chronic respiratory symptoms of bronchiectasis.
BioAgeis, Therapeutics, Inc.
Treatment of the respiratory symptoms of cystic fibrosis.
BioAegis Therapeutics, Inc.
Recombinant human glutamic acid decarboxylase 65KDa isoform
Treatment of Type I diabetes with residual beta cell function
Diamyd Therapeutics AB
recombinant human GM-CSF, molgramostim
Treatment of pulmonary alveolar proteinosis
recombinant human gowth hormone in an ocular delivery system
Treatment of persistent corneal epithelial defects
Recombinant human highly phosphorylated acid alpha-glucosidase
For enzyme replacement therapy in patients with all subtypes of glycogen storage disease type II (GSDII, Pompe Disease)
Novazyme Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 261 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2238
n/a 4/11/20012239
n/a 4/22/2015 aTyr Pharma, Inc.2240
Oncohist 10/20/20082241
n/a 12/14/20092242
Pv802 2/16/2000 GroPep Pty Ltd.2243
n/a 6/15/1999 Insmed, Inc.2244
n/a 10/20/19972245
Hemamax 8/22/2016 Neumedicines, Inc.2246
n/a 10/4/2005 Zymo Genetics, Inc
Recombinant human highly phosphorylated alpha-L-iduronidase (rhHP-IDUA)
Enzyme replacement therapy in patients with all subtypes of Mucopolysaccharidosis I.
Novazyme Pharmaceuticals, Inc.
recombinant human histidyl tRNA synthetase
Treatment of facioscapulohumeral muscular dystrophy
recombinant human histone H1.3; recombinant human N-bis-met-histone H1.3
Treatment of acute myeloid leukemia
Xenetic Biosciences Plc
recombinant human IgG1 monoclonal antibody
Treatment of X-linked hypophosphatemia (formerly known as vitamin D-resistant rickets)
Ultragenyx Pharmaceutical, Inc.
Recombinant human insulin-like growth factor-I
Treatment of short-bowel syndrome as a result of resection of the small bowel or as a result of congenital dysfunction of the intestines.
Recombinant human insulin-like growth factor-I/insulin-like growth factor binding protein-3
Treatment of major burns that require hospitalization.
Recombinant human interleukin-12
Treatment of renal cell carcinoma.
Genetics Institute, Inc.
recombinant human interleukin-12
Treatment of acute radiation syndrome
Recombinant Human Interleukin-21 (rIL-21)
Treatment of stage II (T4), III or IV malignant melanoma.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 262 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2247
n/a 12/20/19992248
n/a 9/2/20102249
Cholestrase 7/14/20052250
n/a 1/23/2006 ThromboGenics Ltd2251
Mubodina(R) 6/7/2011 Adienne S.A2252
Mubodina 2/4/2009 Adienne S.A.2253
n/a 2/4/2009 Adienne S.A.2254
n/a 6/18/2012
Recombinant human keratinocyte growth factor
Reducing the incidence and severity of radiation-induced xerostomia.
Swedish Orphan Biovitrum AB (publ) (SOBI)
recombinant human lecithin:cholesterol acyltransferase (rhLCAT)
Treatment of LCAT deficiency syndromes
AlphaCore Pharma, LLC
Recombinant human lysosomal acid lipase or cholesteryl ester hydrolase
Treatment of lipase deficiencies, including Wolman Disease and cholesteryl ester storage disease
Lysosomal Acid Lipase, LLC
Recombinant human microplasmin
Treatment of peripheral arterial occlusion
recombinant human minibody against complement component
Treatment of atypical hemolytic uremic syndrome associated with an inherited abnormality of the complement system.
recombinant human minibody against complement component C5
Treatment of primary membranoproliferative glomerulonephritis
recombinant human minibody against complement component C5 fused with RGD-motif
Prevention of ischemia/reperfusion injury associated with solid organ transplantation.
recombinant human monoclonal antibody against activin receptors type II
Treatment of inclusion body myositis
Novartis Pharmaceuticals Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 263 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2255
n/a 10/6/20162256
n/a 8/19/20142257
Mycograb 9/16/2002 Treatment of invasive candidiasis2258
n/a 9/21/2015 EMD Serono, Inc.2259
n/a 10/25/2013 Treatment of multiple myeloma Patrys Ltd.2260
n/a 3/5/20132261
n/a 8/8/2013 Dompe s.p.a.2262
n/a 4/16/1999 Genentech, Inc.
Recombinant human monoclonal antibody against human complement component C5a
Treatment of acute graft versus host disease
Alexion Pharmaceuticals, Inc.
recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor
Treatment of pigmented villonodular synovitis, including giant cell tumor of the tendon sheath
Novartis Pharmaceuticals Corp
recombinant human monoclonal antibody to hsp90
Novartis Pharmaceuticals Corp.
recombinant human monoclonal IgG1 antibody against programmed death ligand-1 (anti-PD-L1)
Treatment of merkel cell carcinoma.
recombinant human monoclonal IgM antibody targeting glucose regulated protein 78
recombinant human Naglu- insulin-like growth factor II
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B)
Shire Human Genetic Therapies, Inc.
recombinant human nerve growth factor
Treatment of retinitis pigmentosa
Recombinant human nerve growth factor
Treatment of HIV-associated sensory neuropathy.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 264 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2263n/a 6/23/2014 Dompe s.p.a.
2264n/a 12/9/2003 Treatment of cystic fibrosis Dyax Corporation
2265n/a 8/26/2014 Treatment of myelofibrosis Promedior, Inc.
2266
n/a 2/17/2012 Promedior, Inc.2267
n/a 2/1/20072268
Porphozyme 9/9/2002 Zymenex A/S2269
n/a 7/11/2002 Zymenex A/S2270
n/a 12/10/20082271
n/a 1/13/20162272
n/a 3/22/2010 SuppreMol GmbH2273
n/a 6/23/2014
recombinant human nerve growth factor
Treatment of neurotrophic keratitis
recombinant human neutrophil inhibitor (hNE)
recombinant human Pentraxin-2
recombinant human Pentraxin-2; recombinant human Serum Amyloid P
Treatment of idiopathic pulmonary fibrosis.
Recombinant human platelet derived growth factor BB
Treatment of osteonecrosis of the jaws
Luitpold Pharmaceuticals, Inc.
recombinant human porphobilinogen deaminase
Treatment of acute intermittent porphyria attacks
recombinant human porphobilinogen deaminase, erythropoetic form
Treatment of acute intermittent porphyria preventing attacks
recombinant human proinsulin (Including rhPI-Methionine)
Treatment of retinitis pigmentosa
ProRetina Therapeutics, S.L.
recombinant human serum albumin human growth hormone
Treatment of growth hormone deficiency
TEVA Branded Pharmaceutical Products R&D, Inc.
Recombinant Human soluble Fc-gamma Receptor IIb
Treatment of idiopathic thrombocytopenic purpura
recombinant human surfactant protein D
Prevention of bronchopulmonary dysplasia
Airway Therapeutics LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 265 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2274
n/a 9/29/1997 Genentech, Inc.2275
n/a 4/3/20092276
n/a 6/18/20082277
n/a 6/7/2010 NeuroNova AB2278
n/a 2/11/20152279
n/a 7/16/2015 Treatment of gastric cancer Gilead Sciences, Inc.2280
n/a 1/22/2015 AbbVie, Inc.2281
n/a 4/12/2013 Treatment of sickle cell disease
Recombinant human thrombopoietin
For use in accelerating platelet recovery in patients undergoing hematopoietic stem cell transplantation.
recombinant human type I pancreatic elastase
Prevention of arteriovenous fistula and arteriovenous graft failure in patients with chronic kidney disease (CKD) who are receiving hemodialysis or preparing for hemodialysis
Proteon Therapeutics, Inc.
recombinant human type VII collagen
Treatment of hereditary dystrophic epidermolysis bullosa (DEB)
David T. Woodley, MD and Mei Chen, MD
recombinant human vascular endothelial growth factor
Treatment of amyotrophic lateral sclerosis
recombinant humanized anti-interleukin 13 (IL-13) monoclonal antibody
Treatment of eosinophilic esophagitis
Receptos Services, LLC
recombinant humanized anti-matrix metalloprotein 9 (MMP9) monoclonal antibody IgG4
recombinant humanized anti-tau antibody
Treatment of progressive supranuclear palsy
recombinant humanized IgG1k monoclonal antibody to human invariant T cell receptor (iTCR)
NKT Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 266 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2282
n/a 1/11/20162283
n/a 3/22/1999 Biogen, Inc.2284
n/a 3/22/1999 Biogen, Inc.2285
n/a 10/14/1998 Biogen, Inc.2286
n/a 2/3/1998 Biogen, Inc.2287
n/a 2/18/1998 Biogen, Inc.2288
n/a 5/14/2014 Genentech, Inc.2289
n/a 4/18/2011
recombinant humanized IgG4 monoclonal antibody that binds human colony stimulating factor 1 receptor
Treatment of pigmented villonodular synovitis and tenosynovial giant cell tumor
Five Prime Therapeutics, Inc.
Recombinant humanized MAb 5c8
Prevention of rejection of solid organ transplants.
Recombinant humanized MAb 5c8
Prevention of rejection of pancreatic islet cell transplants.
Recombinant humanized MAb 5c8
Prevention and treatment of Factor VIII/Factor IX inhibitors in patients with hemophilia A or B.
Recombinant humanized monclonal antibody 5c8
Treatment of immune thrombocytopenic purpura.
Recombinant humanized monoclonal antibody 5c8
Treatment of systemic lupus erythematosus.
recombinant humanized monoclonal antibody of the immunoglobulin G1 subclass directed against colony stimulating factor-1 receptor expressed on macrophages
Treatment of pigmented villonodular synovitis and tenosynovial giant cell tumor
recombinant IgA protease of bacterium Heamophilus influenzae
Treatment of immunoglobulin A nephropathy (IgAN, Berger's disease).
Shire Human Genetics Therapies
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 267 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2290
n/a 2/11/20022291
n/a 2/11/20022292
n/a 11/23/2010 Dermadis SA2293
n/a 5/19/2014 Ocugen, Inc.2294
n/a 11/28/1994 Amgen, Inc.2295
n/a 2/10/2015 Treatment of AL amyloidosis2296
n/a 7/14/2015 Pfizer, Inc.
Recombinant immunotoxin combining the functional components of antibody against mesothelin and the enzymatic activity of a pseudomonas exotoxin
Treatment of epithelial ovarian cancer
National Institutes of Health
Recombinant immunotoxin combining the functional components of antibody against mesothelin and the enzymatic activity of a pseudomonas exotoxin
Treatment of malignant mesothelioma
National Institutes of Health
recombinant kallikrein inhibitor
Treatment of Netherton Syndrome.
recombinant lens epithelium derived growth factor 1-326
Treatment of retinitis pigmentosa
Recombinant methionyl brain-derived neurotrophic factor
Treatment of amyotrophic lateral sclerosis.
recombinant monoclonal antibody to human serum amyloid P component
Glaxo Group Limited, England d/b/a GlaxoSmithKline
recombinant multimerized human IgG1 Fc
Treatment of chronic inflammatory demyelinating polyneuropathy (CIDP).
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 268 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2297
n/a 12/8/2014 ARCA Biopharma, Inc.2298
n/a 11/17/20142299
n/a 1/6/2016 Immune Design Corp.2300
Tauferon 1/25/2005 PEPGEN Corporation2301
n/a 3/29/2006 Y's Therapeutics, Inc.2302
n/a 7/26/2007 Y's Therapeutics, Inc.2303
n/a 5/10/2016 Akari Therapeutics Plc2304
n/a 4/12/1999 Schering Corporation2305
n/a 11/15/1993 Genetic Therapy, Inc.
recombinant nematode anticoagulant protein c2 (rNAPc2)
Treatment of viral hemorrhagic fever post-exposure to Ebola virus (treatment of Ebola)
recombinant neuroglobin (rNgb) containing mutations H64Q/C46G/C55S/C120S
Treatment of patients with carbon monoxide poisoning
University of Pittsburgh
recombinant NY-ESO-1 protein mixed with glucopyranosyl lipid A
Treatment of soft tissue sarcoma.
recombinant ovine interferon tau
Treatment of pediatric multiple sclerosis
Recombinant P-Selectin glycoprotein ligand
Prevention of delayed graft function in renal transplant patients
Recombinant P-selectin glycoprotein ligand-immunoglobulin Ig
Prevention of ischemia reperfusion injury in all solid organ transplants
Recombinant protein inhibitor of complement factor 5
Treatment of Guillain-Barre Syndrome.
Recombinant replication deficient adenovirus vector carrying human p53 gene
Treatment of primary ovarian cancer.
Recombinant retroviral vector - glucocerebrosidase
For use as enzyme replacement therapy for patients with types I, II, or III Gaucher disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 269 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2306
n/a 3/29/1991 Treatment of cystic fibrosis. Amgen, Inc.2307
n/a 3/23/1989 Genentech, Inc.2308
n/a 5/2/20032309
n/a 12/13/20102310
n/a 6/24/2005 Treatment of cystic fibrosis Aerovance, Inc.2311
Recombinant urate oxidase n/a 10/11/20002312
n/a 6/8/2016
Recombinant secretory leucocyte protease inhibitor
Recombinant soluble human CD4 (rCD4)
Treatment of AIDS in patients infected with HIV virus.
Recombinant T-cell receptor ligand
Treatment of multiple sclerosis patients who are both HLA-DR2 positive and autoreactive to myelin oligodendrocyte glycoprotein residues 35-55
Artielle ImmunoTherapeutics, Inc.
recombinant thymidine phosphorylase encapsulated with autologous erythrocytes
Treatment of mitochondrial neurogastrointestinal encephalomyopathydue to thymidine phosphorylase deficiency.
St. George's University of London
Recombinant truncated SPINT2 protease inhibitor
Prophylaxis of chemotherapy-induced hyperuricemia.
Sanofi-Synthelabo Research
recombinant, adeno-associated virus serotype 2 vector carrying linear single-stranded AADC mRNA cDNA
Treatment of aromatic L-amino acid decarboxylase deficiency.
Agilis Biotherapeutics, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 270 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2313
n/a 7/25/2016 Treatment of Friedreich's Ataxia2314
n/a 8/25/20162315
n/a 6/29/20162316
n/a 5/26/2016 Treatment of glioma2317
Reduced L-glutathione Cachexon 2/14/1994
recombinant, adeno-associated virus serotype 5 vector, containing the transgene, which encodes for the human protein, frataxin
Agilis Biotherapeuties, LLC
recombinant, fully-human, IgG4 immunoglobulin with antigen-binding specificity for human vascular adhesion protein-1 (VAP-1)
Treatment of primary sclerosing cholangitis.
Acorda Therapeutics, Inc.
recombinant, glycoenfineered humanized IgG1 kappa monoclonal antibody directed against Fibroblast Growth Factor Receptor 2b
Treatment of gastric cancer including cancer of the gastroesophageal junction.
Five Prime Therapeutics, Inc.
recombinant, non-pathogenic poliovirus:rhinovirus chimera that consists of the genome of the live attenuated poliovirus serotype 1 vaccine with its cognate IRES element replaced with that of HRV2
Darell D. Bigner, MD, PhD
Treatment of AIDS-associated cachexia.
Telluride Pharmaceutical Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 271 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2318
refanalin n/a 5/25/20102319
regorafenib Stivarga 6/4/20152320
REMUNE HIV 1 n/a 2/14/20142321
reparixin n/a 9/25/2012 Dompe S.p.A.2322
reparixin n/a 1/27/2003 Dompe S.p.A.2323
Advexin 1/27/20032324
n/a 5/3/2013 MeiraGTx, LLC
To improve renal function and prevent delayed graft function following renal transplantation
Angion Biomedica Corporation
Treatment of hepatocellular carcinoma.
Bayer HealthCare Pharmaceuticals, Inc.
Treatment of pediatric HIV/AIDS (age through 16 years)
Immune Response BioPharma, Inc.
Prevention of graft loss in pancreatic islet transplantation
Prevention of delayed graft function in solid organ transplant
Replication defective recombinant adenovirus serotype 5 vector carrying the p53 gene in its E1 deleted region
Treatment of head and neck cancer
Introgen Therapeutics, Inc.
replication-deficient recombinant serotype 2 adeno-associated viral vector containing hAQP1 cDNA
Treatment of symptoms of Grade 2 and Grade 3 late xerostomia from parotid gland hypofunction caused by radiotherapy for cancer of the oral cavity.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 272 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2325
3/2/2010 Aduro BioTech, Inc.2326
4/30/2010 Aduro BioTech, Inc.2327
Gvax Pancreas 3/1/2010 Treatment of pancreatic cancer. Aduro BioTech, Inc.2328
H.P. Acthar Gel 6/28/20132329
resiniferatoxin n/a 5/13/20032330
reslizumab Cinquil 1/12/20112331
reslizumab n/a 12/19/2007 Teva Respiratory, LLC2332
resminostat n/a 6/29/2011 4SC AG2333
resminostat n/a 9/16/2011 4SC AG
Replication-incompetent GM-CSF-expressing gene-modified allogeneic acute myeloid leukemia cancer cell lines
Gvax Acute Myeloid Leukemia
Treatment of acute myeloid leukemia
Replication-incompetent GM-CSF-expressing gene-modified allogeneic chronic myeloid leukemia cancer cell lines
Gvax Chronic Myeloid Leukemia
Treatment of chronic myeloid leukemia
Replication-incompetent GM-CSF-expressing gene-modified allogeneic pancreatic cancer cell lines
repository corticortropin injection
Treatment of amyotrophic lateral sclerosis
Questor Pharmaceuticals, Inc.
Treatment of intractable pain at end-stage disease
Sorrento Theraeputics, Inc.
Treatment of hypereosinophilic syndrome
Teva Pharmaceuticals, Inc.
Treatment of children with eosinophilic esophagitis
Treatment of hepatocellular carcinoma
Treatment of Hodgkin's lymphoma.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 273 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2334
Hypermune Rsv 9/27/1990 MedImmune, Inc.2335
Retinol palmitate n/a 8/8/20162336
n/a 4/29/20022337
n/a 3/26/2010 Treatment of malaria DesignMedix2338
Revimmune n/a 2/18/20112339
Reviparin sodium Clivarine 6/18/2001 Abbott2340
Reviparin sodium Clivarine 6/18/2001 Abbott2341
revusiran n/a 5/18/20152342
rh-microplasmin, ocriplasmin Jetrea 3/16/2004 ThromboGenics Inc.
Respiratory Syncytial Virus Immune Globulin (human)
Treatment of respiratory syncytial virus lower respiratory tract infections in hospitalized infants and young children.
Prevention of bronchopulmonary dysplasia.
Phillipp Heinrich Novak, DVM, MSc
retroviral gamma-c cDNA containing vector
Treatment of X linked severe combined immune deficiency disease
AVAX technologies, Inc.
Reversal agent linked to chloroquine-like moiety
Treatment of autoimmune hemolytic anemia.
Accentia Biopharmaceuticals
Treatment of deep vein thrombosis which may lead to pulmonary embolism in pediatric patients
Long-term treatment of acute deep vein trombosis with or without pulmonary embolism in pregnant patients
Treatment of transthyretin amyloidosis
Alnylam Pharmaceuticals, Inc.
Adjunct to surgery in cases of pediatric vitrectomy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 274 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2343
rhIGF-I/rhIGFBP-3 Somatokine 12/9/2003 Insmed, Inc.2344
ribavirin Virazole 4/12/19912345
ribavirin elaidate n/a 9/2/20112346
Ribocross 11/8/20162347
ricin vaccine Rivax(R) 1/7/2011 Prevention of ricin intoxication. Soligenix, Inc.2348
Rifabutin n/a 12/18/1989 Pfizer Inc.2349
Rifalazil n/a 4/13/19992350
Rifapentine Priftin 6/9/1995
Treatment of extreme insulin resistance syndromes (type A, Rabson-Mendenhall syndrome, Leprechaunism, Type B syndrome)
Treatment of hemorrhagic fever with renal syndrome
Valeant Pharmaceuticals International
Treatment of follicular, medullary, and anaplastic thyroid carcinoma and metastatic or locally advanced papillary thyroid cancer
Translational Therapeutics, Inc.
riboflavin-vitamin E-TPGS topical solution
Treatment of keratoconus in patients undergoing crosslinking procedure
Institute of Refractive & Ophthalmic Surgery
Treatment of disseminated Mycobacterium avium complex disease.
Treatment of pulmonary tuberculosis.
PathoGenesis Corporation
Treatment of Mycobacterium avium complex in patients with AIDS.
Hoechst Marion Roussel , Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 275 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2351
Rifapentine Priftin 3/12/19962352
rigosertib n/a 3/16/2012 Treatment of ovarian cancer2353
rigosertib n/a 3/18/2011 Treatment of pancreatic cancer.2354
rigosertib n/a 9/3/20092355
RII retinamide n/a 5/6/19932356
rilonacept Arcalyst 1/9/20132357
rilotumumab n/a 6/18/2012 Amgen, Inc.2358
riluzole n/a 2/23/20162359
Riluzole Rilutek 10/15/19962360
riluzole oral suspension Teglutik 9/15/2016 Italfarmaco SpA
Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.
Hoechst Marion Roussel, Inc.
Onconova Therapeutics, Inc.
Onconova Therapeutics, Inc
Treatment of myelodysplastic syndromes
Onconova Therapeutics, Inc.
Treatment of myelodysplastic syndromes.
Sparta Pharmaceuticals, Inc.
Treatment of familial Mediterranean fever
Philip J Hashkes, MD, MSc.
Treatment of gastric cancer including gastroesophageal junction adenocarcinoma
Treatment of spinocereballar ataxia.
Biohaven Pharmaceutical Holding Company, Ltd.
Treatment of Huntington's disease.
Rhone-Poulenc Rorer Pharmaceuticals, Inc.
Treatment of amyotrophic lateral sclerosis (ALS).
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 276 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2361
riluzole orally disolving tablet n/a 11/30/20162362
rindopepimut n/a 11/19/20072363
riociguat Adempas 7/24/2014 Treatment of systemic sclerosis2364
risankizumab n/a 11/29/2016 AbbVie Inc.2365
rituximab Rituxan(R); Mabthera(R) 2/23/2015 Genentech, Inc.2366
rituximab n/a 11/9/20162367
rituximab Rituxan 3/12/2002 Genentech, Inc.2368
Rituxin Sc 8/22/2016 Genentech, Inc.2369
n/a 8/22/2016 Genentech, Inc.2370
Rituximab/hyaluronidase Rituxan 9/7/2016 Genentech, Inc.2371
rociletinib n/a 5/14/2013 Clovis Oncology, Inc.2372
rofecoxib Vioxx 3/16/2004 MERCK & Co., Inc.
Treatment of amyotrophic lateral sclerosis (ALS)
Biohaven Pharmaceutical Holding Company, Ltd
Treatment of EGFRvIII-expressing glioblastoma multiforme
Celldex Therapeutics, Inc.
Bayer HealthCare Pharmaceuticals
Treatment of pediatric Crohn's disease
Treatment of pemphigus vulgaris.
Treatment of Rasmussen's encephalitis
Keck Graduate Institute of Applied Life Sciences
Treatment of immune thrombocytopenic purpura
rituximab and recombinant human hyaluronidase
Treatment of chronic lymphocytic leukemia (CLL)
rituximab and recombinant human hyaluronidase
Treatment of follicular lymphoma
Treatment of diffuse large B-cell lymphoma
Treatment of non-small cell lung cancer and mutations in the epidermal growth factor receptor
Treatment of juvenile rheumatoid arthritis
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 277 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2373
romiplostim n/a 10/31/2007 Amgen Inc.2374
roneparstat n/a 3/19/2015 Treatment of multiple myeloma2375
n/a 5/19/2016 EMEK, Inc.2376
Rose Bengal Disodium n/a 12/21/20062377
Roseburia hominis Rosburix 8/4/20142378
rovalpituzumab tesirine n/a 12/22/2015 AbbVie Inc.2379
rozrolimupab n/a 9/13/2010 Symphogen A/S2380
rsATP7A cDNA n/a 1/10/2014 Treatment of Menkes disease Stephen G. Kaler, MD2381
rSP-C lung surfactant Venticute 4/3/20002382
rSP-C surfactant Venticute 9/18/2006 Altana Pharma
Treatment of thrombocytopenia associated with myelodysplasia syndrome
Sigma-Tau Research Switerland S.A.
ropidoxuridine (5-iodo-2-pyrimidinone-2'-deoxyribose)
Radiosensitizer to be used during radiation treatment of glioblastoma multiforme.
Treatment of metastatic melanoma
Provectus Pharmaceuticals, Inc.
Treatment of ulcerative colitis in pediatric patients age 0 through 16 years
4D Pharma Research Ltd
Treatment of small cell lung cancer
Treatment of primary immune thrombocytopenia.
Treatment of adult respiratory distress syndrome.
Byk Gulden Pharmaceuticals
For use in patients with pneumonia or aspiration of gastric contents leading to intubation, mechanical ventilation, and severe oxygen impairment
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 278 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2383
rt-PA Activase 10/20/2014 Treatment of plastic bronchitis2384
rubitecan n/a 7/17/2002 SuperGen, Inc.2385
rucaparib n/a 7/31/2012 Treatment of ovarian cancer Clovis Oncology, Inc.2386
ruxolitinib Jakafi 11/3/2016 Incyte Corporation2387
Ruxolitinib n/a 7/18/2016 Incyte Corporation2388
ruxolitinib Jakafi 8/16/2013 Treatment of pancreatic cancer Incyte Corporation2389
ruxolitinib phosphate Jakafi 3/22/2010 Incyte Corporation2390
rVIIa-FP n/a 12/22/2011 CSL Behring2391
n/a 3/14/2002 Treatment of multiple myeloma2392
2/14/2011 Cerus Corporation2393
S-adenosylmethionine n/a 4/30/1998 Treatment of AIDS-myelopathy. Genopia USA, Inc.
Kathleen A Stringer, PharmD, FCCP - Professor
Treatment of pediatric patients infected with human immunodeficiency virus and acquired immunodeficiency syndrome
Treatment of graft versus host disease
Treatment of acute lymphoblastic leukemia.
Treatment of essential thrombocythemia
Treatment and prophyllaxis of bleeding episodes in patients with congenital hemophilia and inhibitors to coagulation factor VIII or IX
S(-)-3-[3-amino-phthalimido]-glutaramide
EntreMed Incorporated
S-59 treated FFP (plasma treated with amotosalen hydrochloride & ultraviolet A light
Intercept Blood System For
Treatment of thrombotic thrombocytopenic purpura
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 279 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2394
n/a 8/29/2016 TrioxBio, Inc.2395
S-nitrosoglutathione n/a 5/12/20092396
S-nitrosoglutathione n/a 12/28/20122397
n/a 10/20/2009 Treatment of pancreatic cancer MBiotec GmBH2398
n/a 11/16/2015 Treatment of C3 glomerulopathy.
S-ethylisothiourea diethylphosphate
Treatment of symptomatic intradialytic hypotension, including symptomatic hypotension in the immediate post-dialytic period.
Management of cystic fibrosis patients to improve airway clearance and to improve or stabilize pulmonary function
N30 PHARMAceuticals, LLC
Treatment of severe preeclampsia
Salupont Consulting Ltd
S-[2,3-bispalmitoyloxy-(2R)-propyl]-cysteinly-GNNDESNISFKEK
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide
Amyndas Pharmaceuticals
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 280 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2399
n/a 10/9/20142400
sacituzumab govitecan n/a 5/29/2014 Treatment of pancreatic cancer. Immunomedics, Inc.2401
sacituzumab govitecan n/a 11/27/2013 Immunomedics, Inc.2402
salicylic acid 6% n/a 2/17/2012 Orenova Group, LLC2403
salirasib n/a 12/18/2006 Treatment of pancreatic cancer.2404
n/a 10/29/2009 Lithera, Inc.2405
saposin C n/a 2/3/20152406
Sar9, Met(O2)11-Substance P Homspera 3/16/20112407
sarcosine n/a 10/12/2011
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide)
Treatment of paroxysmal nocturnal hemoglobinuria (PNH)
Amyndas Pharmaceuticals
Treatment of small cell lung cancer
Treatment of rare congenital ichthyoses.
Kadmon Corporation, LLC
salmeterol xinafoate/fluticasone propionate
Treatment of symptomatic exophthalmos associated with thyroid related eye disease
Treatment of glioblastoma multiforme
Bexion Pharmaceuticals, LLC
Treatment of idiopathic pulmonary fibrosis
ImmuneRegen BioSciences, Inc.
Treatment of obsessive compulsive disorder in pediatric patients (0 to 16 years of age)
Guochuan Emil Tsai, MD, PhD
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 281 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2408
sargramostim Leukine 11/9/2016 Sanofi Genzyme2409
sarizotan n/a 7/7/2015 Treatment of Rett syndrome.2410
SC-1 monoclonal antibody n/a 11/12/2003 Patrys Limited2411
SDF-1 (108) Lysine Dlmer n/a 7/7/20052412
n/a 4/15/2013 TransDerm, Inc.2413
Secalciferol Osteo-D 7/26/19932414
n/a 6/30/1992 Synergen, Inc.2415
n/a 9/5/2013 BioLineRx, Ltd.
Treatment of individuals acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome)
Newron Pharmaceuticals US, Inc.
Treatment of patients with CD55 (sc-1) positive gastric tumors
Treatment of osteogenic sarcoma
Chemokine Therapeutics Corporation
sdTD-K6a.513a.12; small interfering RNA composed of 2 strands of hybridized RNAs
Treatment of pachyonychia congenita
Treatment of familial hypophosphatemic rickets.
Teva Pharmaceuticals USA
Secretory leukocyte protease inhibitor
Treatment of bronchopulmonary dysplasia.
selective antagonist of the chemokine receptor type 4
Treatment of acute myeloid leukemia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 282 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2416
n/a 3/28/20112417
n/a 3/3/20162418
n/a 8/19/20142419
n/a 6/4/19962420
n/a 9/27/2013 Hannah's Hope Fund2421
selinexor n/a 1/5/2015 Treatment of multiple myeloma2422
selinexor n/a 7/7/2016 Treatment of soft tissue sarcoma2423
n/a 5/14/20142424
selisistat n/a 12/7/2009 Siena Biotech SpA
selective deacylglycerol acyltransferase 1 inhibitor
Treatment of hypertriglyceridemia in te setting of Type I hyperlipoproteinemia, also known as Familial Chylomicronemia Syndrome
Novartis Pharmaceuticals Corporation
selective inhibitor of fungal CYP51
Treatment of coccidioidomycosis.
Viamet Pharmaceuticals, Inc.
selective inhibitor of fungal lanosterol demethylase
Treatment of cryptococcal meningitis
Viamet Pharmaceuticals, Inc.
Selective inhibitor of polymorphonuclear leukocyte (PMN) elastase
Therapeutic management of patients with lung disease attributable to cystic fibrosis.
DuPont Pharmaceuticals Company
self-complimentary adeno-associated virus vector, serotype 9, packaging the full lenght GAN gene in the viral capsid
Treatment of Giant Axonal Neuropathy
Karyopharm Therapeutics, Inc.
Karyopharm Therapeutics, Inc.
Selinexor; (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N-(pyrazin-2-yl)acrylohydrazide
Treatment of acute myeloid leukemia
Karyopharm Therapeutics, Inc.
Treatment of Huntington's disease
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 283 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2425
selumetinib n/a 5/10/20162426
Seneca Valley virus n/a 8/22/2008 Neotropix, Inc.2427
Imuvert 9/7/1988 Cell Technology, Inc.2428
setmelanotide n/a 9/21/20152429
setmelanotide n/a 4/4/20162430
sevuparin n/a 3/17/2015 Treatment of sickle cell disease Dilaforette AB2431
Short chain fatty acid enema Colomed 8/19/19972432
Short chain fatty acid solution Colomed 5/29/1990 Richard I. Breuer2433
sildenafil Revatio 7/28/2011 Pfizer, Inc.2434
Legalon Sil 2/17/2012 Meda AB
Adjuvant treatment of patients with stage III or stage IV differentiated thyroid cancer.
AstraZeneca Pharmaceuticals LP
Treatment of neuroendocrine tumors
Serratia marcescens extract (polyribosomes)
Treatment of primary brain malignancies.
Treatment of Prader-Willi Syndrome.
Rhythm Metabolics, Inc.
Treatment of pro-opiomelanocortin (POMC) deficiency due to mutations in the POMC gene
Rhythm Pharmaceuticals, Inc.
Treatment of chronic radiation proctitis.
Richard I. Breuer, M.D.
Treatment of the active phase of ulcerative colitis with involvement restricted to the left side of the colon.
Treatment of pediatric (defined as children less than 17 years of age) pulmonary arterial hypertension
Silibinin-C-2',3-dihydrogensuccinate, disodium salt
Prevention of recurrent hepatitis C in liver transplant patients
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 284 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2435
Legalon(R) Sil 9/11/20142436
Flammacerium 11/17/19992437
simtuzumab n/a 1/5/2015 Gilead Sciences, Inc.2438
n/a 7/17/2014 Treatment of Alport syndrome2439
siponimod n/a 7/10/2014 Treatment of dermatomyositis2440
Siponimod n/a 11/26/2013 Treatment of polymyositis2441
sirolimus n/a 3/18/20132442
sirolimus n/a 6/25/20142443
sirolimus n/a 11/17/20112444
sirolimus n/a 6/21/2016 Treatment of beta-thalassemia.
silibinin-C-2',3-dihydrogensuccinate, disodium salt
Treatment of amatoxin poisoning, which includes the prevention and treatment of amatoxin induced hepatic failure
Meda Pharmaceuticals, Inc.
Silver sulfadiazine and cerium nitrate
Treatment of patients with severe dermal burns
Alliance Pharmaceuticals Ltd
Treatment of primary sclerosing cholangitis
single stranded, chemically modified oligonucleotide that binds to and inhibits the function of micro RNA-21
Regulus Therapeutics, Inc.
Novartis Pharmaceuticals Corporation
Novartis Pharmaceuticals Corporation
Treatment of pachyonychia congenita
Palvella Therapeutics LLC
Treatment of lymphangioleiomyomatosis
LAM Therapeutics, Inc.
Treatment of lymphangioleiomyomatosis
Cote Orphan Consulting, LLC
Rare Partners srl Impresa Sociale
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 285 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2445
sirolimus n/a 11/4/2011 Santen Inc.2446
Coll-R, Sirogen 5/10/20122447
Sitaxsentan Sodium n/a 11/2/20042448
sitimagene ceradenovec Cerepro 7/31/2001 Gliotherapy, Ltd.2449
skin tissue Stratagraft 5/21/2012
Treatment of chronic/refractory anterior noninfectious uveitis, noninfectious intermediate uveitis, noninfectious panuveitis and non-infectious, uveitis affecting the posterior of the eye (NICUPS).
sirolimus in an implantable collagen matrix
Prevention of arteriovenous(AV) fistula or AV graft failure in patients with end stage renal disease, receiving hemodialysis or preparing for hemodialysis
Vascular Therapies, LLC
For the treatment of pulmonary arterial hypertension in the absence of chronic obstructive pulmonary disease or congestive heart failure.
Pfizer Global Research and Development
Use with gancyclovir in the treatment of malignant glioma
Treatment of hospitalized patients with complex skin defects resulting from partial and full thickness skin burns requiring excision and grafting
Stratatech Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 286 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2450
n/a 6/30/2016 Treatment of Ewing sarcoma Tokalas, Inc.2451
small molecule FGFR4 inhibitor n/a 9/14/20152452
n/a 1/21/2016 Treatment of mastocytosis2453
n/a 1/6/20162454
n/a 8/8/2013 Epizyme Inc.2455
n/a 8/15/2013 Epizyme Inc.2456
n/a 9/26/2013 Omeros Corporation2457
n/a 6/12/2014 Treatment of ovarian cancer2458
smilagenin Cogane 7/21/2011 Junaxo, Inc.2459
sobetirome n/a 4/29/2011 NeuroVia, Inc.
Small molecule Ewing Sarcoma breakpoint region 1/Friend leukemia virus integration 1 fusion protein inhibitor
Treatment of hepatocellular cancer (HCC).
Blueprint Medicines Corporation
small molecule inhibitor of Exon 17 Mutant KIT
Blueprint Medicines Corporation
Small Molecule Inhibitor of Exon 17 mutant KIT
Treatment of gastrointestinal stromal tumors (GIST).
Blueprint Medicines Corporation
small molecule inhibitor of histone methyltransferase DOT1L
Treatment of acute lymphoblastic leukemia (ALL)
small molecule inhibitor of histone methyltransferase DOT1L
Treatment of Acute Myeloid Leukemia
small molecule inhibitor of phosphodiesterase 10
Treatment of Huntington's disease
small molecule normalizing the p53 function
Critical Outcome Technologies, Inc.
Treatment of amyotrophic lateral sclerosis
Treatment of X-linked adrenoleukodystrophy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 287 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2460
sodium 2, 2 dimethylbutyrate n/a 7/25/2008 Treatment of sickle cell disease.2461
sodium 2-hydroxylinoleate n/a 8/25/2015 Treatment of neuroblastoma.2462
sodium 2-hydroxylinoleate n/a 11/23/2016 Treatment of pancreatic cancer.2463
sodium 4-phenylbutyrate n/a 10/18/2011 GMP-Orphan SAS2464
n/a 2/24/2016 Treatment of cystic fibrosis. AlgiPharma AS2465
Sodium aluminosilicate n/a 3/4/20052466
Apatone 4/15/20132467
Apatone 7/31/20072468
Apatone 4/15/20132469
Apatone 4/9/2015
HemaQuest Pharmaceuticals, Inc.
Ability Pharmaceuticals, SL
Ability Pharmaceuticals, SL
Treatment of spinal muscular atrophy
sodium alginate oligosaccharide
Treatment of chronic hepatic encephalopathy
Castle Creek Pharmaceuticals LLC
sodium ascorbate and menadione sodium bisulfite
Treatment of autosomal dominant polycystic liver disease
IC-Medtech Corporation
Sodium ascorbate and menadione sodium bisulfite
Treatment of metastatic or locally advanced inoperable transitional cell carcinoma of the urothelium (stage III and IV bladder cancer)
IC-MedTech Corporation
sodium ascorbate and menadione sodium bisulfite
Treatment of autosomal dominant polycystic kidney disease
IC-MedTech Corporation
sodium ascorbate and menadione sodium bisulfite
Treatment of noninfected painful total joint without mechanical complication
IC-MedTech Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 288 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2470n/a 12/22/2011
2471
sodium chlorite n/a 8/22/20112472
Sodium dichloroacetate n/a 11/10/19942473
sodium dichloroacetate n/a 7/3/2003 EBD Group2474
Sodium dichloroacetate n/a 6/11/19902475
sodium dichloroacetate n/a 11/29/20102476
Sodium dichloroacetate n/a 6/11/19902477
sodium fusidate n/a 10/23/20132478
Borocell 4/15/19922479
sodium nitrite n/a 7/8/2008
sodium benzoate and clozapine
Treatment of treatment-resistant schizophrenia
SyneuRx International Corp.
For slowing the progression of amyotrophic lateral sclerosis.
Neuraltus Pharmaceuticals, Inc.
Treatment of lactic acidosis in patients with severe malaria.
Stacpoole, Peter W. M.D., Ph.D.
Use as an antidote in the management of systemic monochloroacetic acid poisoning
Treatment of congenital lactic acidosis
Stacpoole, Peter W. M.D., Ph.D.
For pulmonary arterial hypertension.
Peter W. Stackpoole, PhD, MD
Treatment of homozygous familial hypercholesterolemia.
Stacpoole, Peter W. M.D., Ph.D.
Treatment of patients with prosthetic joint infections
Cempra Pharmaceuticals, Inc.
Sodium Monomercaptoundecahydro-closo-dodecaborate
For use in boron neutron capture therapy (BNCT) in the treatment of glioblastoma multiforme.
Neutron Technology Corp.& Neutron R&D Partner
Treatment of pulmonary arterial hypertension
Airess Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 289 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2480
Sodium nitrite n/a 1/17/20072481
Sodium nitrite n/a 4/18/2007 Treatment of cyanide poisoning2482
sodium nitrite n/a 1/9/20122483
sodium nitrite n/a 9/3/20092484
Sodium nitrite n/a 4/2/20072485
sodium nitrite and EDTA n/a 11/10/2015 Arch Biopartners, Inc.2486
Cyanide Antidote Package 3/23/2007 Treatment of cyanide poisoning2487
Ammonul(R) 6/3/2005 Ucyclyd Pharma, Inc.2488
Sodium phenylbutyrate Buphenyl 1/25/2007 OrphaMed, Inc.2489
sodium phenylbutyrate Pheburane 6/6/2013 Lucane Pharma SA
Prevention of vasospasm associated with subarachnoid hemorrhage
Hope Pharmaceuticals
Hope Pharmaceuticals
Treatment of chlorine gas poisoning
Hope Pharmaceuticals
Prevention of ischemia reperfusion injury to donor organ tissue associated with solid organ transplantation
Hope Pharmaceuticals
Treatment of vaso-occlusive crisis associated with sickle cell disease
Hope Pharmaceuticals
Treatment of Pseudomonas aeruginosa pulmonary infections in patients with cystic fibrosis
Sodium nitrite/sodium thiosulfate
Keystone Pharmaceuticals, Inc.
Sodium phenylacetate/sodium benzoate 10%/10% Injection
Treatment of grade III and IV hepatic encephalopathy
Treatment of spinal muscular atrophy
Treatment of urea cycle disorders
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 290 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2490
Sodium phenylbutyrate n/a 4/24/19982491
sodium phenylbutyrate n/a 1/19/2010 Treatment of urea cycle disorder. Navinta LLC2492
sodium phenylbutyrate n/a 7/2/19922493
Sodium phenylbutyrate n/a 3/20/20072494
sodium phenylbutyrate n/a 8/19/20142495
Sodium pyruvate n/a 3/31/2003 Treatment of cystic fibrosis Cellular Sciences, Inc2496
Sodium stibogluconate n/a 6/16/20062497
sodium stibogluconate n/a 10/28/20092498
Suprep 10/31/2012
For use as an adjuct to surgery, radiation therapy and chemotherapy for the treatment of patients with primary or recurrent malignant glioma.
Elan Drug Delivery, Inc.
Treatment for sickling disorders, which include S-S hemoglobinopathy, S-C hemoglobinopathy, and S-thalassemia hemoglobinopathy.
Medicis Pharmaceutical Corp.
Treatment of spinal muscular atrophy
Tikvah Therapeutics, Inc.
Treatment of maple syrup urine disease
Acer Therapeutics, Inc.
Treatment of cutaneous leishmaniasis
VioQuest Pharmaceuticals, Inc.
Treatment of cutaneous leishmaniasis
Surgeon General of the US Army
sodium sulfate, potassium sulfate, and magnesium sulfate
For cleansing of the colon in preparation for colonoscopic diagnosis of colonic disease in children and adolescents
Braintree Laboratories, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 291 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2499
Suclear 9/4/20132500
Sodium tetradecyl sulfate Sotradecol 6/10/1986 Elkins-Sinn, Inc.2501
sodium thiosulfate n/a 10/28/2014 Treatment of dermatomyositis2502
Sodium thiosulfate n/a 3/17/20042503
sodium thiosulfate n/a 12/2/20102504
sodium thiosulfate n/a 11/19/2012 Treatment of calciphylaxis Edinburg BioQuarter2505
sodium thiosulfate n/a 11/9/20112506
sodium thiosulfate n/a 10/13/20112507
sodium thiosulfate n/a 3/3/20112508
Sodium Thiosulfate n/a 2/16/20122509
n/a 12/18/2006 Treatment of cyanide poisoning Akorn, Inc.
sodium sulfate, potassium sulfate, and magnesium sulfate; PEG-3350, sodium chloride, sodium bicarbonate and potassium chloride
For use in cleansing of the colon in preparation for colonoscopy in children and adolescents
Braintree Laboratories, Inc.
Treatment of bleeding esophageal varices.
Hope Pharmaceuticals
Prevention of platinum-induced ototoxicity in pediatric patients
Fennec Pharmaceuticals, Inc.
Treatment of extravasation of meclorethamine hydrochloride into subcutaneous tissues.
Hope Pharmaceuticals
Treatment of uremic and non-uremic calciphylaxis
Hope Pharmaceuticals
Prevention of platinum-induced ototoxicity in pediatric patients
Hope Pharmaceuticals
Treatment of sulfur mustard poisoning
Hope Pharmaceuticals
Treatment of uremic and non-uremic calciphylaxis
Luitpold Pharmaceuticals, Inc.
Sodium thiosulfate and sodium nitrite
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 292 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2510
Cyanide Antidote Package 6/16/2006 Treatment of cyanide poisoning2511
sodium valproate n/a 8/5/2015 Treatment of Wolfram syndrome2512
sofosbuvir Sovaldi 10/25/2016 Gilead Sciences, Inc.2513
Solasonine and solamargine Coramsine 11/2/20052514
Solasonine and solamargine Coramsine 2/6/20062515
solnatide n/a 2/4/20162516
solnatide n/a 2/8/20162517
n/a 3/6/20002518
n/a 11/21/1994 T Cell Sciences, Inc.
Sodium thiosulfate, sodium nitrite, amyl nitrite
Keystone Pharmaceuticals, Inc.
The University of Birmingham
Treatment of pediatric chronic hepatitis C virus infection
Treatment of renal cell carcinoma
Solbec Pharmaceuticals Limited
Treaatment of high risk stage II, stage III and stage IV melanoma
Solbec Pharmaceuticals Limited
Treatment of primary graft dysfunction following lung transplantation
Apeptico Forschung und Entwicklung GmbH
Treatment of generalized systemic pseudohypoaldosteronism type 1 (also known as autosomal recessive PHA-I or PHA-IB)
Apeptico Forschung und Entwicklung GmbH
Soluble complement receptor type 1
Prevention of post-cardiopulmonary bypass syndrome in children undergoing cardiopulmonary bypass.
Avant Immunotherapeutics, Inc.
Soluble recombinant human complement receptor type 1
Prevention or reduction of adult respiratory distress syndrome.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 293 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2519
Uniplas 12/12/2005 Octapharma USA, Inc.2520
n/a 2/4/20162521
somatorelin Somatrel 8/8/19892522
Somatostatin n/a 12/22/19942523
Somatostatin Zecnil 6/20/19882524
Somatropin Serostim 3/16/2004 EMD Serono, Inc.2525
Somatropin (rDNA) Saizen 5/3/1989 EMD Serono, Inc.
solvent/detergent treated non-blood-group specific human coagulation active plasma
Treatment of thrombotic thrombocytopenic purpura
somatic cell therapy product containing hematopoietic progenitor cells (HPC), Facilitating Cells (FC), and alpha beta T cells.
Prevention of organ rejection in living donor kidney transplant recipients
Novartis Pharmaceuticals Corporation
Diagnostic measure of the capacity of the pituitary gland to release growth hormone.
Ferring Laboratories, Inc.
Treatment of bleeding esophageal varices.
Eumedica Pharmaceuticals A.G. (Schweiz)
Adjunct to the non-operative management of secreting cutaneous fistulas of the stomach, duodenum, small intestine (jejunum and ileum), or pancreas.
Ferring Laboratories, Inc.
Treatment of patients with HIV-associated adipose redistribution syndrome
For the enhancement of nitrogen retention in hospitalized patients suffering from severe burns.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 294 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2526
Serostim 3/26/1996 EMD Serono, Inc.2527
sonidegib n/a 3/23/2015 Treatment of medulloblastoma2528
sotalol hydrochloride Sotylize 2/10/20142529
sotatercept n/a 4/28/2014 Celgene Corporation2530
sotatercept n/a 12/5/2013 Celgene Corporation2531
sparsentan n/a 1/5/2015 Retrophin, LLC2532
n/a 6/21/2016 Treatment of ovarian cancer Kiromic, LLC2533
spherical carbon adsorbent n/a 12/19/2007 Treatment of chronic pouchitis2534
spironolactone Aldactone 5/22/2014 CMP Pharma, Inc.
Somatropin (r-DNA) for injection
Treatment of children with AIDS-associated failure-to-thrive including AIDS-associated wasting.
Novartis Pharmaceuticals Corp.
Treatment of life-threatening ventricular arrhythmias in pediatric patients
Arbor Pharmaceuticals, LLC
Treatment of anemias associated with myelodysplastic syndrome and myelodysplastic/myeloproliferative neoplasms.
Treatment of beta-thalassemia intermedia and major
Treatment of Focal Segmental Glomerulosclerosis
Sperm Protein-17 oral microparticle vaccine
Ocera Therapeutics, Inc.
Use in pediatric patients with primary hyperaldosteronism
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 295 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2535
squalamine lactate n/a 5/11/2001 Genaera Corporation2536
stabilized sulforaphane n/a 8/22/2016 Evgen Pharma PLC2537
n/a 6/10/2015 Protalex, Inc.2538
Altastaph 1/29/20042539
Nicord(R) 11/13/2014 Gamida Cell Ltd.2540
Nicord(R) 11/13/2014 Gamida Cell Ltd.2541
Nicord(R) 11/13/2014 Gamida Cell Ltd.2542
stemchymal n/a 12/16/20152543
Accusol 5/29/2009 Nikkiso America, Inc.
Treatment of ovarian cancer refractory or resistant to standard chemotherapy
Treatment of subarachnoid hemorrhage
staphylococcal aureus protein A
Treatment of immune thrombocytopenic purpura
Staphylococcus aureus Immune Globulin (Human)
Prophylaxis against Staphylococcus aureus infections in low birth weight neonates
Biotest Pharmaceuticals Corporation
stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells
Treatment of acute lymphoblastic leukemia.
stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells
Treatment of Hodgkin lymphoma.
stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells
Treatment of myelodysplastic syndrome.
Treatment of polyqlutamine spinocerebellar ataxia
Steminent Biotherapeutics, Inc.
Sterile Bicarbonate Infusate, Hemofiltration, Hemodiafiltration, and Hemopdialysis Solution
For use as a replacement solution in adults and children during Continous Renal Replacement Therapy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 296 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2544Sterile talc Steritalc 12/8/1997 Novatech SA
2545 Sterile talc Steritalc 12/8/1997 Treatment of pneumothorax. Novatech SA2546
stiripentol Diacomit 10/30/2008 Treatment of Dravet syndrome Biocodex2547
n/a 12/22/2015 Qurient Co., Ltd.2548
Succimer Chemet 11/5/1990 Sanofi Winthrop, Inc.2549
Succimer Chemet 3/22/1991 Sanofi Winthrop, Inc.2550
Sucralfate suspension n/a 3/12/19902551
Sucralfate suspension n/a 3/4/19912552
sulfamidase n/a 5/22/20082553
Sulfapyridine n/a 9/10/19902554
n/a 2/17/2012
Treatment of malignant pleural effusion.
Substituted imidazopyridine amide
Treatment of active tuberculosis (TB).
Prevention of cystine kidney stone formation in patients with homozygous cystinuria who are prone to stone development.
Treatment of mercury intoxication.
Treatment of oral complications of chemotherapy in bone marrow transplant patients.
Darby Pharmaceuticals, Inc.
Treatment of oral ulcerations and dysphagia in patients with epidermolysis bullosa.
Darby Pharmaceuticals, Inc.
For treatment of Sanfilippo Syndrome (MPS IIIA)
Shire Human Genetic Therapies, Inc.
Treatment of dermatitis herpetiformis.
Jacobus Pharmaceutical Company
sulfonated monophosphorylated mannose oligosaccharide
Treatment of hepatocellular carcinoma
Medigen Biotechnology Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 297 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2555
sulthiame n/a 7/25/20132556
n/a 3/6/19852557
superoxide dismutase, gliadin Etr019 4/30/2010 Verius Limited2558
Suramin Metaret 5/6/19972559
Synsorb Pk n/a 7/17/1995 Synsorb Biotech Inc.2560
n/a 2/4/2016 Treatment of ovarian cancer.
Treatment of patients with benign epilepsy of childhood with centrotemporal spikes (BECTS) also known as rolandic epilepsy
Marathon Pharmaceuticals, LLC
Superoxide dismutase (human)
For protection of donor organ tissue from damage or injury mediated by oxygen-derived free radicals that are generated during the necessary periods of ischemia (hypoxia, anoxia), and especially reperfusion, associated with the operative procedure.
Pharmacia-Chiron Partnership
Treatment of amyotrophic lateral sclerosis
Treatment of hormone-refractory prostate cancer.
Warner-Lambert Company
Treatment of verocytotoxogenic E. coli infections.
Synthesized peptide of the L-amino acids with the sequence H-Lys-Glu-Phe-Leu-His-Pro-Ser-Lys-Val-Asp-Leu-Pro-Arg-OH
Soricimed Biopharma Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 298 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2561
n/a 6/4/20142562
n/a 3/31/2015 MYR GmbH2563
Drepanol 10/24/19912564
n/a 4/22/20152565
n/a 8/16/2013 Treatment of hemophilia A2566
n/a 8/12/2013 Treatment of hemophilia B2567
n/a 9/25/2012
synthetic 14-mer phosphorothioate antisense oligonucleotide directed against TGF-beta2 mRNA
Prevention of scarring post glaucoma filtration surgery
Isarna Therapeutics GmbH
Synthetic 47 amino acid-long N-terminally myristoylated, HBV-L-protein derived lipopeptide
Treatment of hepatitis D virus infection.
Synthetic derivative of 16-hydroxy-9Z, 12Z, 14E-octadecatrienoic acid
Prophylactic treatment of sickle cell disease.
Omex International, Inc.
synthetic double-stranded (hybridized duplex) ribonucleic acid oligonucleotide specific to hydroxyacid oxidase 1 gene
Treatment of primary hyperoxaluria type 1
Dicerna Pharmaceuticals, Inc.
synthetic double-stranded siRNA oligonucleotide against antithrombin (AT) mRNA
Alnylam Pharmaceuticals
synthetic double-stranded siRNA oligonucleotide against antithrombin mRNA
Alnylam Pharmaceuticals
Synthetic double-stranded siRNA oligonucleotide against caspase 2 mRNA
Treatment of ischemic optic neuropathy
Quark Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 299 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2568
n/a 12/23/20092569
n/a 6/14/20122570
n/a 2/8/20162571
n/a 8/29/20162572
Synthetic human secretin n/a 6/16/1999 ChiRhoClin, Inc.2573
Synthetic human secretin n/a 6/16/1999 ChiRhoClin, Inc.2574
Synthetic human secretin n/a 9/18/2006
synthetic double-stranded siRNA oligonucleotide against p53 mRNA
Prophylaxis of delayed graft function in renal transplant patients
Quark Pharmaceuticals, Inc.
synthetic double-stranded siRNA oligonucleotide against transthyretin (TTR) mRNA
Treatment of familial amyloidotic polyneuropathy
Alnylam Pharmaceuticals, Inc.
synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 (HAO1) mRNA
Treatment of primary hyperoxaluria type 1
Alnylam Pharmaceuticals, Inc.
Synthetic double-stranded small interfering RNA (siRNA) oligonucleotide targeting delta-aminoevulinic acid synthase 1
Treatment of acute hepatic porphyria
Alnylam Pharmaceuticals
For use in the evaluation of exocrine pancreas function.
For use in obtaining desquamated pancreatic cells for cytopathologic examination in pancreatic carcinoma.
For use in conjunction with diagnostic procedures (excluding ERCP) for pancreatic disorders to increase pancreatic fluid secretion
Innovate Biopharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 300 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2575
Synthetic human secretin n/a 3/7/2000 ChiRhoClin, Inc.2576
n/a 1/13/20152577
n/a 10/12/2011 Atox Bio, Inc.2578
n/a 1/16/20132579
Synthetic porcine secretin n/a 6/18/1999 ChiRhoClin, Inc.2580
n/a 6/16/2015 Treatment of multiple myeloma.2581
n/a 3/16/2012 Chiesi USA, Inc.
For use in conjunction with diagnostic, therapeutic, or combined diagnostic/therapeutic procedures for pancreatic disorders to increase pancreatic fluid secretion.
synthetic oligomer of 16 nucleotides
Treatment of myotonic dystrophy Type I
Ionis Pharmaceuticals, Inc.
synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH
Treatment of necrotizing soft tissue infections (NSTI)
synthetic peptide; cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-ALA-Lys-Pro-Trp-Tyr-Cys
Treatment of high altitude pulmonary edema
Apeptico Forschung und Entwicklung GmbH
For use in obtaining desquamated pancreatic cells for cytopathologic examination in pancreatic carcinoma.
synthetic signal peptide of human mucin-1 (amino acids 1-21)
Vaxil Bio Therapeutics Ltd.
synthetic surfactant comprised of DPPC, POPG Na, synthetic SP-C analogue and synthetic SP-B analogue
Treatment of preterm neonatal respiratory distress syndrome
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 301 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2582
Neurovax 2/6/20142583
n/a 11/1/20012584
n/a 6/27/1989 AGI Dermatics2585
taberminogene vadenovec Trinam 10/24/20002586
tacrolimus n/a 5/4/20152587
tacrolimus n/a 3/16/2015 Selten Pharma, Inc.2588
Tacrolimus Prograf 4/6/1998 Fujisawa USA, Inc.2589
tacrolimus n/a 7/6/20122590
n/a 10/4/20162591
tadalafil Cialis(R); Adcirca(R) 5/4/2015 Eli Lilly and Company
T cell receptor (TCR) peptide (BV5S2, BV6S5, BV13S1) vaccine
Treatment of Pediatric Multiple Sclerosis
Immune Response BioPharma, Inc.
T-cell depleted stem cell enriched cellular product from peripheal b lood stem cells
Treatment of chronic granulomatous disease
Nexell Therapeutics Inc.
T4 endonuclease V, liposome encapsulated
To prevent cutaneous neoplasms and other skin abnormalities in xeroderma pigmentosum.
Prevention of complications due to neointimal hyperplasia disease in certain vascular anastomoses.
Finvector Vision Therapies, Ltd.
Treatment of pulmonary arterial hypertension
Stanford University School of Medicine
Treatment of pulmonary arterial hypertension.
Prophylaxis of graft-versus-host-disease.
Treatment of hemorrhagic cystitis
Lipella Pharmaceuticals Inc.
Tacrolimus granules for oral suspension
Prevention of rejection in kidney, liver or heart transplant in pediatric patients.
Astellas Pharma Global Development, Inc.
Treatment of Duchenne Muscular Dystrophy (DMD)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 302 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2592tafamidis n/a 5/23/2006 Pfizer, Inc.
2593
tafamidis meglumine n/a 2/17/2012 Pfizer, Inc.2594
tafenoquine n/a 1/15/2013 Treatment of malaria2595
talactoferrin alfa n/a 8/20/2003 Agennix, Inc.2596
talactoferrin alfa n/a 8/20/2003 Agennix, Inc.2597
Tamibarotene n/a 10/11/2007 CytRx Corporation2598
Tanespimycin n/a 9/9/2004 Treatment of multiple myeloma Bristol-Myers Squibb2599
tarextumab n/a 1/26/2015 Treatment of pancreatic cancer2600
tarextumab n/a 1/26/20152601
n/a 5/14/2014 Treatment of pancreatic cancer Tyg Oncology Ltd2602
tasimelteon n/a 4/30/20102603
taurine n/a 3/22/2010
Treatment of familial amyloid polyneuropathy
Treatment of symptomatic transthyretin (TTR) amyloid cardiomyopathy.
Glaxo Group Limited, England
For the treatment of graft versus host disease
For the prevention of graft-versus-host disease
Treatment of acute promyelocytic leukemia (APL).
OncoMed Pharmaceuticals, Inc.
Treatment of small cell lung cancer
OncoMed Pharmaceuticals, Inc.
Targeted Gastrin 17 Complexed Peptide
Treatment of sleep-wake disorder in Smith-Magenis syndrome associated with diurnal melatonin secretion
Vanda Pharmaceuticals, Inc.
Treatment of cystathionine beta-synthase deficient homocystinuria
Johan L. Van Hove, MD, PhD
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 303 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2604taurolidine n/a 5/16/2016 Treatment of pancreatic cancer.
2605tazemetostat n/a 2/4/2016 Epizyme Inc.
2606TD-K6a.513a.12 Reveker 6/15/2006 TransDerm, Inc.
2607
Lymphoscan 4/7/1992 Immunomedics, Inc.2608
Apomate 11/3/20002609
tecovirimat n/a 9/29/20102610
tecovirimat n/a 12/18/20062611
tecovirimat n/a 12/27/2006 Treatment of smallpox.2612
Tegafur/gimeracil/oteracil n/a 7/20/2006 Treatment of gastric cancer2613 telatinib n/a 5/17/2010 Treatment of gastric cancer ACT Biotech Inc.
Geistlich Pharma NA, Inc.
Treatment of malignant rhabdoid tumors (MRTs)
Treatment of pachyonychia congenita
Technetium Tc99m murine monoclonal antibody (IgG2a) to B cell
Diagnostic imaging in the evaluation of the extent of disease in patients with histologically confirmed diagnosis of non-Hodgkin's B-cell lymphoma, acute B-cell lymphoblastic leukemia (in children and adults), and chronic B-cell lymphocytic leukemia.
Technetium Tc99m rh-Annexin V
Diagnosis or assessment of rejection status in heart, heart-lung, single lung, or bilateral lung transplants.
Theseus Imaging Corporation
Treatment of orthopoxvirus infections.
SIGA Technologies, Inc.
post exposure prophylaxis against smallpox
SIGA Technologies, Inc.
SIGA Technologies, Inc.
Taiho Pharma USA, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 304 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2614
telotristat etiprate n/a 3/9/20122615
temocillin sodium Negaban 4/21/2004 Belpharma S.A.2616
Temoporfin Foscan 10/28/19992617
Temozolomide Temodal 10/18/20042618
Temozolomide Temodal 10/14/19982619
temozolomide n/a 11/25/20152620
tenofovir disoproxil fumarate Viread 9/27/2016 Gilead Sciences, Inc.2621
teprotumumab n/a 5/6/2013 River Vision, Inc.2622
terguride Mysalfon, Teluron 5/17/2013 Treatment of systemic sclerosis Medac GmbH2623
Teriparatide Parathar 10/28/1999 Biomeasure, Inc.
Treatment of carcinoid syndrome in patients with neuroendocrine tumors
Lexicon Pharmaceuticals, Inc.
Treatment of pulmonary infections caused by Burkholderia cepacia
Palliative treatment of recurrent, refractory or second primary squamous cell carcinomas of the head and neck in patients considered to be incurable with surgery or radiotherapy.
Biolitec Pharma Ireland Ltd.
Treatment of newly diagnosed high grade glioma
Schering-Plough Research Institute
Treatment of advanced metastatic melanoma.
Schering-Plough Research Institute
Treatment glioblastoma multiforme in pediatric patients
AmpliPharm Pharmaceuticals, LLC
Prevention of mother to child transmission of hepatitis B virus
Treatment of active (dynamic) phase Grave's orbitopathy
Treatment of idiopathic osteoporosis.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 305 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2624teriparatide Forteo 4/18/2014 Entera Bio, Ltd.
2625terlipressin n/a 9/8/2016 Bio Vie Inc.
2626Terlipressin Glypressin 3/6/1986
2627terlipressin n/a 10/29/2004
2628 tesetaxel n/a 12/22/2008 Treatment of gastric cancer Genta Inc.2629
tesetaxel n/a 11/21/2008 Genta, Inc.2630
Tesevatinib n/a 3/1/20162631
Testosterone 9/22/1997 Watson Laboratories2632
n/a 7/31/1991 Treatment of vulvar dystrophies.2633
n/a 2/13/2013
Treatment of hypoparathyroidism
Treatment of ascites due to all etiologies except cancer
Treatment of bleeding esophageal varices.
Ferring Laboratories, Inc.
Treatment of Hepatorenal Syndrome
Ikaria (INO Therapeutics)
Treatment of stages IIB, IIC, III, and stage IV melanoma
Treatment of patients with autosomal recessive polycystic kidney disease (ARPKD)
Kadmon Corporation, LLC
Theraderm Testosterone Transdermal System
For use as physiologic testosterone replacement in androgen deficient HIV+ patients with an associated weight loss.
Testosterone propionate ointment 2%
Star Pharmaceuticals, Inc.
testosterone undecanoate (oral)
Treatment of constitutional delay in growth and puberty in adolescent boys (14-17 yrs of age)
SOV Therapeutics, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 306 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2634
n/a 1/17/20142635
n/a 3/16/20152636
Tetrabenazine n/a 5/12/19982637
tetrabenazine n/a 7/1/20092638
10/31/2012 Treatment of infantile spasms2639
n/a 6/20/2016 Treatment of infantile spasms2640
Tetraiodothyroacetic acid n/a 5/1/20002641
tetrandrine n/a 5/19/2016
tetra substituted prophyrin derivative containing manganese (III)
For use in patients exposed to radiation following a nuclear accident or detonation in order to treat or mitigate acute radiation syndrome.
Aeolus Pharmaceuticals, Inc.
tetra-substituted porphyrin derivative containing manganese (III)
Treatment of idiopathic pulmonary fibrosis
Aeolus Pharmaceuticals
Treatment of moderate/severe tardive dyskinesia.
Prestwick Pharmaceuticals, Inc.
Treatment of Tourette's Syndrome in school-age children, ages 5-16
Valeant International (Barbados) SRL
tetracosactide hexaacetate (beta 1-24-corticotrophin)
Synacthen Depot, S. Retard
Cerium Pharmaceuticals, Inc.
tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride
Anavex Life Sciences Corp.
Suppression of thyroid stimulating hormone in patients with well-differentiated cancer of the thyroid gland.
Danforth, Jr., MD, Elliot
Treatment of chronic myeloid leukemia.
Escend Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 307 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2642
Tezacitabine n/a 1/27/20032643
Thalidomide n/a 2/27/1998 Celgene Corporation2644
Thalidomide n/a 5/15/19952645
Thalidomide n/a 7/29/1998 Treatment of Kaposi's sarcoma. Celgene Corporation2646
Thalidomide n/a 3/5/19902647
Thalidomide n/a 5/1/1995 Celgene Corporation2648
Thalidomide n/a 3/5/19902649
thalidomide n/a 9/19/19882650
Thalidomide Thalomid 4/6/1999 Treatment of Crohn's disease. Celgene Corporation
Treatment of adenocarcinoma of the esophagus and stomach
Sanofi-Aventis US, Inc.
Treatment of primary brain malignancies.
Treatment and prevention of recurrent aphthous ulcers in severely, terminally immunocompromised patients.
Andrulis Research Corporation
Treatment of graft versus host disease.
Andrulis Research Corporation
Treatment of severe recurrent aphthous stomatitis in severely, terminally immunocompromised patients.
Prevention of graft versus host disease.
Andrulis Research Corporation
1. Treatment of graft versus host disease in patients receiving bone marrow transplantation 2. Prevention of graft versus host disease in patients receiving bone marrow transplantation
Pediatric Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 308 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2651Thalidomide Synovir 3/11/1996 Celgene Corporation
2652
Thalidomide n/a 1/12/1993 Celgene Corporation2653
Thalidomide n/a 11/15/19882654
Thalidomide Thalomid 9/27/2004 Celgene Corporation2655
n/a 10/23/2008 Aldagen, Inc.2656
Theranost 68 Ga RGD n/a 10/1/20142657
Theranost 68Ga RGD n/a 1/26/20152658
theranost 68Ga-RGD n/a 12/8/2014
Treatment of HIV-associated wasting syndrome.
Treatment of the clinical manifestations of mycobacterial infection caused by Mycobacterium tuberculosis and non-tuberculous mycobacteria.
Treatment and maintenance of reactional lepromatous leprosy.
Pediatric Pharmaceuticals, Inc.
Treatment of myelodysplastic syndrome
Thawed donor matched banked umbilical cord blood enriched by cell sorting to produce a subpopulation expressing high levels of intracellular aldehyde dehydrogenase
To improve patient outcomes by decreasing time to platlet and neutrophil engraftment in patients with inherited metabolic diseases (IMD) undergoing umbilical cord blood transplantation.
A Diagnostic for the management of Moyamoya disease (MMD)
Advanced Imaging Projects, LLC (AIP)
Diagnostic for the clinical management of patients with tuberculosis
Advanced Imaging Projects, LLC
Diagnostic for clinical management of patients with neuroblastoma
Advanced Imaging Projects, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 309 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2659
Thiotepa Tepadina 4/2/2007 Adienne S.A.2660
thioureidobutyronitrile Kevetrin 7/14/2015 Treatment of ovarian cancer2661
thioureidobutyronitrile Kevetrin 11/17/2015 Treatment of retinoblastoma.2662
thioureidobutyronitrile Kevetrin 1/20/2016 Treatment of pancreatic cancer.2663
n/a 7/14/20162664
Thymalfasin Zadaxin 3/6/20002665
Thymalfasin Zadaxin 3/13/20062666
Thymalfasin Zadaxin 1/8/19982667
Thymalfasin Zadaxin 5/3/19912668
thymopentin n/a 2/4/2011 Treatment of sarcoidosis.2669
thymosin beta 4 n/a 12/31/2013 ReGenTree, LLC
Conditioning treatment prior to hematopoietic stem cell transplantation
Cellceutix Corporation
Cellceutix Corporation
Cellceutix Corporation
three human IgG1 mAbs (REGN3470, REGN3471, and REGN3479) directed against different epitopes on Ebola virus glycoprotein
Treatment of ebola virus infection
Regeneron Pharmaceuticals, Inc.
Treatment of hepatocellular carcinoma.
SciClone Pharmaceuticals, Inc.
Treatment of stage IIb through Stage IV malignant melanoma
SciClone Pharmaceuticals, Inc.
Treatment of DiGeorge anomaly with immune defects.
SciClone Pharmaceuticals, Inc.
Treatment of chronic active hepatitis B.
SciClone Pharmaceuticals, Inc.
Mondobiotech Laboratories AG
Treatment of patients with neurotrophic keratopathy
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 310 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2670Thymosin beta 4 n/a 5/28/2004 GtreeBNT Co., Ltd
2671
n/a 12/27/20002672
tigecycline n/a 10/23/20132673
Tilarginine acetate n/a 4/11/2005 Treatment of cardiogenic shock2674
tin ethyl etiopurpurin n/a 11/4/20032675
Tiotropium bromide Spiriva 1/8/20082676
tipelukast n/a 10/20/2014 MediciNova, Inc.2677
Tipifarnib Zarnestra 7/6/20042678
tiprelestat Elafin 12/28/2012 Proteo Biotech AG2679
tirapazamine n/a 10/23/20022680
tirapazamine n/a 11/30/2015 Teclison Limited2681
tirasemtiv n/a 3/2/2010 Cytokinetics Inc.
Treatment of epidermolysis bullosa.
Tiazofurin (2-Beta-D-ribofuranosyl-4-thiazolecarboxamide)
Chronic myelogenous leukemia (CML)
Valeant Pharmaceuticals North America
Treatment of acute myeloid leukemia.
Trillium Therapeutics, Inc.
Arginox Pharmceuticals, Inc.
Prevention of access graft disease in hemodialysis patients
Miravant Medical Technologies
To improve pulmonary function in conjunction with standard therapy in the management of patients with cystic fibrosis
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of idiopathic pulmonary fibrosis
Treatment of acute myeloid leukemia
Johnson & Johnson Pharmaceutical Research & Dev.
Treatment of pulmonary arterial hypertension
Treatment of head and neck cancer
Sanofi-Aventis US, Inc.
Treatment of hepatocellular carcinoma
Treatment of amyotrophic lateral sclerosis (ALS)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 311 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2682
Tiratricol Triacana 8/13/1991 Laphal Laboratoires2683
tisagenlecleucel-T n/a 2/3/20152684
Tissue Plasminogen activator n/a 4/18/20112685
n/a 1/25/2007 Vericel Corporation.2686
n/a 3/13/2006 Treatment of osteonecrosis.2687
tivantinib n/a 10/16/20132688
Tizanidine HCl Zanaflex 1/31/1994
For use in combination with levo-thyroxine to suppress thyroid stimulating hormone in patients with well-differentiated thyroid cancer who are intolerant to adequate doses of levo-thyroxine alone.
Treatment of diffuse large B-cell lymphoma
Novartis Pharmaceuticals Corporation
Treatment of acute ischemic stroke (AIS) in children age 16 years and younger.
Catherine Amlie-Lefond, MD
Tissue repair cells obtained from autologous bone marrow expanded ex vivo with a cell production system
Treatment of dilated cardiomyopathy
Tissue repair cells obtained from autologous bone marrow expanded ex vivo with a cell production system
Aastrom Biosciences, Inc.
Treatment of hepatocelular carcinoma
Daiichi Sankyo Pharma Development
Treatment of spasticity associated with multiple sclerosis and spinal cord injury.
Athena Neurosciences, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 312 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2689
Tobramycin Tobi 6/18/19992690
tocilizumab Actemra 4/17/2013 Treatment of systemic sclerosis Genentech, Inc.2691
Tolcapone n/a 12/24/20132692
tolerogen n/a 2/5/2015 Treatment of myasthenia gravis Toleranzia AB2693
tolfenamic acid n/a 7/13/2016 Nasser H. Zawia2694
tolfenamic acid n/a 7/13/2016 Nasser H. Zawia2695
tolvaptan Samsca 4/6/20122696
topiramate injection n/a 7/24/20132697
Toremifene n/a 8/17/1993 Treatment of desmoid tumors. Orion Corporation2698
Tosedostat n/a 12/10/2008
Treatment of bronchiectasis patients infected with Pseudomonas aeruginosa.
Novartis Pharmaceuticals Corp.
Treatment of transthyretin amyloidosis
SOM Innovation Biotech SL (SOM Biotech)
Treatment of Frontotemporal Dementia
Treatment of progressive supranuclear palsy
Treatment of autosomal dominant polycystic kidney disease
Otsuka Pharmaceuticals Co., Ltd.
Treatment of partial onset or primary generalized tonic-clonic seizures for hospitalized epilepsy patients or epilepsy patients being treated in an emergency care setting who are unable to take oral topiramate
CURx Pharmaceuticals, Inc.
Treatment of acute myeloid leukemia
CTI BioPharma Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 313 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2699
trabectedin Yondelis 3/29/20052700
trabedersen n/a 7/21/2009 Treatment of pancreatic cancer. Oncotelic, Inc.2701
trabedersen Oncomun 6/5/2002 Treatment of malignant glioma Oncotelic Inc.2702
trabedersen n/a 8/22/2011 Oncotelic, Inc.2703
Tramadol hydrochloride n/a 4/26/20052704
Tramadol hydrochloride n/a 1/28/20052705
Tranilast Rizaben 12/2/20032706
tranilast Rizaben 12/23/2010 Altacor Ltd2707
trans sodium crocetinate n/a 7/21/20112708
trans sodium crocetinate n/a 12/3/2012 Treatment of brain metastasis2709
n/a 12/18/1992
Treatment of patients with ovarian cancer
Janssen Research & Development, LLC
Treatment of Stage IIB through Stage IV malignant melanoma.
Management of postherpetic neuralgia
TheraQuest Biosciences, LLC
Treatment of painful HIV-associated neuropathy
TheraQuest Biosciences, LLC
For the treatment of maligant glioma
Angiogen Pharmaceuticals, Pty. Ltd.
Prevention of scarring following glaucoma filtration surgery
Treatment of glioblastoma in conjunction with radiotherapy
Diffusion Pharmaceuticals, LLC
Diffusion Pharmaceuticals, LLC
Transforming growth factor-beta 2
Treatment of full thickness macular holes.
Celtrix Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 314 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2710
n/a 3/11/2013 Treatment of glioma Eli Lilly and Company2711
n/a 4/1/2013 Eli Lilly and Company2712
n/a 5/19/19992713
trastuzumab Herceptin 12/14/1999 Genentech, Inc.2714 trebananib n/a 8/30/2013 Treatment of ovarian cancer Amgen, Inc.2715
trehalose n/a 11/17/2014 BioBlast Pharma Ltd2716
trehalose Cabaletta 10/25/2013 BioBlast Pharma, Ltd.2717
tremelimumab n/a 3/18/2015 MedImmune, LLC2718
tremelimumab n/a 9/18/2006 MedImmune2719 Treosulfan Ovastat 5/16/1994 Treatment of ovarian cancer. Medac GmbH
transforming growth factor-beta receptor 1 kinase inhibitor
transforming growth factor-beta receptor 1 kinase inhibitor
Treatment of hepatocellular carcinoma
Transgenic human alpha 1 antitrypsin
Treatment of emphysema secondary to alpha 1 antitrypsin deficiency.
PPL Therapeutics (Scotland) Limited
Treatment of patients with pancreatic cancer that overexpress p185HER2.
Treatment of spinal cerebellar ataxia type 3 (also known as SCA3 or Machado Joseph disease)
Treatment of occulopharyngeal muscular dystrophy
Treatment of malignant mesothelioma
Treatment of stage IIb to stage IV metastatic melanoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 315 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2720
treosulfan n/a 4/8/2015 Medac GmbH2721
n/a 12/29/20152722
Tretinoin Atra-Iv 4/11/2003 Antigenics, Inc.2723
Tretinoin n/a 4/15/19852724
Tretinoin Atra-Iv 1/14/1993 Antigenics, Inc.2725
n/a 11/23/2001 Cell Works Inc.2726
triciribine n/a 2/1/2008 Treatment of multiple myeloma
Conditioning treatment prior to hematopoietic stem cell transplantation (HSCT) in malignant and non-malignant diseases in adults and pediatric patients.
treprostinil sodium solution enclosed within a pre-filled, pre-programmed, single-use, electronically-controlled infusion pump
Treatment of pulmonary arterial hypertension
SteadyMed Therapeutics
Treatment of T-cell non-Hodgkin's lymphoma
Treatment of squamous metaplasia of the ocular surface epithelia (conjunctiva and/or cornea) with mucous deficiency and keratinization.
Hannan Ophthalmic Marketing Services, Inc
Treatment of acute and chronic leukemia.
tri-antennary glycotripeptide derivative of 5-fluorodeoxyuridine monophosphate
Treatment for hepatocellular carcinoma
VioQuest Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 316 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2727
triclabendazole Triclathera(Tm) 6/27/2016 Treatment of fascioliasis.2728
trientine hydrochloride Syprine(R) 11/2/2010 Treatment of manganism.2729
trientine tetrahydrochloride n/a 3/10/2016 treatment of Wilson’s disease GMP-Orphan SAS2730
trifarotene n/a 6/6/2014 Galderma R&D, LLC2731
Triheptanoin 5/26/2006 Treatment of fatty acid disorders2732
triheptanoin n/a 10/21/20142733
triheptanoin n/a 4/15/20152734
Triheptanoin 2/1/20082735
triptorelin pamoate n/a 8/20/20122736
Trisaccharides A and B Biosynject 4/20/1987 Chembiomed, Ltd.
Tropicalis Pharmaceuticals, LLC
Cerberus Princeton, LLC
Treatment of congenital ichthyosis
Triheptanoim-Sasol Special Oil
Baylor Research Institute
Treatment of glucose transporter type-1 deficiency syndrome
Ultragenyx Pharmaceutical, Inc.
Treatment of fatty acid oxidation disorders
Ultragenyx Pharmaceutical, Inc.
Triheptanoin-Sasol Special Oil
Treatment of glycogen storage disorder II (Pompe disease)
Baylor Research Institute
Treatment of central precocious puberty
Arbor Pharmaceuticals, LLC
For use in ABO-incompatible solid organ transplantation, including kidney, heart, liver and pancreas.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 317 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2737
Trisaccharides A and B Biosynject 4/12/1987 Chembiomed, Ltd.2738
Trisaccharides A and B Biosynject 4/15/1988 Chembiomed, Ltd.2739
Hemocitrate 6/15/19952740
n/a 2/27/20042741
triterpenoid saponin n/a 7/23/20142742
n/a 1/6/1993 EMD Serono, Inc.2743
n/a 1/6/1993 EMD Serono, Inc.
Treatment of moderate to severe clinical forms of hemolytic disease of the newborn arising from placental transfer of antibodies against blood group substances A and B.
Prevention of ABO medical hemolytic reactions arising from ABO-incompatible bone marrow transplantation.
Trisodium citrate concentration
For use in leukapheresis procedures.
Hemotec Medical Products, Inc.
Trisodium zinc Diethylenetriaminepentaacetate
Treatment of patients with known or suspected internal contamination with plutonium, americium, or curium to increase the rate of elimination.
CustomCare Pharmacy
Treatment of mantle cell lymphoma
Avicin Therapeutics, Ltd.
Tumor necrosis factor-binding protein 1
Treatment of symptomatic patients with AIDS including all patients with CD4 counts less than 200 cells per mm3.
Tumor necrosis factor-binding protein II
Treatment of symptomatic patients with the AIDS including all patients with CD4 T-cell counts less than 200 cells per mm3.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 318 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2744
n/a 6/9/20152745
TXA127 n/a 10/25/2013 US Biotest, Inc.2746
Tyloxapol Supervent 3/8/1995 Treatment of cystic fibrosis.2747
n/a 2/1/20082748
ubenimex n/a 11/19/20152749
ubiquinol Ubi-Q-Nol, Li-Q-Nol 4/12/20042750
Ubi-Q-Nol 4/12/20042751
Ubiquinone Ubi-Q-Gel 12/14/19992752
ublituximab n/a 9/5/2013 TG Therapeutics, Inc.2753
ublituximab n/a 9/5/2013 TG Therapeutics, Inc.2754
ublituximab n/a 8/6/2010 TG Therapeutics, Inc.2755
ublituximab n/a 8/25/2016 TG Therapeutics, Inc.
tumor-inflitrating lymphocytes; LN-144
Treatment of malignant melanoma Stages IIb to IV
Lion Biotechnologies, Inc.
Treament of acute radiation syndrome
Kennedy & Hoidal, M.D.'s
Type 1 native bovine skin collagen
Treatment of diffuse systemic sclerosis
arGentis Pharmaceuticals, LLC
Treatment of pulmonary arterial hypertension.
Eiger BioPharmaceuticals, Inc.
Treatment of Huntington's Disease
Gel-Tec, Division of Tishcon Corp.
Ubiquinol, coenzyme Q10, ubiquinone
Treatment of pediatric congestive heart failure
Gel-Tec, Division of Tishcon Corporation
Treatment of mitochondrial cytopathies.
Gel-Tec, Division of Tishcon Corp.
Treatment of Extranodal marginal zone lymphoma (mucosa-associated lymphatic tissue, MALT)
Treatment of Nodal marginal zone lymphoma
Treatment of chronic lymphocytic leukemia
Treatment of neuromyelitis optica and neuromyelitis optica spectrum disorder
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 319 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2756
udenafil n/a 8/31/20152757
ulocuplumab n/a 1/12/20152758
urea n/a 11/7/2011 Orenova Group, LLC2759
Urea for intravitreal injection Neurosolve 12/14/20052760
uridine triacetate n/a 9/3/20092761
Urogastrone n/a 11/1/1984 Chiron Vision
Treatment of single ventricle congenital heart disease with Fontan physiology
Mezzion Pharma Co. Ltd.
Treatment of acute myeloid leukemia
Bristol-Myers Squibb Company
Treatment of rare congenital ichthyoses (CHILD syndrome, collodion baby, congenital ichthyosform erythroderma, Conradi-flunermann, epidermolytic hyperkeratosis, erythrokeratoderma variabilis, harlequin ichthyosis, KID syndrome, lamellar ichthyosis, Netherton syndrome, neutral lipid storage disease, Sjorgren-Larsson syndrome, trichothiodystrophy, X-linked ichthyosis).
Treatment of retinitis pigmentosa
Vitreo Retinal Techologies, Inc
Treatment of mitchondrial disease
Wellstat Therapeutics Corporation
For acceleration of corneal epithelial regeneration and healing of stromal incisions from corneal transplant surgery.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 320 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2762
ursodiol Ursofalk Suspension 10/23/20072763
ustekinumab Stelara 5/18/20162764
ustekinumab Stelara 11/29/20102765
vadastuximab talirine n/a 11/10/2015 Seattle Genetics, Inc.2766
Valine, isoleucine and leucine Vil 1/5/19962767
valine-valine-ganciclovir n/a 5/21/20072768
Valproate n/a 5/5/2008 Treatment of fragile X syndrome Neuropharm Ltd,2769
vancomycin n/a 12/27/2012 Treatment of endophthalmitis2770
n/a 9/20/20122771
vapreotide Sanvar 1/10/2000 Debiovision, Inc.2772
vapreotide Octastatin 1/10/2000 Debiopharm S.A.
Treatment of cystic fibrosis liver disease
Asklepion Pharmaceuticals, LLC
Treatment of pediatric Crohn's disease (0 through 16 years of age)
Janssen Research & Development, LLC
Treatment of type 1 diabetes mellitus patients with residual beta-cell function.
Johnson & Johnson Pharmaceutical & Development LLC
Treatment of acute myeloid leukemia.
Treatment of hyperphenylalaninemia
Leas Research Products
Treatment of primary keratoconjunctivitis and recurrent epithelial keratitis due to HSV1 and HSV2.
Verenta Pharmaceuticals, Inc.
Fera Pharmaceuticals, LLC
vancomycin hydrochloride (inhalational)
Treatment of persistent methicillin-resistant S. aureus lung infection in patients with cystic fibrosis
Savara Pharmaceuticals, Inc.
Treatment of esophageal variceal hemorrhage patients with portal hypertension.
Treatment of gastrointestinal and pancreatic fistulas.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 321 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2773Vapreotide Sanvar 4/6/2004 H3 Pharma, Inc.
2774
vapreotide Octastatin 3/6/2000 Debiopharm S.A.2775 vapreotide Sanvar 11/4/2003 Treatment of acromegaly H3 Pharma, Inc.2776
Varbulin Azixa 11/5/2009 Myrexis, Inc.2777
Varlitinib n/a 8/5/20152778
varlitinib n/a 6/20/2016 Treatment of gastric cancer.2779
n/a 6/24/2008 PhiloGene, Inc.2780
Vasoactive intestinal peptide n/a 3/9/2001 mondoBIOTECH2781
n/a 5/13/20142782
n/a 6/23/1993
Treatment of symptomatic carcinoid tumors
Prevention of early postoperative complications following pancreatic resection.
Treatment of glioblastoma multiforme.
Treatment of cholangiocarcinoma.
ASLAN Pharmaceuticals
ASLAN Pharmaceuticals
vascular endothelial growth factor 165b
Treatment of advanced melanoma stages IIb through IV.
Treatment of Acute Respiratory Distress Syndrome.
vasoactive intestinal peptide (VIP)-elastin-like peptide (ELP) fusion protein
Treatment of pulmonary arterial hypertension, WHO Group 1
PhaseBio Pharmaceuticals, Inc.
Vasoactive intestinal polypeptide
Treatment of acute esophageal food impaction.
Research Triangle Pharmaceuticals
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 322 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2783
Vasomera n/a 11/19/20152784
vatiquinone Vincerinone 1/31/2014 Treatment of Friedreich's ataxia2785
vatiquinone Vincerinone 11/17/2014 Treatment of Rett syndrome2786
vatiquinone Vincerinone 6/4/2014 Treatment of Leigh Syndrome2787
vedolizumab n/a 8/4/20162788
veliparib n/a 9/3/2009 AbbVie, Inc.2789
veliparib n/a 12/17/2014 AbbVie Inc.2790
veliparib n/a 11/3/2016 AbbVie Inc.
Treatment of cardiomyopathy associated with dystrophinopathies; Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy BMD), and X-linked dilated cardiomyopathy (XL-dCMP).
PhaseBio Pharmaceuticals, Inc.
Edison pharmaceuticals, Inc.
Edison pharmaceuticals, Inc.
Edison Pharmaceuticals, Inc.
Prevention of graft versus host disease.
Millennium Pharmaceuticals, Inc.
Treatment of epithelial ovarian cancer in combination with DNA-damaging agents
Treatment of brain metastases when used in combination with DNA-damaging agents
Treatment of squamous non-small cell lung cancer
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 323 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2791
veliparib n/a 5/9/2008 AbbVie, Inc.2792
veliparib n/a 11/20/2009 AbbVie, Inc.2793
veltuzumab n/a 7/28/2015 Immunomedics, Inc.2794
veltuzumab n/a 11/17/2014 Treatment of pemphigus Immunomedics, Inc.2795
veltuzumab n/a 8/28/2008 Immunomedics, Inc.2796
vemurafenib Zelboraf 9/8/2014 Genentech, Inc.2797
vemurafenib Zelboraf 11/26/2013 Genentech, Inc.2798
vemurafenib Zelboral 8/2/2016 Genentech, Inc.2799
vemurafenib Zelboraf 8/26/2014 Genentech, Inc.2800
Venetoclax n/a 8/29/2016 Treatment of multiple myeloma AbbVie Inc.2801
venetoclax n/a 2/4/2016 AbbVie Inc.
Treatment of glioblastoma multiforme when used in combination with DNA-damaging agents
Treatment of hepatocellular carcinoma in combination with DNA-damaging agents
Treatment of immune thrombocytopenic purpura.
Treatment of chronic lymphocytic leukemia
Treatment of patients with non-small cell lung cancer (NSCLC) with BRAF V600 mutation
Treatment of anaplastic thyroid carcinoma and advanced papillary thyroid cancer whose tumors harbor a BRAF V600 mutation
Treatment of Erdheim-Chester Disease
Treatment of hairy cell leukemia (HCL).
Treatment of acute myeloid leukemia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 324 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2802
verteporfin Visudyne(R) 3/9/20122803
n/a 10/12/20162804
vibriolysin Vibrilase 6/16/20062805
vincristine sulfate liposomes Marqibo 6/24/2008 Talon Therapeutics2806 vintafolide n/a 12/16/2013 Treatment of ovarian cancer Endocyte, Inc.2807
Virulizin Virulizin 2/1/2001 Treatment of pancreatic cancer.2808
vitamin A palmitate n/a 7/14/20152809
n/a 6/23/20152810
volasertib n/a 4/14/20142811
Wilfactin 5/29/2014 rEVO Biologics, Inc.2812
vorinostat n/a 10/13/2016
Treatment of chronic or recurrent central serous chorioretinopathy
Valeant Pharmaceuticals North America LLC
Vibrio alginolyticus collagenase
Treatment of advanced Dupuytren's disease (Dupuytren's contracture)
Fidia Farmaceutici S.p.A.
Debridement of severe, deep dermal burns in hospitalized patients
BioMarin Pharmaceutical Inc.
Treatment of metastatic uveal melanoma.
ZOR Pharmaceuticals, LLC
Prevention of bronchopulmonary dysplasia.
Advent Therapeutics, Inc.
volanesorsen sodium, apolipotrotein C-III antisense oligonucleotide
treatment of familial chylomicronemia syndrome
Ionis Pharmaceuticals, Inc.
Treatment of acute myeloid leukemia
Boehringer Ingelheim Pharmaceuticals, Inc.
von Willebrand Factor Human Concentrate
Treatment of Von Willebrand Disease
Treatment of advanced melanoma (stages IIb, IIc, III, and IV)
Qameleon Therapeutics
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 325 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2813
vosaroxin n/a 10/28/20092814
Aquasol A Parenteral 3/26/2010 Fox Pharma, Inc.2815
n/a 9/27/20162816
Xenogeneic hepatocytes 11/27/1998 Treatment of severe liver failure. Circe Biomedical, Inc.2817
xenon gas n/a 12/3/2014 Neuroprotexeon2818
xenon gas n/a 5/18/2015 Neuroprotexeon2819
n/a 11/30/20162820
Ferritarg P 9/18/2006 Treatment of Hodgkin's disease. Alissa Pharma, LLC2821
n/a 12/3/2012 Treatment of soft tissue sarcoma
Treatment of acute myeloid leukemia
Sunesis Pharmaceuticals, Inc.
Water-miscible vitamin A palmitate
Prevention of bronchopulmonary dysplasia.
Wharton's jelly mesenchymal stem cells
Treatment of graft-versus-host disease
Midwest Stem Cell Therapy Center
Hepatassist Liver Assist System
Treatment of hypoxic ischemic encephalopathy
To improve neurological outcome in hospitalized cardiac arrest patients.
Yersinia pestis plague recombinant antigen (rF1V; F1 capsular protein fused to V antigen; E. coli) vaccine with aluminum hydroxide
Prevention of plague (Yersinia pestis)
DynPort Vaccine Company, LLC, a CSRA Comapny
Yttrium (90Y) antiferritin polyclonal antibodies
Yttrium(90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10
OncoTherapy Science, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 326 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2822
Cea-Cide 8/3/1999 Treatment of ovarian carcinoma. Immunomedics, Inc.2823
Zalcitabine n/a 12/9/1986 Treatment of AIDS.2824
n/a 4/15/2015 Thar Pharmaceuticals2825
zoledronic acid Zometa, Reclast, Aclasta 5/6/20132826
Zosuquidar trihydrochloride n/a 12/15/20052827
n/a 9/17/20082828
n/a 11/4/20032829
n/a 2/14/2014 Daiichi Sankyo, Inc.
Yttrium-90 radiolabeled humanized monoclonal anti-carcinoembroyonic antigen IgG antibody
National Cancer Institute, DCT
zoledronate D,L-lysine monohydrate (ZLM)
Treatment of complex regional pain syndrome (CRPS)
Treatment of complex regional pain syndrome (CRPS).
Axsome Therapeutics, Inc.
Treatment of acute myeloid leukemia
Kanisa Pharmaceuticals, Inc.
[131I]-N-(2-(diethylamino) ethyl-4-(4 flurobenzamido)-5-(iodo)-2-methoxybenzamide
Treatment of metastatic melanoma, stages IIB, IIC, III and IV
Molecular Insight Pharmaceuticals, Inc. (Progenics Subsidiary)
[5,10,15,20-tetrakis(1,3-diethylimidazolium-2-yl)porphyrinato] manganese(III)pentachloride
Treatment of Amyotrophic Lateral Sclerosis
Aeolus Pharmaceuticals, Inc.
[5-(5-Chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride salt
Treatment of pigmented villonodular synovitis/giant cell tumor of the tendon sheath
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 327 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2830
n/a 10/20/2014 Treatment of pancreatic cancer Synovo GmbH2831
n/a 5/14/2014 Angiochem, Inc.2832
n/a 1/12/2011 Tocagen, Inc.2833
[F18] florilglutamic acid n/a 7/13/2016 Piramal Imaging S.A.2834
[F18] florilglutamic acid n/a 7/13/2016 Piramal Imaging S.A.2835
n/a 1/12/20092836
[met5]-enkephalin Opioid Growth Factor 1/24/2013 Treatment of pancreatic cancer TNI BioTech, Inc.
[5-Amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxy-propoxy)-phenyl]-methanone
[a-N-(2'succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly-[epsilon-N-(2'succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[epsilon-N-(2'succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr
Treatment of glioblastoma multiforme
[AC3-yCD2(V)] and Flucytosine (5-FC XR)
Treatment of glioblastoma multiforme
Diagnostic for the management of hepatocellular carcinoma
For the diagnosis of hepatocellular carcinoma
[Lu-177]-DOTA-Tyr3-Octreotate
Treatment of gastro-entero-pancreatic neuroendocrine tumors
Advanced Accelerator Applications
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 328 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2837
n/a 4/15/2015 Angiochem, Inc.2838
n/a 4/10/2014 Treatment of ovarian cancer2839
Nicord 4/28/2014 Gamida Cell Ltd2840
Dysport(R) 12/5/19912841
acetylcysteine Acetadote 10/19/20012842
Cetylev 2/24/20152843
adalimumab Humira 3/21/2005 AbbVie Inc.2844
adalimumab Humira 10/19/2006 AbbVie, Inc.
[α⿿N-(2⿿succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly-[ε⿿N-(2⿿succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[ε⿿N-(2⿿succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr
Treatment of breast cancer patients with brain metastases
{2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl](3H-benzo[f]azepin-4-yl)}-N,N-dipropycarboxamide
VentiRx Pharmaceuticals, Inc.
For the treatment of acute myeloid leukemia
Treatment of essential blepharospasm.
Ipsen Biopharmaceuticals, Inc.
For the intravenous treatment of moderate to severe acetaminophen overdose
Cumberland Pharmaceuticals, Inc.
acetylcysteine effervescent tablets for oral solution
Preventing hepatic injury from acetaminophin overdose
Arbor Pharmaceuticals, Inc.
Treatment of juvenile rheumatoid arthritis
Treatment of pediatric Crohn's disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 329 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2845adalimumab Humira 3/21/2005 AbbVie Inc.
2846
adalimumab Humira 5/13/2014 AbbVie, Inc.2847
adalimumab Humira 5/13/2015 AbbVie, Inc.2848
afatinib Gilotrif(R) 8/3/20152849
afatinib Gilotrif 12/3/20122850
Albendazole Albenza 1/17/1996
Treatment of juvenile rheumatoid arthritis
Treatment of non-infectious intermediate, posterior, or pan-uveitis, or chronic non-infectious anterior uveitis
Treatment of moderate to severe hidradenitis suppurativa (Hurley stage 2 and Hurley stage 3 disease)
Treatment of non-small cell lung cancer with squamous histology.
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC).
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of hydatid disease (cystic echinococcosis due to E. granulosus larvae or alveolar echinococcosis due to E. multilocularis larvae).
Amedra Pharmaceuticals LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 330 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2851
Albendazole Albenza 1/18/19962852
Aldesleukin Proleukin 9/10/1996 Chiron Corporation2853
Aldesleukin Proleukin 9/14/1988 Chiron Corporation2854
alectinib Alecensa 1/27/2015 Genentech, Inc.2855
alemtuzumab Campath 10/20/1997 Genzyme Corporation2856
Alglucerase injection Ceredase 3/11/1985 Genzyme Corporation2857
Alitretinoin Panretin 3/24/1998 Eisai, Inc
Treatment of neurocysticercosis due to Taenia solium as: 1) chemotherapy of parenchymal, subarachnoidal and racemose (cysts in spinal fluid) neurocysticercosis in symptomatic cases and 2) prophylaxis of epilepsy and other sequelae in asymptomatic neurocysticercosis.
Amedra Pharmaceuticals LLC
Treatment of metastatic melanoma.
Treatment of metastatic renal cell carcinoma.
Treatment of ALK-positive non-small cell lung cancer
Treatment of chronic lymphocytic leukemia.
For replacement therapy in patients with Gaucher's disease type I.
Treatment of AIDS-related Kaposi's sarcoma.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 331 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2858
Allopurinol sodium Aloprim For Injection 10/16/19922859
Prolastin 12/7/1984 Bayer Corporation2860
Altretamine Hexalen 2/9/19842861
ambrisentan Letairis 7/16/2004 Gilead Colorado2862
Amifostine Ethyol 5/30/19902863
Amifostine Ethyol 5/12/19982864
Aminosalicylic acid Paser Granules 2/19/1992
Management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy which causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy.
Catalytica Pharmaceuticals, Inc
Alpha1-proteinase inhibitor (human)
For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state.
Treatment of advanced adenocarcinoma of the ovary.
Medimmune Oncology, Inc.
Treatment of pulmonary arterial hypertension
For use as a chemoprotective agent for cisplatin in the treatment of advanced ovarian carcinoma.
Medimmune Oncology, Inc.
Reduction of the incidence of moderate to severe xerostomia in patients undergoing post-operative radiation treatment for head and neck cancer.
Medimmune Oncology, Inc.
Treatment of tuberculosis infections
Jacobus Pharmaceutical Company
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 332 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2865
Amiodarone HCl Cordarone 3/16/19942866
Amphotericin B lipid complex Abelcet 12/5/19912867
Anagrelide Agrylin 1/27/19882868
anakinra Kineret 8/19/20102869
n/a 7/29/20082870
Anthrax Vaccine Adsorbed Biothrax 4/11/20142871
Feiba 4/12/2013 Baxalta US, Inc.2872
Antihemophilic factor (human) Alphanate 1/5/1996 Grifols Biologicals Inc.
For the acute treatment and prophylaxis of life-threatening ventricular tachycardia or ventricular fibrillation.
Wyeth-Ayerst Laboratories
Treatment of invasive fungal infections.
Liposome Company, Inc.
Treatment of essential thrombocythemia.
Roberts Pharmaceutical Corp.
Treatment of cryopyrin-associated periodic syndromes
Swedish Orphan Biovitrum AB (publ)
anthrax immune globulin (human)
Treatment of inhalational anthrax
Cangene Corp. - Emergent Biosolutions
For post-exposure prophylaxis of anthrax disease resulting from suspected or confirmed Bacillus anthracis exposure
Emergent BioDefense Operations Lansing LLC
anti-inhibitor coagulant complex
Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in hemophilia A and B patients with inhibitors
Treatment of von Willebrand's disease
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 333 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2873
Kogenate 9/25/1989 Bayer Corporation2874
Refacto, Xyntha 2/8/19962875
Eloctate 11/23/2010 Treatment of hemophilia A Biogen Idec Inc.2876
Obizur 3/16/2004 Baxalta US, Inc.2877
Humate-P 10/16/1992 CSL Behring2878
Antithrombin III (human) Thrombate Iii 11/26/1984 Bayer Corporation
Antihemophilic factor (recombinant)
Prophylaxis and treatment of bleeding in individuals with hemophilia A or for prophylaxis when surgery is required in individuals with hemophilia A.
Antihemophilic factor (recombinant)
For the control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
Wyeth Pharmaceuticals, Inc.
antihemophilic factor (recombinant), Fc fusion protein
antihemophilic factor (recombinant), porcine sequence
Treatment and prevention of episodic bleeding in patients with inhibitor antibodies to human coagulation factor VIII
Antihemophilic factor/von Willebrand factor complex (human), dried, pasteurized
Treatment of patients with von Willebrand's disease
For replacement therapy in congenital deficiency of AT-III for prevention and treatment of thrombosis and pulmonary emboli.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 334 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2879
Antithrombin III (human) Atnativ 2/8/19852880
Anavip 1/29/20042881
Crofab 1/12/1994 Protherics, Inc.2882
Apomorphine HCl Apokyn 4/22/1993 US WorldMeds, LLC
For the treatment of patients with hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or when they suffer from thromboembolism.
Pharmacia & Upjohn AB
Antivenin crotaline (pit-viper) equine immune F(ab)2)
Treatment of envenomation by Crotaline snakes
Rare Disease Therapeutics, Inc.
Antivenin, crotalidae polyvalent immune Fab (ovine)
Treatment of envenomations inflicted by North American crotalid snakes.
Treatment of the on-off fluctuations associated with late-stage Parkinson's disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 335 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2883
Aprotinin Trasylol 11/17/1993 Bayer Corporation2884
aripiprazole Abilify 1/25/20062885
Arsenic trioxide Trisenox 3/3/1998 Cephalon2886
artemether/lumefantrine Coartem 8/31/20072887
Asfotase alfa Strensiq 9/12/2008 Treatment of hypophosphatasia
For prophylactic use to reduce perioperative blood loss and the homologous blood transfusion requirement in patients undergoing cardiopulmonary bypass surgery in the course of repeat coronary artery bypass graft surgery, and in selected cases of primary coronary artery bypass graft surgery where the risk of bleeding is especially high (impaired hemostasis) or where transfusion is unavailable or unacceptable.
Treatment of Tourette's syndrome
Otsuka Pharmaceutical Development
Treatment of acute promyelocytic leukemia.
For the treatment of infections due to Plasmodium falciparum or mixed infections including P. falciparum.
Novartis Pharmaceuticals
Alexion Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 336 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2888
Atovaquone Mepron 8/14/19912889
Atovaquone Mepron 9/10/1990 Glaxo Wellcome Inc.2890
azacitidine Vidaza 12/3/2001 Celgene Corporation2891
aztreonam Cayston 3/12/20022892
baclofen Lioresal Intrathecal 11/10/1987 Medtronic, Inc.2893
Balsalazide disodium Colazal 8/12/2005
Prevention of Pneumocystis carinii pneumonia (PCP) in high-risk, HIV-infected patients defined by a history of one or more episodes of PCP and/or a peripheral CD4+ (T4 helper/inducer) lymphocyte count less than or equal to 200/mm3.
Glaxo Wellcome Research and Development
Treatment of AIDS associated Pneumocystis Carinii Pneumonia.
Treatament of myelodysplastic syndromes
Inhalation therapy for control of gram-negative bacteria in the respiratory tract of patients with cystic fibrosis
Gilead Sciences (formerly Corus Pharma)
Treatment of intractable spasticity caused by spinal cord injury, multiple sclerosis, and other spinal diseases (including spinal ischemia, spinal tumor, transverse myelitis, cervical spondylosis, and degenerative myelopathy).
Treatment of pediatric patients with ulcerative colitis
Salix Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 337 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2894
Basiliximab Simulect 12/12/19972895
Sirturo 1/10/2005 Treatment of active tuberculosis2896
belatacept Nulojix 2/20/20082897
Belinostat Beleodaq 9/3/20092898
Bendeka 7/2/20142899
Bendeka 7/2/2014
Prophylaxis of solid organ rejection.
Novartis Pharmaceuticals Corporation
bedaquiline; (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol
Janssen Research & Development, LLC
Prophylaxis of organ rejection in renal allograft recipients
Bristol-Myers Squibb Company
Treatment of peripheral T-cell lymphoma (PTCL).
Spectrum Pharmaceuticals, Inc.
bendamustine for 50 ml admixture
Treatment of chronic lymphocytic leukemia
Eagle Pharmaceuticals, INc.
bendamustine for 50ml admixture
Treatment of Follicular Lymphoma, Small Lymphocytic Lymphoma, Lymphoplasmacytic Lymphoma, Splenic Marginal Zone Lymphoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-Associated Lymphoma Tissue (MALT), and Nodal Marginal Zone Lymphoma (Collectively Indolent B-cell Non-Hodgkin's Lymphoma)
Eagle Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 338 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2900
bendamustine hydrochloride Treanda 11/26/2013 Cephalon, Inc.2901
Bendamustine hydrochloride Treanda 8/17/2007 Cephalon, Inc.2902
Benzoate and phenylacetate Ucephan 1/21/1986 ImmunexImmunex2903
benzoate/phenylacetate Ammonul 11/22/1993
Treatment of Follicular Lymphoma, Small Lymphocytic Lymphoma, Lymphoplasmacytic Lymphoma, Splenic Marginal Zone Lymphoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-Associated Lymphoma Tissue (MALT), and Nodal Marginal Zone Lymphoma (Collectively Indolent B-cell Non-Hodgkin's Lymphoma)
Treatment of chronic lymphocytic leukemia
For adjunctive therapy in the prevention and treatment of hyperammonemia in patients with urea cycle enzymopathy due to carbamylphosphate synthetase, ornithine, transcarbamylase, or argininosuccinate synthetase deficiency.
Treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle.
Medicis Pharmaceutical Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 339 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2904
Beractant 2/5/1986 Ross Laboratories2905
Beractant 2/5/1986 Ross Laboratories2906
Betaine Cystadane 5/16/1994 Treatment of homocystinuria. Jazz Pharmaceuticals2907
bevacizumab Avastin 11/6/2003 Genentech, Inc.2908
bevacizumab Avastin 11/2/2010 Genentech, Inc.2909
bevacizumab Avastin 2/9/2006 Genentech, Inc.2910
bevacizumab Avastin 11/23/2010 Genentech, Inc.2911
bevacizumab Avastin 5/26/2006 Treatment of malignant glioma Genentech, Inc.2912
Bexarotene Targretin 6/18/1999 Eisai, Inc.2913
biocarbonate infusate Normocarb Hf 8/9/2005 Dialysis Solutions, Inc.2914
Bleomycin sulfate Blenoxane 9/17/19932915
blinatumomab Blincyto 5/16/2008 Amgen, Inc.
Survanta Intratracheal Suspension
Treatment of neonatal respiratory distress syndrome
Survanta Intratracheal Suspension
Treatment of neonatal respiratory distress syndrome
Treatment of renal cell carcinoma
Treatment of primary peritoneal carcinoma.
Therapeutic treatment of patients with ovarian cancer
Treatment of fallopian tube carcinoma
Treatment of cutaneous T-cell lymphoma.
Use in the management of patients undergoing continuous renal replacement therapy with hemofiltration
Treatment of malignant pleural effusion.
Bristol-Myers Squibb Pharmaceutical Research Institute
Treatment of acute lymphocytic leukemia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 340 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2916
bortezomib Velcade 5/30/20122917
bortezomib Velcade 1/15/2003 Treatment of multiple myeloma2918
bortezomib Velcade 1/15/2003 Treatment of multiple myeloma2919
bortezomib Velcade 1/15/2003 Treatment of multiple myeloma2920
bortezomib Velcade 5/30/20122921
Bosentan Tracleer 10/6/20002922
bosutinib Bosulf 2/24/20092923
Botulinum toxin type A Botox 3/22/1984 Allergan, Inc.2924
Botulinum toxin type A Botox 3/22/1984 Allergan, Inc.2925
Botulinum toxin type A Dysport 10/20/1999 Ipsen Limited2926
Botulinum toxin type A Botox 8/20/1986 Treatment of cervical dystonia. Allergan, Inc.
Treatment of mantle cell lymphoma.
Millennium Pharmaceuticals, Inc.
Millennium Pharmaceuticals, Inc.
Millennium Pharmaceuticals, Inc.
Millennium Pharmaceuticals, Inc.
Treatment of mantle cell lymphoma.
Millennium Pharmaceuticals, Inc.
Treatment of pulmonary arterial hypertension.
Actelion Life Sciences Ltd.
Treatment of chronic myelogenous leukemia
Wyeth Pharmaceuticals, Inc.
Treatment of strabismus and blepharospasms
Treatment of strabismus and blepharospasms
Treatment of dynamic muscle contractures in pediatric cerebral palsy patients.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 341 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2927Botulinum toxin type B Myobloc 1/16/1992 Treatment of cervical dystonia.
2928
n/a 6/29/2011 Treatment of botulism. Cangene Corporation2929
Botulism immune globulin Babybig 1/31/1989 Treatment of infant botulism.2930
brentuximab vedotin Adcetris 10/23/2008 Seattle Genetics, Inc.2931
brentuximab vedotin Adcetris 1/30/2007 Seattle Genetics, Inc.2932
brentuximab vedotin Adcetris 1/30/2007 Seattle Genetics, Inc.2933
Elliotts B Solution 8/24/1994 Lukare Medical, LLC2934
Buprenorphine hydrochloride Subutex 6/15/19942935
Suboxone 10/27/19942936
Busulfan Busulfex 7/28/1994
Soltice Neurosciences, LLC
botulism antitoxin heptavalent (A, B, C, D, E, F, G) (Equine)
California Department of Health Services
Treatment of anaplastic large cell lymphoma
Treatment of Hodgkin's lymphoma
Treatment of Hodgkin's lymphoma
Buffered intrathecal electrolyte/dextrose injection
For use as a diluent in the intrathecal administration of methotrexate and cytarabine for the prevention or treatment meningeal leukemia and lymphocytic lymphoma
Treatment of opiate addiction in opiate users.
Reckitt Benckiser Pharmaceuticals, Inc.
buprenorphine in combination with naloxone
Treatment of opiate addiction in opiate users
Reckitt Benckiser Pharmaceuticals, Inc.
As preparative therapy in the treatment of malignancies with bone marrow transplantation.
Otsuka Pharmaceutical Company
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 342 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2937C1 esterase inhibitor (human) Cinryze(R) 7/16/2004 Treatment of angioedema Shire
2938
Ruconest 2/23/1999 Santarus, Inc.2939
Berinert 10/16/1992 CSL Behring LLC2940
cabozantinib Cometriq 11/29/2010 Exelixis, Inc.2941
Caffeine Cafcit 9/20/19882942
Calcitonin-human for injection Cibacalcin 1/20/19872943
Calcium acetate Phos-Lo 12/22/19882944
calfactant Infasurf 6/7/1985 ONY, Inc.2945
canakinumab Ilaris 9/4/2012
C1-esterase inhibitor (recombinant)
Treatment of (acute attacks of) angioedema caused by hereditary or acquired C1-esterase inhibitor deficiency.
C1-esterase-inhibitor, human, pasteurized
Prevention and/or treatment of acute attacks of hereditary angioedema.
Treatment of follicular, medullary and anaplastic thyroid carcinoma and metastatic or locally advanced papillary thyroid cancer.
Treatment of apnea of prematurity.
O.P.R. Development, L.P.
Treatment of symptomatic Paget's disease (osteitis deformans).
Novartis Pharmaceutical Corporation
Treatment of hyperphosphatemia in end stage renal failure.
Fresenius Medical Care North America
Treatment and prevention of respiratory failure due to pulmonary surfactant deficiency in preterm infants.
Treatment of TNF-receptor associated periodic syndrome (TRAPS)
Novartis Pharmaceuticals Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 343 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2946
canakinumab Ilaris 9/30/20082947
canakinumab Ilaris 12/5/20132948
canakinumab Ilaris 12/18/20072949
canakinumab Ilaris 12/5/20132950
capsaicin Qutenza 5/22/20092951
carfilzomib Kyprolis 1/18/2008 Treatment of multiple myeloma2952
carglumic acid Carbaglu 1/20/1998 Orphan Europe SARL2953
Centruroides immune F(ab)2 Anascorp 6/12/20002954
Fabrazyme 1/19/1988 Treatment of Fabry's disease. Genzyme Corporation
Treatment of pediatric (age 16 and under) juvenile rheumatoid arthritis.
Novartis Pharmaceuticals Corporation
Treatment of familial mediterranean fever
Novartis Pharmaceuticals Corporation
Treatment of cryopyrin-associated periodic syndromes
Novartis Pharmaceuticals Corporation
Treatment of hyperimmunoglobulinemia D and periodic fever syndrome
Novartis Pharmaceuticals Corporation
Management of neuropathic pain in patients with postherpetic neuralgia
Acorda Therapeutics, Inc.
Onyx Therapeutics, Inc.
Treatment of N-acetylglutamate synthetase deficiency.
Treatment of scorpion envenomations requiring medical attention.
Rare Disease Therapeutics, Inc.
Ceramide trihexosidase/alpha-galactosidase A
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 344 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2955
ceritinib Zykadia 9/27/20132956
cetuximab Erbitux 7/3/20002957
chenodiol Chenix 9/21/1984 Solvay2958
cholic acid Cholbam 7/18/2003 Retrophin, Inc.2959
cinacalcet Sensipar 5/12/2003 Amgen, Inc.
Treatment of patients with non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase(ALK)-positive
Novartis Pharmaceuticals Corp.
Treatment of squamous cell cancer of the head and neck in patients who express epidermal growth factor receptor
ImClone Systems Incorporated
For patients with radiolucent stones in well opacifying gallbladders, in whom elective surgery would be undertaken except for the presence of increased surgical risk due to systemic disease or age
Treatment of inborn errors of cholesterol and bile acid synthesis and metabolism
Treatment of hypercalcemia in patients with parathyroid carcinoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 345 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2960
cinacalcet Sensipar 4/30/2010 Amgen, Inc.2961
cinacalcet Sensipar 4/30/2010 Amgen, Inc.2962
Renacidin Irrigation 8/28/1989 United-Guardian, Inc.2963
Cladribine Leustatin Injection 11/15/1990 Treatment of hairy cell leukemia.2964
clobazam Onfi 12/18/2007 Lundbeck, Inc.2965
clofarabine Clolar 2/7/2002 Genzyme Corporation
Treatment of hypercalcemia in patients with primary hyperparathyroidism for whom parathyroidectomy would be indicated on the basis of serum calcium levels, but who are unable to undergo surgery
Treatment of hypercalcemia in patients with primary hyperparathyroidism for whom parathyroidectomy would be indicated on the basis of serum calcium levels, but who are unable to undergo surgery
Citric acid, glucono-delta-lactone and magnesium carbonate
Treatment of renal and bladder calculi of the apatite or struvite variety.
R. W. Johnson Pharmaceutical Research Institute
Treatment of Lennox-Gastaut Syndrome
Treatment of acute lymphoblastic leukemia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 346 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2966
Clofazimine Lamprene 6/11/19842967
Clonidine Duraclon 1/24/19892968
Coagulation factor IX Mononine 6/27/19892969
Coagulation Factor IX (human) Alphanine 7/5/1990
Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum.
Novartis Pharmaceutical Corporation
For continous epidural administration as adjunctive therapy with intraspinal opiates for the treatment of pain in cancer patients tolerant to, or unresponsive to, intraspinal opiates.
Roxane Laboratories, Inc.
Replacement treatment and prophylaxis of the hemorrhagic complications of hemophilia B.
Armour Pharmaceutical Company
For use as replacement therapy in patients with hemophilia B for the prevention and control of bleeding episodes, and during surgery to correct defective hemostasis.
Alpha Therapeutic Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 347 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2970
Rixubis 10/31/2012 Baxalta US, Inc.2971
Benefix 10/3/1994 Treatment of hemophilia B.2972
Alprolix 10/30/2008 Biogen Idec Inc.2973
Novoseven 7/21/2004 Novo Nordisk, Inc.2974
Novoseven 9/10/2004 Novo Nordisk, Inc.2975
Novoseven 6/6/1988 Novo Nordisk, Inc.
coagulation factor IX (recombinant)
Prophylactic use to prevent or reduce the frequency of bleeding episodes in patients with hemophilia B (routine prophylaxis in patients where there is no evidence or suspicion of bleeding)
Coagulation Factor IX (recombinant)
Genetics Institute, Inc.
Coagulation factor IX (recombinant), Fc fusion protein
Control and prevention of hemorrhagic episodes in patients with hemophilia B (congenital factor IX deficiency or Christmas disease)
Coagulation factor VIIa (recombinant)
Prevention of bleeding episodes in patients with acquired inhibitors to Factor VIII or Factor IX
Coagulation factor VIIa (recombinant)
Treatment of bleeding episodes in patients with congenital factor VII deficiency
Coagulation factor VIIa (recombinant)
Treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 348 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2976
Novoseven 9/10/2004 Novo Nordisk, Inc.2977
Novoseven 6/18/2004 Novo Nordisk, Inc.2978
Novoseven 7/16/2004 Novo Nordisk, Inc.2979
Novoseven Rt 6/18/2004 Novo Nordisk Inc.2980
Tretten 11/6/2003 Novo Nordisk, Inc.2981
cobimetinib n/a 1/31/2014 Genentech, Inc.2982
n/a 12/19/20072983
colchicine Colcrys 9/25/2007
Coagulation factor VIIa (recombinant)
Prevention of bleeding episodes in patients with congenital Factor VII deficiency
Coagulation factor VIIa (recombinant)
Prevention of bleeding episodes in patients with hemophilia A or B, with or without inhibitors
Coagulation Factor VIIa (Recombinant)
Treatment of bleeding episodes in patients with acquired inhibitors to Factor VIII or Factor IX
coagulation factor VIIa (recombinant)
Treatment of bleeding episodes in Glanzmann's thrombasthenia
coagulation factor XIII A-subunit (recombinant)
Prophylaxis of bleeding associated with congential factor XIII deficiency
Treatment of stage IIb, IIc, III, and IV melanoma with BRAFV600 mutation
coccidioidin SD Skin Test Antigen
For the diagnosis of Coccidioidomycosis
Allermed Labortories, Inc.
Treatment of familial Mediterranean fever
AR Holding Company, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 349 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2984
10/20/1989 Glaxo Wellcome Inc.2985
Xiaflex 5/23/19962986
Xiaflex 3/12/1996 Treatment of Peyronie's disease.2987
n/a 10/9/20142988
Corticorelin ovine triflutate Acthrel 11/24/19892989
crizotinib Xalkori 9/13/2010 Pfizer, Inc.
Colfosceril palmitate, cetyl alcohol, tyloxapol
Exosurf Neonatal For Intratracheal Suspension
1. Prevention of hyaline membrane disease, also known as respiratory distress syndrome, in infants born at 32 weeks gestation or less 2. prevention of hyaline membrane disease (HMD), also known as respiratory distress syndrome (RDS), in infants born at 32 weeks gestation or less and in the treatment of established HMO at all gestational ages.
collagenase clostridium histolyticum
Treatment of advanced (involutional or residual stage) Dupuytren's disease.
Auxilium Pharmaceuticals, Inc.
collagenase clostridium histolyticum
Auxilium Pharmaceuticals, Inc.
Combination of nivolumab and ipilimumab
Treatment of Stage IIb to Stage IV melanoma
Bristol-Myers Squibb Company
For use in differentiating pituitary and ectopic production of ACTH in patients with ACTH-dependent Cushings syndrome.
Ferring Laboratories, Inc.
Treatment of ALK-positive, MET-positive, or ROS-positive non-small cell lung cancer
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 350 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
2990 Cromolyn sodium Gastrocrom 3/8/1984 Treatment of mastocytosis. Fisons Corporation2991
7/24/1985 Fisons Corporation2992
Cysteamine Cystagon 1/25/19912993
cysteamine enteric coated Procysbi 10/24/2006 Treatment of cystinosis Horizon Orphan LLC2994
cysteamine enteric coated Procysbi 10/24/2006 Treatment of cystinosis Horizon Orphan LLC2995
Cysteamine hydrochloride Cystaran 8/19/19972996
Cytarabine liposomal Depocyt 6/2/19932997
Cytogam 8/3/1987 CSL Behring LLC2998
dabrafenib Tafinlar 1/12/20112999
Daclizumab Zenapax 3/5/19933000
dalfampridine Ampyra 6/2/1987 Acorda Therapeutics3001
Ryanodex 8/16/2013
Cromolyn sodium 4% ophthalmic solution
Opticrom 4% Ophthalmic Solution
Treatment of vernal keratoconjunctivitis.
Treatment of nephropathic cystinosis.
Mylan Laboratories, Inc.
Treatment of corneal cystine crystal accumulation in cystinosis patients.
Sigma-Tau Pharmaceuticals, Inc.
Treatment of neoplastic meningitis.
Pacira Pharmaceuticals, Inc.
Cytomegalovirus immune globulin (human)
Prevention or attenuation of primary cytomegalovirus disease in immunosuppressed recipients of organ transplants.
Treatment BRAF V600 mutation positive Stage IIB through IV melanoma
Novartis Pharmaceuticals Corp.
Prevention of acute renal allograft rejection.
Hoffmann-La Roche, Inc.
Relief of symptoms of multiple sclerosis
dantrolene sodium suspension for injection
Treatment of malignant hyperthermia syndrome
Eagle Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 351 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3002daratumumab Darzalex 5/6/2013 Treatment of multiple myeloma. Janssen Biotech, Inc.
3003daratumumab Darzalex 5/6/2013 Treatment of multiple myeloma. Janssen Biotech, Inc.
3004
Dasatinib Sprycel 11/18/20053005
Dasatinib Sprycel 11/28/20053006
Daunoxome 5/14/19933007
decitabine Dacogen 3/8/19993008
Deferasirox Exjade 11/21/20023009
deferasirox Exjade 2/24/20153010
deferiprone Ferriprox 12/12/20013011
defibrotide Defitelio 5/21/20033012
denileukin diftitox Ontak 8/21/1996 Eisai, Inc.
Treatment of Philadelphia-positive acute lymphoblastic leukemia
Bristol-Myers Squibb Company
Treatment of chronic myelogenous leukemia
Bristol-Myers Squibb Company
Daunorubicin citrate liposome injection
Treatment of patients with advanced HIV-associated Kaposi's sarcoma.
NeXstar Pharmaceuticals, Inc.
Treatment of myelodysplastic syndromes.
Otsuka Pharmaceutical Development
Treatment of chronic iron overload in patients with transfusion-dependent anemias
Novartis Pharmaceuticals Corporation
Treatment of chronic iron overload in alpha-thalassemia
Novartis Pharmaceuticals Corporation
Treatment of iron overload in patients with hematologic disorders requiring chronic transfusion therapy
ApoPharma, Inc. A Division of
For the treatment of hepatic veno-occlusive disease
Jazz Pharmaceuticals, Inc.
Treatment of patients with cutaneous T-cell lymphoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 352 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3013denosumab Xgeva 12/20/2010 Amgen, Inc.
3014denosumab Xgeva 9/11/2013 Amgen, Inc.
3015
Desmopressin acetate n/a 1/22/1991 Aventis Behring L.L.C.3016
Ozurdex 9/11/1998 Allergan3017
Dexrazoxane Zinecard 12/17/1991 Pharmacia & Upjohn3018
dexrazoxane Totect(R) 3/25/20043019
n/a 2/25/19923020
dichlorphenamide n/a 9/2/2010 Taro Pharmaceuticals
Treatment of patients with giant cell tumor of bone
Treatment of hypercalcemia in malignancy
Treatment of mild hemophilia A and von Willebrand's disease.
dexamethasone intravitreal implant
Treatment of non-infectious ocular inflammation of the posterior segment in patients with intermediate, posterior, and panuveitis
For the prevention of cardiomyopathy associated with doxorubicin administration.
Treatment of anthracycline extravasation during chemotherapy
Clinigen Healthcare Ltd.
Diazepam viscous solution for rectal administration
For the management of selected, refractory, patients with epilepsy, on stable regimens of antiepileptic drugs (AEDs), who require intermittent use of diazepam to control bouts of increased seizure activity.
Valeant Pharmaceuticals
Treatment of primary periodic paralyses
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 353 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3021
n/a 4/28/20043022
difluprednate Durezol 9/30/20083023
Digoxin immune FAB (Ovine) Digibind 11/1/1984 Glaxo Wellcome Inc.3024
dinutuximab Unituxin 12/20/2010 Treatment of neuroblastoma3025
Dornase alfa Pulmozyme 1/16/1991 Genentech, Inc.3026
Doxil 12/29/2004 Treatment of multiple myeloma3027 Doxorubicin liposome Doxil 11/4/1998 Treatment of ovarian cancer. Alza Corporation3028
Dronabinol Marinol 1/15/1991
Diethylenetriaminepentaacetic acid (DTPA)
Treatment of patients with known or suspected internal contamination with plutonium, americium, or curium to increase the rates of elimination.
Hameln Pharmaceuticals gmbh
Treatment of endogenous and traumatic anterior uveitis and panuveitis.
Alcon Pharmaceuticals. Ltd.
Treatment of potentially life threatening digitalis intoxication in patients who are refractory to management by conventional therapy.
United Therapeutics Corporation
To reduce mucous viscosity and enable the clearance of airway secretions in patients with cystic fibrosis.
doxorubicin HCL liposome injection
Johnson & Johnson Pharmaceutical Research & Dev.
For the stimulation of appetite and prevention of weight loss in patients with a confirmed diagnosis of AIDS.
Unimed Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 354 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3029
droxidopa Northera 1/17/2007 Lundbeck LLC3030 ecallantide Kalbitor 2/4/2003 Treatment of angioedema Dyax Corp.3031 ecallantide Kalbitor 2/4/2003 Treatment of angioedema Dyax Corp.3032
eculizumab Soliris 8/20/20033033
eculizumab Soliris 4/29/20093034
Eflornithine HCl Ornidyl 4/23/19863035
eliglustat Cerdelga 9/17/2008 Genzyme Corporation3036
elosulfase alfa Vimizim 5/15/20093037
elotuzumab Empliciti 9/1/2011 Treatment of multiple myeloma3038
eltrombopag Promacta 11/8/2013 Treatment of aplastic anemia
Treatment of neurogenic symptomatic orthostatic hypotension in patients with primary autonomic failure, dopamine-beta-hydroxylase deficiency, and nondiabetic autonomic neuropathy.
Treatment of paroxysmal nocturnal hemoglobinuria
Alexion Pharmaceuticals, Inc.
Treatment of atypical hemolytic uremic syndrome
Alexion Pharmaceuticals, Inc.
Treatment of Trypanosoma brucei gambiense infection (sleeping sickness).
Hoechst Marion Roussel
Treatment of Type I Gaucher disease
Use in the treatment of mucopolysaccharidosis (MPS) Type IV A (Morquio A syndrome)
BioMarin Pharmaceutical Inc.
Bristol-Myers Squibb Co.
Novartis Pharmaceuticals Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 355 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3039
eltrombopag Promacta 5/5/20083040
Epirubicin Ellence 9/14/1999 Treatment of breast cancer.3041
Epoetin alfa Epogen 4/10/1986 Amgen, Inc.3042
Epoetin alfa Epogen 7/1/1991 Amgen, Inc.3043
epoprostenol Flolan 3/22/1999 GlaxoSmithKline3044
Epoprostenol Flolan 9/25/1985 Glaxo Wellcome Inc.3045
eribulin mesylate Halaven 5/14/2012 Treatment of soft tissue sarcoma Eisai Inc.3046
Erwinia L-asparaginase Erwinase 7/30/19863047
etanercept Enbrel 10/27/1998 Immunex Corporation
Treatment of idiopathic thrombocytopenia purpura
Novartis Pharmaceuticals Corp.
Pharmacia & Upjohn Company
Treatment of anemia associated with end stage renal disease.
Treatment of anemia associated with HIV infection or HIV treatment.
Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease.
Treatment of primary pulmonary hypertension.
Treatment of acute lymphocytic leukemia.
Jazz Pharmaceuticals, Inc.
Reduction in signs and symptoms of moderately to severely active polyarticular-course juvenile rheumatoid arthritis in patients who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 356 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3048eteplirsen Exondys 51 10/23/2007
3049
Ethanolamine oleate Ethamolin 3/22/1984 QOL Medical3050
ethiodized oil injection Lipiodol 9/26/2013 Guerbet LLC3051
Etidronate disodium Didronel 3/21/1986 MGI Pharma, Inc.3052
everolimus Afinitor 2/14/20083053
everolimus Afinitor 6/8/2009
Treatment of Duchenne Muscular Dystrophy.
Sarepta Therapeutics, Inc.
Treatment of patients with esophageal varices that have recently bled, to prevent rebleeding.
Management of patients with known hepatocellular carcinoma (HCC)
Treatment of hypercalcemia of malignancy inadequately managed by dietary modification and/or oral hydration.
Treatment of neuroendocrine tumors
Novartis Pharmaceuticals Corporation
Treatment of tuberous sclerosis complex including TSC-associated subependymal giant cell astrocytoma (SEGA), TSC-associated angiomyolipoma and TSC-associated lymphangioleiomyomatosis (LAM)
Novartis Pharmaceuticals Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 357 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3054
everolimus Afinitor 6/8/20093055
everolimus Afinitor 2/14/20083056
evolocumab Repatha 9/12/2013 Amgen Inc.3057
Exemestane Aromasin 9/19/1991 Pharmacia & Upjohn3058
Corifact 1/16/1985 CSL Behring LLC3059
Corifact 1/16/1985 CSL Behring LLC3060
Felbamate Felbatol 1/24/1989 Wallace Laboratories3061
Filgrastim Neupogen 10/1/1990 Amgen, Inc.
Treatment of tuberous sclerosis complex including TSC-associated subependymal giant cell astrocytoma (SEGA), TSC-associated angiomyolipoma and TSC-associated lymphangioleiomyomatosis (LAM)
Novartis Pharmaceuticals Corporation
Treatment of neuroendocrine tumors
Novartis Pharmaceuticals Corporation
Treatment of homozygous familial hypercholesterolemia
Treatment of advanced breast cancer in postmenopausal women whose disease has progressed following tamoxifen therapy.
factor XIII concentrate (human)
Treatment of congenital factor XIII deficiency
factor XIII concentrate (human)
Treatment of congenital factor XIII deficiency
Treatment of Lennox-Gastaut syndrome.
Treatment of neutropenia associated with bone marrow transplants.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 358 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3062
Filgrastim Neupogen 7/17/1995 Amgen, Inc.3063
filgrastim Neupogen 11/20/2013 Amgen, Inc.3064
Filgrastim Neupogen 11/7/1990 Amgen, Inc.3065
Filgrastim Neupogen 11/7/1996 Amgen, Inc.3066
Fludarabine phosphate Fludara 4/18/19893067
n/a 12/18/20073068
Fluocinolone Retisert 7/31/2000
For use in the mobilization of peripheral blood progenitor cells for collection in patients who will receive myeloablative or myelosuppressive chemotherapy.
Treatment of subjects at risk of developing myelosuppression after a radiological or nuclear incident
Treatment of patients with severe chronic neutropenia (absolute neutrophil count less than 500/mm3).
Reduction in the duration of neutropenia, fever, antibiotic use, and hospitalization, following induction and consolidation treatment for acute myeloid leukemia.
Treatment of chronic lymphocytic leukemia (CLL), including refractory CLL.
Berlex Laboratories, Inc.
Fludarabine phosphate oral tablets
Treatment of B-cell chronic lymphocytic leukemia
Sanofi-Aventis U.S., Inc.
Treatment uveitis involving the posterior segment of the eye.
Bausch & Lomb Pharmaceuticals, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 359 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3069
Follitropin alfa, recombinant Gonal-F 12/21/1998 EMD Serono, Inc.3070
Fomepizole Antizole 12/22/1988 Jazz Pharmaceuticals3071
Fomepizole Antizole 12/22/1988 Jazz Pharmaceuticals3072
Fosphenytoin Cerebyx 6/4/19913073
gabapentin Gralise 11/8/2010 Depomed, Inc.3074
gabapentin enacarbil Horizant 6/7/2011
For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism. AMENDED indication 6/27/00: For the induction of spermatogenesis in men with primary and secondary hypogonadotropic hypogonadism in whom the cause of infertility is not due to primary testicular failure.
Treatment of methanol or ethylene glycol poisoning.
Treatment of methanol or ethylene glycol poisoning.
For the acute treatment of patients with status epilepticus of the grand mal type.
Warner-Lambert Company
Management of postherpetic neuralgia
Treatment of postherpetic neuralgia
Arbor Pharmaceuticals, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 360 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3075
Netspot 12/31/20133076
ganciclovir Zirgan 3/22/20073077
Ganciclovir intravitreal implant Vitrasert Implant 6/7/19953078
gefitinib Iressa 8/26/20143079
Gemtuzumab ozogamicin Mylotarg 11/24/19993080
Glatiramer acetate Copaxone 11/9/1987 Treatment of multiple sclerosis.3081
glucarpidase Voraxaze 8/19/2003 BTG International Inc.
Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide
Diagnostic for the clinical management of neuroendocrine tumors
Advanced Accelerator Applications, USA
Treatment of acute herpetic keratitis (dendritic and geographic ulcers)
Sirion Therapeutics, Inc.
Treatment of cytomegalovirus retinitis.
Bausch & Lomb Surgical, Chiron Vision Products
Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer
AstraZeneca Pharmaceuticals LP
Treatment of acute myeloid leukemia
Wyeth Pharmaceuticals, Inc., a Pfizer Company
Teva Pharmaceuticals USA
Treatment of patients at risk of methotrexate toxicity
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 361 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3082
Glutamine Nutrestore 3/6/1995 Emmaus Medical, Inc.3083
glycerol phenylbutyrate Ravicti 4/27/2009 Horizon Pharma, Inc.3084
glycopyrrolate Cuvposa 6/9/2006 Shionogi, Inc.3085
Gonadorelin acetate Lutrepulse 4/22/19873086
Halofantrine Halfan 11/4/1991
For use with human growth hormone in the treatment of short bowel syndrome (nutrient malabsorption from the gastrointestinal tract resulting from an inadequate absorptive surface).
Maintenance treatment of patients with deficiencies in enzymes of the urea cycle
Treatment of pathologic (chronic moderate to severe) drooling in pediatric patients
For induction of ovulation in women with hypothalamic amenorrhea due to a deficiency or absence in the quantity or pulse pattern of endogenous GnRH secretion.
Ferring Laboratories, Inc.
Treatment of mild to moderate acute malaria caused by susceptible strains of P. falciparum and P. vivax.
SmithKline Beecham Pharmaceuticals
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 362 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3087
Hemin Panhematin 3/16/1984 Abbott Laboratories3088
Hepagam 3/24/2008 Cangene Corporation3089
Histrelin Supprelin La 11/18/20053090
Histrelin acetate Supprelin Injection 8/10/19883091
Human factor X Coagadex 11/8/20073092
Riastap 3/13/2008 CSL Behring, LLC3093
Yervoy 10/21/2010
Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.
hepatitis B immune globulin (human)
Prevention of hepatitis B recurrence following orthotopic liver transplant
Treatment of central precocious puberty
Endo Pharmaceuticals Solutions, Inc.
Treatment of central precocious puberty.
Roberts Pharmaceutical Corp.
Treatment of hereditary factor X deficiency
Bio Products Laboratory
human fibrinogen concentrate, pasteurized
Treatment of fibrinogen deficient patients.
human monoclonal anti-PA antibody
For post-exposure prophylaxis and treatment of inhalation anthrax.
Emergent Product Development Gaithersburg, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 363 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3094
Wilate 4/18/2007 Octapharma USA, Inc.3095
Hydroxocobalamin Cyanokit 11/25/2003 SERB, S.A.3096
hydroxyprogesterone caproate Makena 1/25/20073097
Hydroxyurea Droxia 10/1/19903098
ibritumomab tiuxetan Zevalin 9/6/19943099
ibrutinib Imbruvica 4/6/2012 Pharmacyclics, LLC3100
ibrutinib Imbruvica 10/15/2013 Pharmacyclics, LLC3101
ibrutinib Imbruvica 4/6/2012 Pharmacyclics, LLC3102
ibrutinib Imbruvica 5/30/2013 Pharmacyclics, LLC3103
ibrutinib Imbruvica 12/3/2012 Pharmacyclics, LLC3104
ibrutinib Imbruvica 4/6/2012 Pharmacyclics, LLC
Human plasma coagulation Factor VIII and human plasma von Willebrand Factor
Treatment of von Willebrand disease except for surgical and/or invasive procedures in patients with von Willebrand disease in whom desmopressin is either ineffective or contraindicated
Treatment of acute cyanide poisoning
Prevention of preterm birth in singleton pregnancies
KV Pharmaceutical Company
Treatment of patients with sickle cell anemia as shown by the presence of hemoglobin S.
Bristol-Myers Squibb Pharmaceutical Research Institute
Treatment of B-cell non-Hodgkin's lymphoma.
Spectrum Pharmaceuticals, Inc.
Treatment of chronic lymphocytic leukemia (CLL)
Treatment of Waldenstrom's macroglobulinemia
Treatment of chronic lymphocytic leukemia (CLL)
Treatment of small lymphocytic lymphoma
Treatment of mantle cell lymphoma
Treatment of chronic lymphocytic leukemia (CLL)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 364 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3105Ibuprofen lysine Neoprofen 10/29/1996 Lundbeck, Inc.
3106icatibant Firazyr 11/25/2003 Treatment of angioedema
3107
7/18/19973108
Idarubicin HCl for injection Idamycin 7/25/19883109
idarucizumab n/a 5/28/20153110
idelalisib Zydelig 9/26/2013 Gilead Sciences, Inc.3111
idelalisib Zydelig 10/15/2013 Gilead Sciences, Inc.
Treatment of patent ductus arteriosus
Shire Orphan Therapies
Icodextrin 7.5% with Electrolytes Peritoneal Dialysis Solution
Extraneal (With 7.5% Icodextrin) Peritoneal Dialysis Solutio
Treatment of those patients having end stage renal disease and requiring peritoneal dialysis treatment.
Baxter Healthcare Corporation
Treatment of acute myelogenous leukemia, also referred to as acute nonlymphocytic leukemia.
Adria Laboratories, Inc.
To reverse the anticoagulant effect of dabigatran due to uncontrolled life-threatening bleeding requiring urgent intervention or a need to undergo an emergency surgery/urgent invasive procedure
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of follicular lymphoma
Treatment of chronic lymphocytic leukemia and small lymphocytic lymphoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 365 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3112
idursulfase Elaprase 11/28/20013113
Ifosfamide Ifex 1/20/1987 Treatment of testicular cancer.3114
Iloprost inhalation solution Ventavis 8/17/2004 CoTherix, Inc.3115
imatinib Gleevec 10/11/20053116
Imatinib Gleevec 1/31/20013117
imatinib Gleevec 10/11/20053118
imatinib mesylate Gleevec 11/1/20013119
imatinib mesylate Gleevec 11/1/20013120
imatinib mesylate Gleevec 12/19/2005
Long term enzyme replacement therapy for patients with mucopolysaccharidosis II (Hunter Syndrome)
Shire Human Genetic Therapies, Inc.
Bristol-Myers Squibb Pharmaceutical Research Institute
Treatment of pulmonary arterial hypertension
Treatment of Philadelphia-positive acute lymphoblastic leukemia
Novartis Pharmaceuticals Corporation
Treatment of chronic myelogenous leukemia
Novartis Pharmaceuticals Corporation
Treatment of Philadelphia-positive acute lymphoblastic leukemia
Novartis Pharmaceuticals Corporation
Treatment of gastrointestinal stromal tumors
Novartis Pharmaceuticals Corp.
Treatment of gastrointestinal stromal tumors
Novartis Pharmaceuticals Corp.
Treatment of dermatofibrosarcoma protuberans
Novartis Pharmaceuticals Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 366 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3121
Imatinib mesylate Gleevec 10/5/20053122
Imatinib mesylate Gleevec 9/9/20053123
Imatinib mesylate Gleevec 8/25/20053124
Imiglucerase Cerezyme 11/5/1991 Genzyme Corporation3125
Immune Globulin (Human) Gamunex(R)-C 7/27/20043126
Gammagard Liquid 7/20/2006 Baxalta US, Inc.3127
Gamimune N 2/18/1993 Bayer Corporation3128
Gammaplex 4/29/2011
Treatment of myeloproliferative disorders/myelodysplastic syndromes associated with platelet-derived growth factor gene re-arrangements
Novartis Pharmaceuticals Corporation
Treatment of systemic mastocytosis without the D816V c-kit mutation
Novartis Pharmaceuticals Corporation
Treatment of idiopathic hypereosinophilic syndrome including acute and chronic eosinophilic leukemia
Novartis Pharmaceuticals Corporation
Replacement therapy in patients with types I, II, and III Gaucher's disease.
Treatment of chronic inflammatory demyelinating polyneuropathy
Grifols Therapeutics, Inc.
immune globulin infusion (human)
Treatment of multifocal motor neuropathy
Immune globulin intravenous, human
Infection prophylaxis in pediatric patients affected with the human immunodeficiency virus.
immune globulin intraveous (human)
Treatment of idiopathic thrombocytopenic purpura (also known as primary immune thrombocytopenia)
Bio Products Laboratory
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 367 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3129
Infliximab Remicade 11/12/2003 Centocor, Inc.3130 infliximab Remicade 11/14/1995 Treatment of Crohn's disease Centocor, Inc.3131
infliximab Remicade 11/12/2003 Janssen Biotech Inc.3132
Interferon alfa-2a Roferon A 6/6/19893133
Roferon-A 12/14/19873134
Intron A 6/24/1987 Schering Corporation3135
Interferon beta-1a Avonex 12/16/1991 Treatment of multiple sclerosis. Biogen, Inc.3136
Interferon beta-1b Betaseron 11/17/1988 Treatment of multiple sclerosis.3137
Interferon gamma 1-b Actimmune 9/30/19883138
Interferon gamma-1b Actimmune 9/30/19963139
intravenous carbamazepine Carnexiv 6/27/2013 Lundbeck LLC3140
Iobenguane I 123 Adreview 12/1/2006 GE Healthcare, Inc.3141
Iobenguane I 123 Adreview 12/1/2006 GE Healthcare, Inc.
Treatment of pediatric (0 to 16 years of age) Crohn's Disease
Treatment of pediatric (0 to 16 years of age) ulcerative colitis
Treatment of chronic myelogenous leukemia.
Hoffmann-La Roche, Inc.
Interferon alfa-2a (recombinant)
Treatment of AIDS related Kaposi's sarcoma.
Hoffmann-La Roche, Inc.
Interferon alfa-2b (recombinant)
Treatment of AIDS-related Kaposi's sarcoma.
Chiron Corp. & Berlex Laboratories
Treatment of chronic granulomatous disease.
Horizon Pharma Ireland Limited
Delaying time to disease progression in patients with severe, malignant osteopetrosis.
Horizon Pharma Ireland Limited
Treatment of epilepsy patients who cannot take anything by mouth (NPO)
For the diagnosis of neuroblastomas
For the diagnosis of pheochromocytomas
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 368 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3142
ipilimumab Yervoy 6/3/20043143
ipilimumab Yervoy 6/3/20043144
iron(III)-hexacyanoferrate(II) Radiogardase 5/1/20033145
isavuconazonium sulfate Cresemba 5/6/20133146
isavuconazonium sulfate Cresemba 10/25/2013 Treatment of zygomycosis3147
ivacaftor Kalydeco 12/20/20063148
ixazomib citrate Ninlaro 2/18/2011 Treatment of multiple myeloma3149
Ixiaro 9/25/2012 Intercell AG3150
Lamotrigine Lamictal 8/23/19953151 Lanreotide Somatuline Depot 9/11/2000 Treatment for acromegly IPSEN, Inc.
Treatment of high risk Stage II, Stage III, and Stage IV melanoma
Bristol-Myers Squibb Company
Treatment of high risk Stage II, Stage III, and Stage IV melanoma
Bristol-Myers Squibb Company
Treatment of patients with known or suspected internal contamination with radioactive or non-radioactive cesium or thallium
Heyl Chemisch-Pharmzeutische Fabrik GMBH & Co, KG
Treatment of invasive aspergillosis
Astellas Pharma Global Development Inc.
Astellas Pharma Global Development Inc.
Treatment of patients with cystic fibrosis
Vertex Pharmaceuticals, Inc.
Millennium Pharmaceuticals
Japanese encephalitis vaccine, inactivated, adsorbed
Prevention of Japanese encephalitis virus in pediatric patients.
Treatment of Lennox-Gastaut syndrome.
Glaxo Wellcome Research and Development
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 369 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3152
lanreotide acetate Somatuline Depot 8/25/20113153
laronidase Aldurazyme 9/24/19973154
lenalidomide Revlimid 4/27/2009 Celgene Corporation3155
lenalidomide Revlimid 1/29/2004 Celgene Corporation3156
lenalidomide Revlimid 9/20/2001 Treatment of multiple myeloma Celgene Corporation3157
lenalidomide Revlimid 9/20/2001 Treatment of multiple myeloma Celgene Corporation3158
lenvatinib Lenvima 12/27/2012 Eisai, Inc.3159
Lepirudin Refluden 2/13/19973160
Leucovorin Leucovorin Calcium 12/8/1986 Immunex Corporation3161
Leucovorin Leucovorin Calcium 8/17/1988 Immunex Corporation
Treatment of neuroendocrine tumors
Ipsen Biopharmaceuticals, Inc.
Treatment of patients with mucopolysaccharidosis-I.
BioMarin Pharmaceutical, Inc.
Treatment of mantle cell lymphoma
Treatment of myelodysplastic syndromes
Treatment of follicullar, medullary, anaplastic, and metastatic or locally advanced papillary thyroid cancer
Treatment of heparin-associated thrombocytopenia type II.
Hoechst Marion Roussel
For use in combination with 5-fluorouracil for the treatment of metastatic colorectal cancer.
For rescue use after high dose methotrexate therapy in the treatment of osteosarcoma.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 370 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3162
Leuprolide acetate Lupron Injection 7/25/19883163
Levocarnitine Carnitor 7/26/19843164
Levocarnitine Carnitor 9/6/19883165
Levocarnitine Carnitor 2/28/19843166
Levodopa and carbidopa Duodopa 1/18/2000 AbbVie, Inc.3167
Levoleucovorin Fusilev 8/1/19913168
levoleucovorin Fusilev 12/18/19903169
Orlaam 1/24/1985
Treatment of central precocious puberty
Tap Pharmaceuticals, Inc.
Treatment of primary and secondary carnitine deficiency of genetic origin.
Sigma-Tau Pharmaceuticals, Inc.
Treatment of manifestations of carnitine deficiency in patients with end stage renal disease who require dialysis.
Sigma-Tau Pharmaceuticals, Inc.
Treatment of genetic carnitine deficiency.
Sigma-Tau Pharmaceuticals, Inc.
Treatment of late stage Parkinson's disease
For use in conjunction with high-dose methotrexate in the treatment of osteosarcoma.
Spectrum Pharmaceuticals, Inc.
For use in combination chemotherapy with the approved agent 5-fluorouracil in the palliative treatment of metastatic adenocarcinoma of the colon and rectum
Spectrum Pharmaceuticals, Inc.
Levomethadyl acetate hydrochloride
Treatment of heroin addicts suitable for maintenance on opiate agonists.
Biodevelopment Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 371 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3170
Lidocaine patch 5% Lidoderm Patch 10/24/19953171
Liothyronine sodium injection Triostat 7/30/19903172
Liposomal amphotericin B Ambisome 12/6/1996 Fujisawa USA, Inc.3173
Liposomal amphotericin B Ambisome 12/10/1996 Fujisawa USA, Inc.3174
liposomal irinotecan n/a 7/21/2011 Treatment of pancreatic cancer3175
Lodoxamide tromethamine 10/16/19913176
lomitapide Juxtapid 10/23/20073177
lumacaftor/ivacaftor Orkambi 6/30/2014 Treatment of cystic fibrosis3178
lumacaftor/ivacaftor Orkambi 6/30/2014 Treatment of cystic fibrosis3179
macitentan Opsumit 9/3/2009
For relief of allodynia (painful hypersensitivity), and chronic pain in postherpetic neuralgia.
Teikoku Pharma USA, Inc.
Treatment of myxedema coma/precoma.
SmithKline Beecham Pharmaceuticals
Treatment of visceral leishmaniasis.
Treatment of cryptococcal meningitis.
Merrimack Pharmaceuticals, Inc.
Alomide Ophthalmic Solution
Treatment of vernal keratoconjunctivitis.
Alcon Laboratories, Inc.
Treatment of homozygous familial hypercholesterolemia
Aegerion Pharmaceuticals, Inc.
Vertex Pharmaceuticals Inc.
Vertex Pharmaceuticals Inc.
Treatment of pulmonary arterial hypertension
Actelion Pharmaceuticals Ltd
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 372 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3180
Mafenide acetate solution Sulfamylon Solution 7/18/19903181
mebendazole 9/3/20143182
Mecasermin Increlex 12/12/19953183
mecasermin rinfabate Iplex 5/17/2002 Insmed, Inc.3184
meclorethamine Valchlor 8/17/2004 Treatment of mycosis fungoides
For use as an adjunctive topical antimicrobial agent to control bacterial infection when used under moist dressings over meshed autografts on excised burn wounds.
Mylan Laboratories, Inc.
Vermox 500 Mg Chewable Tablets
Treatment of single or mixed gastrointestinal infestations by Trichuris trichiura (whipworm), Ascaris lumbricoides (large roundworm), and Ancylostoma duodenale and Nectar americanus (hookworm).
Janssen Pharmaceutical Research & Development, LLC
Treatment of growth hormone insensitivity syndrome.
Ipsen Biopharmaceuticals, Inc.
Treatment of growth hormone insensitivity syndrome (GHIS)
Actelion Pharmaceuticals Ltd.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 373 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3185
mefloquine HCL Lariam 4/13/19883186
mefloquine HCL Lariam 4/13/19883187
Megestrol acetate Megace 4/13/19883188
meloxicam Mobic 11/22/20023189
Melphalan Alkeran For Injection 2/24/1992 Glaxo Wellcome Inc.
For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs
Hoffmann-La Roche, Inc.
For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs
Hoffmann-La Roche, Inc.
Treatment of patients with anorexia, cachexia, or significant weight loss (=/>10% of baseline body weight) and confirmed diagnosis of AIDS.
Bristol-Myers Squibb Pharmaceutical Research Institute
Treatment of juvenile rheumatoid arthritis
Boehringer Ingelheim Pharmaceuticals, Inc.
Treatment of patients with multiple myeloma for whom oral therapy is inappropriate.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 374 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3190
melphalan Evomela 11/24/20083191
mercaptopurine oral solution Purixan 8/20/20123192
Mesna Mesnex 11/14/1985 Degussa Corporation3193
Methotrexate sodium Methotrexate 10/21/1985 Lederle Laboratories3194
methylene blue0.5% Provayblue 12/18/2012 Provepharm SAS3195
metreleptin Myalept 8/22/20013196
Metronidazole (topical) Metrogel 10/22/1987 Treatment of acne rosacea.3197
Midodrine HCl Amatine 6/21/1985 Shire3198
mifepristone Korlym 7/5/20073199
miglustat Zavesca 5/29/1998 Treatment of Gaucher disease.
High dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation
Spectrum Pharmaceuticals, INc.
Treatment of acute lymphoblastic leukemia in pediatric patients
Nova Laboratories Limited
For use as a prophylactic agent in reducing the incidence of ifosfamide-induced hemorrhagic cystitis.
Treatment of osteogenic sarcoma.
Treatment of hereditary and acquired methemoglobinemia
Treatment of metabolic disorders secondary to lipodystrophy
Aegerion Pharmaceuticals, Inc.
Galderma Laboratories, Inc.
Treatment of patients with symptomatic orthostatic hypotension.
Treatment of the clinical manifestations of endogenous Cushing's syndrone
Corcept Therapeutics, Inc.
Actelion Pharmaceuticals Ltd
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 375 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3200miltefosine Impavido 10/10/2006 Treatment of leishmaniasis.
3201
mipomersen Kynamro 5/23/20063202
mitomycin-C Mitosol 1/8/20083203
Mitoxantrone Novantrone 8/13/1999 Serono, Inc.3204
Mitoxantrone Novantrone 8/13/1999 Serono, Inc.3205
Mitoxantrone Novantrone 8/21/1996 Serono3206
Mitoxantrone HCl Novantrone 7/13/1987 Lederle Laboratories3207
Modafinil Provigil 3/15/1993 Cephalon, Inc.3208
Monooctanoin Moctanin 5/30/19843209
Infumorph 7/12/1990 Elkins-Sinn, Inc.
Knight Therapeutics (USA)
Treatment of homozygous familial hypercholesterolemia
Kastle Therapeutics, LLC
Treatment of refractory glaucoma as an adjunct to ab externo glaucoma surgery.
Mobius Therapeutics, LLC
Treatment of secondary-progressive multiple sclerosis.
Treatment of progressive-relapsing multiple sclerosis.
Treatment of hormone refractory prostate cancer.
Treatment of acute myelogenous leukemia, also referred to as acute nonlymphocytic leukemia.
Treatment of excessive daytime sleepiness in narcolepsy.
For dissolution of cholesterol gallstones retained in the common bile duct.
Ethitek Pharmaceuticals, Inc.
Morphine sulfate concentrate (preservative free)
For use in microinfusion devices for intraspinal administration in the treatment of intractable chronic pain.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 376 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3210
M.V.I.-12 3/8/20043211
Naglazyme 2/17/19993212
Nafarelin acetate Synarel Nasal Solution 7/20/1988 Syntex (USA), Inc.3213
Naltrexone HCl Trexan 3/11/19853214
necitumumab n/a 11/20/2015 Eli Lilly and Company3215
nelarabine Arranon 8/10/20043216
nilotinib Tasigna 4/27/2006
multi-vitamin infusion without vitamin K
Prevention of vitamin deficiency and thromboembolic complications in people receiving home parenteral nutrition and warfarin-type anticoagulant therapy
Mayne Pharma (USA) Inc.
N-acetylgalactosamine-4-sulfatase, recombinant human
Treatment of mucopolysaccharidosis Type VI (Maroteaux-Lamy syndrome).
BioMarin Pharmaceutical, Inc.
Treatment of central precocious puberty.
For blockade of the pharmacological effects of exogenously administered opioids as an adjunct to the maintenance of the opioid-free state in detoxified formerly opioid-dependent individuals.
DuPont Pharmaceuticals
Treatment of squamous non-small cell lung cancer
Treatment of acute lymphoblastic leukemia and lymphoblastic lymphoma
Novartis Pharmaceuticals Corp.
Treatment of chronic myelogenous leukemia
Novartis Pharmaceutical Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 377 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3217
nimodipine Nymalize 9/16/20113218
nintedanib Ofev 6/29/20113219
nitazoxanide Alinia 2/14/2002 Treatment of intestinal giardiasis3220
Nitazoxanide Alinia 12/12/1996 Treatment of cryptosporidiosis.3221
Nitisinone Orfadin 5/16/1995 Treatment of tyrosinemia type 1.3222
Nitisinone Orfadin 5/16/1995 Treatment of tyrosinemia type 1.3223
Nitric oxide Inomax 6/22/1993 INO Therapeutics, Inc.3224
nivolumab n/a 1/23/20133225
nivolumab Opdivo 8/7/2014 Treatment of Hodgkin lymphoma3226
obeticholic acid Ocaliva 4/9/20083227
obiltoxaximab Anthim 6/9/20063228
obinutuzumab Gazyva 4/15/2015 Genentech, Inc.3229
obinutuzumab Gazyva 2/17/2012 Genentech, Inc.
Treatment of subarachnoid hemorrhage.
Arbor Pharmaceuticals, Inc.
Treatment of patients with idiopathic pulmonary fibrosis.
Boehringer Ingelheim Pharmaceuticals, Inc.
Romark Laboratories, L.C.
Romark Laboratories, L.C.
Swedish Orphan Biovitrum AB
Swedish Orphan Biovitrum AB
Treatment of persistent pulmonary hypertension in the newborn.
Treatment of Stage IIb to IV melanoma
Bristol-Myers Squibb Co.
Bristol-Myers Squibb Co.
Treatment of primary biliary cirrhosis
Intercept Pharmaceuticals, Inc.
Treatment of exposure to B. anthracis spores
Elusys Therapeutics, Inc.
Treatment of follicular lymphoma
Treatment of chronic lymphocytic leukemia
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 378 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3230
Octreotide Sandostatin Lar 8/24/19983231
Octreotide Sandostatin Lar 8/24/19983232
Octreotide Sandostatin Lar 8/24/1998 Treatment of acromegaly.3233
ofatumumab Arzerra 3/10/20093234
ofatumumab Arzerra 3/10/20093235
ofatumumab Arzerra 3/10/20093236
ofatumumab Arzerra 3/10/20093237
Ofloxacin 4/18/1991 Allergan, Inc.3238
olaparib Lynparza 10/16/2013 Treatment of ovarian cancer3239
olaratumab Lartruvo 10/9/2014 Treatment of soft tissue sarcoma Eli Lilly and Company3240
omacetaxine mepesuccinate Synribo 3/10/2006
Treatment of severe diarrhea and flushing associated with malignant carcinoid tumors.
Novartis Pharmaceuticals Corporation
Treatment of diarrhea associated with vasoactive intestinal peptide tumors (VIPoma).
Novartis Pharmaceuticals Corporation
Novartis Pharmaceuticals Corporation
Treatment of chronic lymphocytic leukemia
Novartis Pharmaceuticals Corporation
Treatment of chronic lymphocytic leukemia
Novartis Pharmaceuticals Corporation
Treatment of chronic lymphocytic leukemia
Novartis Pharmaceuticals Corporation
Treatment of chronic lymphocytic leukemia
Novartis Pharmaceuticals Corporation
Ocuflox Ophthalmic Solution
Treatment of bacterial corneal ulcers.
AstraZeneca Pharmaceuticals LP
Treatment of chronic myelogenous leukemia
IVAX International GmbH
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 379 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3241
Oprelvekin Neumega 12/17/19963242
osimertinib Tagrisso 9/4/20143243
oxybate Xyrem 11/7/1994 Treatment of narcolepsy. Jazz Pharmaceuticals3244
oxybate Xyrem 11/7/1994 Treatment of narcolepsy. Jazz Pharmaceuticals3245
Paclitaxel Taxol 3/25/19973246
Abraxane 9/3/2009 Treatment of pancreatic cancer.3247
panobinostat Farydak 8/20/2012 Treatment of multiple myeloma3248
parathyroid hormone Natpara 8/31/20073249
paricalcitol Zemplar 10/27/2015 Abbvie, inc.3250
pasireotide n/a 8/25/2009 Treatment of acromegaly3251
pasireotide Signifor 7/24/2009 Treatment of Cushing's disease
Prevention of severe chemotherapy-induced thrombocytopenia.
Genetics Institute, Inc.
Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer
AstraZeneca Pharmaceuticals LP
Treatment of AIDS-related Kaposi's sarcoma.
Bristol-Myers Squibb Pharmaceutical Research Institute
paclitaxel protein-bound particles
Abraxis BioScience, LLC
Novartis Pharmaceuticals Corporation
Treatment of hypoparathyroidism
Shire-NPS Pharmaceuticals, Inc.
Treatment of pediatric hyperparathyroidism
Novartis Pharmaceuticals Corporation
Novartis Pharmaceuticals Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 380 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3252
pazopanib Votrient 10/20/20093253
Pegademase bovine Adagen 5/29/19843254
Pegaspargase Oncaspar 10/20/1989 Baxalta US, Inc.3255
Pegfilgrastim Neulasta 11/20/2013 Amgen, Inc.3256
peginterferon alfa-2b Sylatron 4/9/20083257
pegloticase Krystexxa 2/21/20013258
Pegvisomant Somavert 6/24/1997 Treatment of acromegaly. Sensus Corporation3259
pembrolizumab Keytruda 11/19/2012
Treatment of soft tissue sarcomas
Novartis Pharmaceuticals Corp
For enzyme replacement therapy for ADA deficiency in patients with severe combined immunodeficiency.
Sigma-tau Pharmaceuticals, Inc.
Treatment of acute lymphocytic leukemia.
Treatment of subjects at risk of developing myelosuppression after a radiological or nuclear incident
Treatment of malignant melanoma stages IIb through IV.
Schering-Plough Corporation
To control the clinical consequences of hyperuricemia in patients with severe gout in whom conventional therapy is contraindicated or has been ineffective.
Horizon Pharma Rheumatology, LLC
Treatment of Stage IIB through IV malignant melanoma
Merck, Sharp & Dohme Corp.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 381 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3260
pembrolizumab Keytruda 11/19/20123261
pemetrexed disodium Alimta 8/28/2001 Eli Lilly and Company3262
Pentamidine isethionate Nebupent 1/12/1988 Fujisawa USA, Inc.3263
Pentamidine isethionate Pentam 300 2/28/1984 Fujisawa USA, Inc.3264
Pentastarch Pentaspan 8/28/19853265
Pentosan polysulfate sodium Elmiron 8/7/1985 Treatment of interstitial cystitis. Alza Corporation3266
Pentostatin for injection Nipent 9/10/1987 Treatment of hairy cell leukemia. SuperGen, Inc.3267
phoxilium n/a 2/14/20143268
Pilocarpine Salagen 9/24/1990 MGI Pharma, Inc.3269
Pilocarpine HCl Salagen 2/28/1992 MGI Pharma, Inc.
Treatment of Stage IIB through IV malignant melanoma
Merck, Sharp & Dohme Corp.
Treatment of malignant pleural mesothelioma
Prevention of Pneumocystis carinii pneumonia in patients at high risk of developing this disease.
Treatment of Pneumocystis carinii pneumonia.
As an adjunct in leukapheresis to improve the harvesting and increase the yield of leukocytes by centrifugal means.
DuPont Pharmaceuticals
For use as a replacement solution in patients undergoing continuous renal replacement therapy
Gambro Renal Products, Inc.
Treatment of xerostomia induced by radiation therapy for head and neck cancer.
Treatment of xerostomia and keratoconjunctivitis sicca in Sjogren's syndrome patients.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 382 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3270pirfenidone Esbriet 3/5/2004 Genentech
3271
plerixafor Mozobil (R) 7/10/2003 Genzyme Corporation3272
Gliadel 12/13/1989 Treatment of malignant glioma.3273
Gliadel 12/13/1989 Treatment of malignant glioma.3274
pomalidomide Pomalyst 1/15/2003 Treatment of multiple myeloma Celgene Corporation3275
ponatinib Iclusig 11/20/20093276
ponatinib Iclusig 11/20/20093277
porfimer Photofrin 10/19/2001
Treatment of idiopathic pulmonary fibrosis
For use to improve the yield of progentor cells in the apheresis product for subsequest stem cell transplantation following myelosuppressive or myeloablative chemotherapy
polifeprosan 20 with carmustine
Guilford Pharmaceuticals, Inc.
polifeprosan 20 with carmustine
Guilford Pharmaceuticals, Inc.
Treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL)
ARIAD Pharmaceuticals Inc.
Treatment of chronic myeloid leukemia
ARIAD Pharmaceuticals Inc.
For the ablation of High-Grade Dysplasia in Barrett's Esophagus in patients who are not considered to be candidates for esophagectomy
Axcan Scandipharm Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 383 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3278
Porfimer sodium Photofrin 6/6/19893279
Potassium citrate Urocit-K 11/1/19843280
Potassium citrate Urocit-K 11/1/19843281
Potassium Iodide Oral Solution Thyroshield 11/17/20043282
pralatrexate Folotyn 7/20/2006 Treatment of T-cell lymphoma3283
propranolol Hemangeol 9/5/20083284
Protein C concentrate Ceprotin 6/23/1992 Baxalta US, Inc.
For the photodynamic therapy of patients with primary or recurrent obstructing (either partially or completely) esophageal carcinoma.
QLT Phototherapeutics, Inc.
Prevention of uric acid nephrolithiasis.
University of Texas Health Science Center at Dallas
1. Prevention of calcium renal stones in patients with hypocitraturia 2. For avoidance of the complication of calcium stone formation in patients with uric lithiasis.
University of Texas Health Science Center at Dallas
For use as a thyroid blocking agent in pediatric patients exposed to radiactive iodine
Fleming & Company, Pharmaceuticals
Allos Therapeutics, Inc.
Treatment of proliferating infantile hemangiomas requiring systemic therapy
Pierre Fabre Dermatologie
For replacement therapy in congenital protein C deficiency for the prevention and treatment of thrombosis, pulmonary emboli, and purpura fulminans.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 384 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3285
Kcentra 12/27/2012 CSL Behring3286
Kcentra 12/27/2012 CSL Behring3287
Curosurf 8/2/1993 Chiesi USA, Inc.3288
quinine Sulfate n/a 6/3/2004 Treatment of malaria3289
raloxifene Evista 7/14/2005 Eli Lilly and Company3290
ramucirumab Cyramza 2/16/2012 Treatment of gastric cancer Eli Lilly and Company3291
ramucirumab Cyramza 2/16/2012 Treatment of gastric cancer Eli Lilly and Company3292
rasburicase Elitek 10/11/20003293
raxibacumab Abthraxtm 11/12/2003 Treatment of anthrax
prothrombin complex concentrate (human)
Treatment of patients needing urgent reversal of Vitamin K antagonist therapy for treatment of major bleeding and/or surgical procedures
prothrombin complex concentrate (human)
Treatment of patients needing urgent reversal of Vitamin K antagonist therapy for treatment of major bleeding and/or surgical procedures
Pulmonary surfactant replacement, porcine
For the treatment and prevention of respiratory distress syndrome in premature infants.
AR Holding Company, Inc.
Reduction of the risk of breast cancer in postmenopausal women
Treatment of malignancy-associated or chemotherapy-induced hyperuricemia.
Sanofi-Synthelabo Research
Human Genome Sciences, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 385 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3294
Idelvion 4/27/2012 CSL Behring, LLC3295
1. Myozyme 2. Lumizyme 8/19/1997 Genzyme Corporation3296
1. Myozyme 2. Lumizyme 8/19/1997 Genzyme Corporation3297
1. Myozyme 2. Lumizyme 8/19/1997 Genzyme Corporation3298
Atryn 12/7/20073299
Luveris 10/7/1994 EMD Serono, Inc.3300
n/a 11/23/2010 Baxalta US, Inc.
recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP)
Treatment of patients with congenital factor IX deficiency (hemophilia B).
Recombinant human acid alpha-glucosidase; alglucosidase alfa
Treatment of glycogen storage disease type II.
Recombinant human acid alpha-glucosidase; alglucosidase alfa
Treatment of glycogen storage disease type II.
Recombinant human acid alpha-glucosidase; alglucosidase alfa
Treatment of glycogen storage disease type II.
recombinant human antithrombin
Treatment of congenital antithrombin deficiency to prevent the occurrence of serious, potentially life-threatening venous thromboembolisms which may develop as a result of surgical or obstetrical procedures
GTC Biotherapeutics, Inc.
Recombinant human luteinizing hormone
For use in association with recombinant human follicle stimulating hormone for the treatment of women with chronic anovulation due to hypogonadotropic hypogonadism.
recombinant von Willebrand factor (rhVWF)
Treatment of von Willebrand disease.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 386 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3301
regorafenib Stivarga 1/12/20113302
H.P. Acthar Gel 5/21/2003 Treatment of infantile spasms3303
Respigam 9/27/19903304
Winrho Sd 11/9/19933305
ribavirin Rebetol 4/4/2003 Schering Corporation3306
Photrexa Viscous 9/2/2011 Treatment of keratoconus Avedro, Inc.3307
Photrexa Viscous 12/2/2011 Avedro, Inc.3308
Rifabutin Mycobutin 12/18/19893309
Rifampin Rifadin I.V. 12/9/1985
Treatment gastrointestinal stromal tumors
Bayer HealthCare Pharmaceuticals, Inc.
repository corticotropin or adrenocorticotropic hormone
Questcor Pharmaceuticals, Inc.
Respiratory syncytial virus immune globulin (Human)
Prophylaxis of respiratory syncytial virus lower respiratory tract infections in infants and young children at high risk of RSV disease.
MedImmune & Massachussetts Public Health Biologics Labs.
Rho (D) immune globulin intravenous (human)
Treatment of immune thrombocytopenic purpura.
Rh Pharmaceuticals, Inc.
Treatment of chronic hepatitis C in pediatric patients
riboflavin ophthalmic solution & ultraviolet A
riboflavin ophthalmic solution ultraviolet-A (UVA) irradiation
Treatment of corneal ectasia following refractive surgery
Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.
Adria Laboratories, Inc.
For antituberculosis treatment where use of the oral form of the drug is not feasible.
Hoechst Marion Roussel
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 387 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3310Rifater 9/12/1985
3311Rifapentine Priftin 6/9/1995
3312
rifaximin Normix 2/10/19983313
Rilonacept Arcalyst 12/20/20043314
Riluzole Rilutek 3/16/19933315
riociguat Adempas 9/19/20133316
riociguat Adempas 9/19/20133317
rituximab Rituxan 1/29/2004 Genentech, Inc.3318
rituximab Rituxan 6/13/1994 Genentech, Inc.3319
rituximab Rituxan 2/14/2006 Genentech, Inc.
Rifampin, isoniazid, pyrazinamide
For the short-course treatment of tuberculosis.
Hoechst Marion Roussel
Treatment of pulmonary tuberculosis.
Hoechst Marion Roussel
Treatment of hepatic encephalopathy
Salix Pharmaceuticals, Inc.
Treatment of CIAS1-Associated Periodic Syndromes
Regeneron Pharmaceuticals, Inc.
Treatment of amyotrophic lateral sclerosis.
Rhone-Poulenc Rorer Pharmaceuticals, Inc.
Treatment of chronic thromboembolic pulmonary hypertension
Bayer HealthCare Pharmaceuticals, Inc.
Treatment of pulmonary arterial hypertension.
Bayer HealthCare Pharmaceuticals, Inc.
Treatment of chronic lymphocytic leukemia
Treatment of non-Hodgkin's B-cell lymphoma
Treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (Wegener's Granulomatosis, Microscopic Polyangiitis, and Churg-Strauss Syndrome)
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 388 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3320romidepsin Istodax 9/30/2004 Celgene Corporation
3321romidepsin Istodax 9/30/2004 Celgene Corporation
3322romiplostim Nplate 3/27/2003 Amgen, Inc.
3323
rosuvastatin Crestor 2/14/20143324
rufinamide Banzel 10/8/2004 Eisai, Inc.3325
ruxolitinib Jakafi 3/26/2010 Treatment of polycythemia vera Incyte Corporation3326 ruxolitinib phosphate Jakafi 9/5/2008 Treatment of myelofibrosis Incyte Corporation3327
Sacrosidase Sucraid 12/10/1993 QOL Medical, LLC3328
sapropterin Kuvan 1/29/20043329
Sargramostim Leukine 3/6/1995 Immunex Corporation
Treatment of non-Hodgkin T-cell lymphomas
Treatment of non-Hodgkin T-cell lymphomas
Treatment of immune thrombocytopenic purpura
For the treatment of pediatric homozygous familial hypercholesterolemia
iPR Pharmaceuticals, Inc.
Treatment of Lennox-Gastaut Syndrome.
Treatment of congenital sucrase-isomaltase deficiency
Treatment of hyperphenylalaninemia
BioMarin Pharmaceutical, Inc.
To reduce neutropenia and leukopenia and decrease the incidence of death due to infection in patients with acute myelogenous leukemia.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 389 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3330
Sargramostim Leukine 5/3/1990 Immunex Corporation3331
Satumomab pendetide Oncoscint Cr/Ov 9/25/1989 Detection of ovarian carcinoma. Cytogen Corporation3332
sebelipase alfa Kanuma 7/1/20103333
Selegiline HCl Eldepryl 11/7/19843334
selexipag Uptravi 4/30/2010 Actelion Ltd3335
Sermorelin acetate Geref 9/14/1988 EMD Serono, Inc.3336
siltuximab Sylvant 5/26/2006 Janssen Biotech, Inc.3337
sirolimus Rapamune 10/31/2012 Pfizer, Inc.3338
Nithiodote 4/9/2008
Treatment of neutropenia associated with bone marrow transplant, for the treatment of graft failure and delay of engraftment, and for the promotion of early engraftment.
Treatment of lysosomal acid lipase deficiency
Alexion Pharmaceuticals
As an adjuvant to levodopa and carbidopa treatment of idiopathic Parkinson's disease (paralysis agitans), postencephalitic Parkinsonism, and symptomatic Parkinsonism.
Somerset Pharmaceuticals, Inc.
Treatment of pulmonary arterial hypertension
Treatment of idiopathic or organic growth hormone deficiency in children with growth failure.
Treatment of Castleman's disease
Treatment of lymphangioleiomyomatosis
sodium nitrite and sodium thiosulfate
Treatment of known or suspected cyanide poisoning
Hope Pharmaceuticals
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 390 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3339
sodium phenylbutyrate Buphenyl 11/22/19933340
Somatrem for injection Protropin 12/9/1985 Genentech, Inc.3341
Somatropin Norditropin 8/9/2006 Novo Nordisk Inc.3342
Somatropin Nutropin 3/6/1987 Genentech, Inc.3343
Somatropin Genotropin 9/6/1994 Pharmacia & Upjohn3344
Somatropin Humatrope 5/8/1990 Eli Lilly and Company
Treatment of urea cycle disorders: carbamylphosphate synthetase deficiency, ornithine transcarbamylase deficiency, and arginiosuccinic acid synthetase deficiency.
Medicis Pharmaceutical Corp.
1. Long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion (prevalence 15,000) 2. Treatment of short stature associated with Turner's syndrome (prevalence 8000).
Treatment of short stature in patients with Noonan syndrome
For use in the long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion.
Treatment of adults with growth hormone deficiency.
Treatment of short stature associated with Turner syndrome.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 391 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3345
Somatropin Humatrope 12/15/2005 Eli Lilly and Company3346
Somatropin (r-DNA) Zorbtive 3/6/1995 EMD Serono, Inc.3347
Somatropin (rDNA origin) Saizen 3/6/1987 EMD Serono, Inc.3348
Somatropin (rDNA origin) Nutropin Depot 10/28/1999 Genentech, Inc.3349
Norditropin 7/10/19873350
Somatropin for injection Nutropin 11/18/1996 Genentech, Inc.3351
Somatropin for injection Serostim 11/15/1991 EMD Serono, Inc.
Treatment of short stature in pediatric patients with short stature homeobox-containing gene (SHOX) deficiency
For use alone or in combination with glutamine in the treatment of short bowel syndrome.
Treatment of idiopathic or organic growth hormone deficiency in children with growth failure.
Long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion.
Somatropin (rDNA origin) injection
1. Treatment of growth failure in children due to inadequate growth hormone secretion 2. Treatment of short stature associated with Turner's syndrome
Novo Nordisk Pharmaceuticals
As replacement therapy for growth hormone deficiency in adults after epiphyseal closure.
Treatment of AIDS-associated catabolism/weight loss.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 392 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3352
Somatropin for injection Nutropin 8/4/1989 Genentech, Inc.3353
Somatropin for injection Humatrope 6/12/1986 Eli Lilly and Company3354
Somatropin for injection Nutropin 3/23/1989 Genentech, Inc.3355
Somatropin [rDNA] Genotropin 7/6/1999 Pharmacia & Upjohn3356
somatropin [rDNA] Genotropin 12/27/20003357
Sorafenib Nexavar 10/8/20043358
sorafenib Nexavar 12/12/20113359
Sorafenib Nexavar 4/20/2006
Treatment of growth retardation associated with chronic renal failure.
For the long-term treatment of children who have growth failure due to inadequate secretion of normal endogenous growth hormone.
Treatment of short stature associated with Turner's syndrome.
Treatment of short stature in patients with Prader-Willi syndrome.
Treatment of growth failure in children who were born small for gestational age.
Pharmacia and Upjohn Company
Treatment of renal cell carcinoma.
Bayer Pharmaceutical Corporation
Treatment of medullary thyroid cancer, anaplastic thyroid cancer, and recurrent or metastatic follicular or papillary thyroid cancer
Bayer HealthCare Pharmaceuticals, Inc.
Treatment of hepatocellular carcinoma
Bayer Pharmaceuticals Corporation
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 393 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3360
sotalol (IV) So-Aqueous 7/25/20083361
Sotalol HCl Betapace 9/23/19883362
Sterile talc powder 9/18/1995 Bryan Corporation3363
Succimer Chemet Capsules 5/9/19843364
Sulfadiazine n/a 3/14/19943365
Synthetic human secretin Chirostim 6/16/1999 ChiRhoClin, Inc.3366
Synthetic porcine secretin Secreflo 6/18/1999 ChiRhoClin, Inc.3367
Synthetic porcine secretin Secreflo 6/18/1999 ChiRhoClin, Inc.
For ventricular tachycardia, ventricular fibrillation, or the maintenance of sinus rhythm in patients converted from atrial fibrillation or atrial flutter when oral administration is not possible.
Academic Pharmaceuticals
1. Treatment of life-threatening ventricular tachyarrhythmias 2. Prevention of life treatening ventricular tachyarrhythmias
Berlex Laboratories, Inc.
Sclerosol Intrapleural Aerosol
Treatment of malignant pleural effusion.
Treatment of lead poisoning in children.
Bock Pharmacal Company
For use in combination with pyrimethamine for the treatment of Toxoplasma gondii encephalitis in patients with and without AIDS.
Eon Labs Manufacturing, Inc.
For use in the diagnosis of gastrinoma associated with Zollinger-Ellison syndrome.
For use in the evaluation of exocrine pancreas function.
For use in the diagnosis of gastrinoma associated with Zollinger-Ellison syndrome.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 394 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3368
Synthetic porcine secretin Secreflo 3/7/2000 ChiRhoClin, Inc.3369
tacrolimus Envarsus Xr 12/20/20133370
Tacrolimus Prograf 6/6/20053371
tadalafil Adcirca 12/18/2006 Eli Lilly and Company3372
Taliglucerase alfa Elelyso For Injection 9/3/2009 Treatment of Gaucher's disease Pfizer, Inc.3373
talimogene laherparepvec Imlygic 3/14/20113374
tasimelteon Hetlioz 1/19/20103375
Lymphoseek 9/17/2014
For use in conjunction with diagnostic, therapeutic, or combined diagnostic/therapeutic procedures for pancreatic disorders to increase pancreatic fluid secretion.
Prophylaxis of organ rejection in patients receiving allogeneic kidney transplant
Veloxis Pharmaceuticals, Inc.
Prophylaxis of organ rejection in patients receiving heart transplants.
Astellas Pharma US, Inc.
Treatment of pulmonary arterial hypertension
Treatment of stage IIb-stage IV melanoma
BioVex, Inc. (subsidiary of Amgen)
Non-24-hour sleepwake disorder in blind individuals without light perception
Vanda Pharmaceuticals, Inc.
technetium Tc 99m tilmanocept
Use in sentinel lymph node detection (SLN) with a hand-held gamma-counter, with scintigraphic imaging, in patients with cancer of the head and neck
Navidea Biopharmaceuticals
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 395 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3376
3/17/2009 Pharmalucence, Inc.3377
teduglutide [rDNA origin] Gattex 6/29/20003378
temozolomide Temodar 10/5/19983379
temozolomide Temodar 10/5/19983380
Temsirolimus Torisel 12/16/20043381
Teniposide Vumon For Injection 11/1/19843382
tenofovir Viread 3/17/2009 Gilead Sciences, Inc.3383
Teriparatide Parathar 1/9/19873384
Tetrabenazine Xenazine 12/11/1997
Technetium Tc99m sulfur colloid injection, lyophilized
Technetium Tc99m Sulfur Colloi
For localization of sentinel lymph nodes in patients with melanoma.
Treatment of short bowel syndrome.
NPS Pharmaceuticals, Inc.
Treatment of recurrent malignant glioma.
Schering-Plough Research Institute
Treatment of recurrent malignant glioma.
Schering-Plough Research Institute
Treatment of renal cell carcinoma
Wyeth Pharmaceuticals, Inc.
Treatment of refractory childhood acute lymphocytic leukemia.
Bristol-Myers Squibb Pharmaceutical Research Institute
Treatment of pediatric HIV infection.
Diagnostic agent to assist in establishing the diagnosis in patients presenting with clinical and laboratory evidence of hypocalcemia due to either hypoparathyroidism or pseudohypoparathyroidism.
Rhone-Poulenc Rorer Pharmaceuticals, Inc.
Treatment of Huntington's disease
Prestwick Pharmaceuticals, Inc
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 396 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3385Thalidomide Thalomid 7/26/1995 Celgene Corporation
3386Thalidomide Thalomid 10/14/1998 Treatment of multiple myeloma Celgene Corporation
3387
Thyrotropin alfa Thyrogen 8/3/2001 Genzyme Corporation3388
Thyrotropin alpha Thyrogen 2/24/1992 Genzyme Corporation3389
tinidazole Tindamax 4/18/2002 Treatment of giardiasis3390
Tinidazole Tindamax 8/20/2003 Treatment of amebiasis3391
Tiopronin Thiola 1/17/1986 Pak, Charles Y.C. M.D.3392
Tobramycin for inhalation Tobi 10/13/19943393
tocilizumab Actemra 7/31/2012 Genentech, Inc.3394
Topiramate Topamax 11/25/1992
Treatment of erythema nodosum leprosum.
Treatment of well-differentiated papillary, follicular or combined papillary/follicular carcinomas of the thyroid
As an adjunct in the diagnosis of thyroid cancer.
Presutti Laboratories, Inc.
Presutti Laboratories, Inc.
Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.
Treatment of bronchopulmonary infections of Pseudomonas aeruginosa in cystic fibrosis patients.
Novartis Pharmaceuticals Corp
Treatment of pediatric patients (age 16 years and younger) with polyarticular-course juvenile idiopathic arthritis
Treatment of Lennox-Gastaut syndrome.
Johnson & Johnson Pharmaceutical R & D, LLC
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 397 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3395
Toremifene Fareston 9/19/1991 Orion Corporation3396
Bexxar 5/16/1994 GlaxoSmithKline LLC3397
trabectedin Yondelis 9/30/2004 Treatment of soft tissue sarcoma3398
trametinib Mekinist 12/20/20103399
trametinib and dabrafenib Mekinist And Tafinlar 9/20/20123400
trametinib and dabrafenib Mekinist And Tafinlar 9/20/20123401
trastuzumab Herceptin 10/13/2009 Genentech, Inc.3402
treprostinil Remodulin 6/4/19973403
treprostinil (inhalational) Tyvaso 6/17/2010 LungRx, Inc.3404
Tretinoin Vesanoid 10/24/1990
Hormonal therapy of metastatic carcinoma of the breast.
Tositumomab and iodine I 131 tositumomab
Treatment of non-Hodgkin's B-cell lymphoma.
Janssen Research & Development, LLC
Treatment of Stage IIb through Stage IV melanoma
Novartis Pharmaceuticals Corp.
Treatment of Stage IIb through IV melanoma.
Novartis Pharmaceuticals Corp.
Treatment of Stage IIb through IV melanoma.
Novartis Pharmaceuticals Corp.
Treatment of HER2-overexpressing advanced adenocarcinoma of the stomach, including gastroesophageal junction
Treatment of pulmonary arterial hypertension.
United Therapeutics Corp.
Treatment of pulmonary arterial hypertension
Treatment of acute promyelocytic leukemia.
Hoffmann-La Roche, Inc.
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 398 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3405
Trientine HCl Syprine 12/24/19843406
Trimetrexate glucuronate Neutrexin 5/15/19863407
Trypan blue Membraneblue 8/2/20063408
uridine triacetate n/a 5/1/20093409
uridine triacetate n/a 8/9/20133410
Urofollitropin Metrodin 11/25/1987 EMD Serono, Inc.3411
Ursodiol Urso 250 6/20/19913412
n/a 6/18/20043413
Cnj-016 6/18/2004 Cangene Corporation
Treatment of patients with Wilson's disease who are intolerant, or inadequately responsive to penicillamine.
Merck Sharp & Dohme Research
Treatment of Pneumocystis carinii pneumonia in AIDS patients.
Medimmune Oncology, Inc.
Selectively staining epiretinal membranes during ophthalmic surgical vitrectomy procedures
Dutch Ophthalmic Research Center Int'l BV
An antidote in the treatment of 5-fluorouracil or capecitabine poisoning
Wellstat Therapeutics Corp.
Treatment of hereditary orotic aciduria
Wellstat Therapeutics, Inc.
For induction of ovulation in patients with polycystic ovarian disease who have an elevated LH/FSH ratio and who have failed to respond to adequate clomiphene citrate therapy.
Treatment of patients with primary biliary cirrhosis
Aptalis Pharma US, Inc.
Vaccinia Immune Globulin (Human) Intravenous
Treatment of severe complications from the smallpox vaccine
DynPort Vaccine Company LLC
Vaccinia Immune Globulin (Human) Intravenous
Treatment of complications of vaccinia vaccination
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 399 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3414
Valrubicin Valstar 5/23/19943415
vandetanib Caprelsa(R) 10/21/2005 Genzyme Corporation3416
Varizig 11/7/20063417
velaglucerase-alfa Vpriv 6/8/2009 Treatment of Gaucher disease3418
vemurafenib Zelboraf 12/20/20103419
venetoclax Venclexta 9/20/2012 AbbVie, Inc3420
vigabatrin Sabril 6/12/2000 Treatment of infantile spasms. H. Lundbeck A/S3421
Marqibo 1/8/20073422
vorinostat Zolinza 3/16/2004 Merck & Co., Inc.
Treatment of carcinoma in situ of the urinary bladder.
Anthra Pharmaceuticals, Inc.
Treatment of patients with follicular thyroid carcinoma, medullary thyroid carcinoma, anaplastic thyroid carcinoma, and locally advanced and metastatic papillary thyroid carcinoma
Varicella Zoster Immune Globulin (Human)
Passive immunization for the treatment of exposed, susceptible individuals who are at risk of complications from varicella
Cangene bioPharma, Inc.
Shire Human Genetics Therapies, Inc.
Treatment of patients with IIb to Stage IV melanoma positive for the BRAF(v600) mutation
Hoffmann-La Roche, Inc.
Treatment of chronic lymphocytic leukemia
vinCRIStine sulfate LIPOSOME injection
Treatment of acute lymphoblastic leukemia
Talon Therapeutics, Inc.
Treatment of T-cell non-Hodgkin's lymphoma
Orphan Drug Designations and Approvals List as of 09 01 2016‐ ‐Governs October 1, 2016 Decemberber 31, 2016‐
Page 400 of 400
Generic Name Trade Name Designation Date DesignationRow Num
Contact Company/Sponsor
3423Zalcitabine Hivid 6/28/1988 Treatment of AIDS.
3424zidovudine Retrovir 7/17/1985 Treatment of AIDS Glaxo Wellcome Inc.
3425Zinc acetate Galzin 11/6/1985 Treatment of Wilson's disease. Lemmon Company
3426
Zoledronate Zometa, Zabel 8/18/2000
Hoffmann-La Roche, Inc.
Treatment of tumor induced hypercalcemia.
Novartis Pharmaceuticals Corp.