Those attending the recent Aramco Houston Half Marathon may have noticed the famil-iar Octapharma logo on the orange t-shirt of one runner, Leah Loredo of Wakefield, Kansas. Leah, who was diagnosed with von Willebrand’s Disease (VWD) when she was 8, ran her first half marathon as a fundraising effort for the Midwest Hemophilia Association (MHA), to enable children in need to attend summer camp.

“I was at the National Hemophilia Conference and visited the Octapharma booth and got excited because I finally saw the words von Willebrand Disease,” said Leah, 18, who will be attending Kansas State University in the Fall. “This was the first time I had personally seen anything focused on my disease at the conference. I wanted to wear the Octapharma shirt at my first half marathon because they seemed to be the only pharma-ceutical company that was advocating for von Willebrand Disease education at that meeting.”

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From the Desk of Flemming NielsenPresident of Octapharma USA

July 2014

It is my pleasure to share some of the latest developments at Octapharma USA. Attending many medical congresses globally, including the World Federation of Hemophilia (WFH) Congress in Australia, we have been busy partnering with colleagues in support of patient interests.

WFH represented a great opportunity to gain an improved understanding of practices, treatment regimens, product usage and availability from both U.S. and global perspec-tives. One particular message of note from the congress was that there are significant challenges in attaining and maintaining quality patient care in developing countries.

Patient therapy adherence challenges are universal, but different drivers exist. Barriers in developing countries focus on geography, demographics, financial, and educational issues. Challenges in developed countries, particularly the U.S., focus more on psychosocial barriers.

Working with our colleagues from around the globe at WFH allowed a unique opportunity for us to expand our knowledge base and determine strategies for further successful collaborations. It is clear that successful diagnosis and treatment of von Willebrand Disease (VWD) has made great strides, but so much more work needs to be done to ensure barriers to timely medical care are eliminated. VWD patients need greater recognition in the bleeding disorder community and Octapharma USA is committed to making steady prog-ress in this area.

With that goal in mind, this issue of Inside Octapharma shares the story of one Kansas teenager and her life’s journey with VWD. We also share the latest on Octapharma’s human cell-line recombinant factor VIII (Human-cl rhFVIII), the first purely human recombinant coagulation FVIII from a human cell-line.

Octapharma USA remains focused on helping the medical community ensure that patients receive the life saving and enhanc-ing therapies they need. Our work collaborating with colleagues and partners globally is extremely important to advancing the therapies critical to patient quality of life.

On behalf of Octapharma USA, please allow me to wish you a wonderful Summer and continued success throughout the year.

Octapharma Supports Kansas Teen’s Effortsto Raise Funds for Midwest Hemophilia Camp

Octapharma USA, Inc.• 121 River Street, Suite 1201• Hoboken, NJ 07030 • 201-604-1130 • www.octapharmausa.com

Leah Loredo displays the medals she earned at the Aramco Houston Half Marathon.

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Octapharma USA, Inc.• 121 River Street, Suite 1201• Hoboken, NJ 07030 • 201-604-1130 • www.octapharmausa.com

New Staff Russell Veitenheimer has joined Octapharma USA as Area Business Manager, Central Region, bringing over 25 years of sales experience from a variety of therapeutic areas, including dermatology, rheumatology, endocrinology, urology and neurol-ogy. Russell was with Novartis previously where he gained extensive experience working in a number of sales, sales training and sales management roles, during which time he won several U.S. and international awards. He is based in Fort Worth, Texas.

Katy Calicchia has joined Octapharma USA as Territory Business Manager for East Texas, Louisiana and Arkansas. Katy brings over 25 years of successful sales experience at Bayer Healthcare, where she was able to develop strong relationships in many key hospitals in Eastern and South Texas. Katy earned the President’s Circle Award as well as the District and Area Sales Consul-tant of the Year honors. She is based in Houston, Texas.

Ken Wagg, R.Ph., has joined Octapharma USA as Coagulation Sales Specialist, Mountain States – Arizona, Colorado, Mon-tana, Utah and Wyoming. Ken brings over 25 years of experience from a variety of areas including Pharmacy, Pharmacy Man-agement, Hospital Management as well as Account Management in bleeding disorders. Previously, he was with Accredo where he received consecutive Presidents Club awards. Ken is based in Albuquerque, New Mexico.

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Octapharma Supports Kansas Teen’s Effortsto Raise Funds for Midwest Hemophilia CampOctapharma sponsored Leah in the half marathon by directly providing a charitable donation to the MHA camp fund through local collaboration with our Kansas Territory Business Manager Chad Tebbe. Leah, cheered on by Octapharma staff, stunned family and friends by finishing 26th out of 1201 participants in her age bracket.

“We were amazed at how well Leah did because it was very hard for her to train due to bad weather at home,” said Blanca Loredo, her mother. “What started out as something small has snowballed into an amazing awareness campaign. Our entire community has asked Leah about VWD and she is open to talking about it. Most important of all, Leah has owned VWD. She is also brainstorming ideas for future fundraising projects.”

Leah started participating in Cross Country and Track in her freshman year at Wakefield High School, accomplishments that she would have never dreamed of when she first learned about VWD.

“I remember when I was diagnosed, I was told that I would not be able to participate in all the physical activities that all the other little boys and girls played,” she recalls. “You have to understand I loved playing soccer. I was told I would have to be careful and sometimes wear protective gear in order to play sports or with other children. Living life with caution as I learned is not the end of the world. All my hopes and dreams were not shattered by VWD. I ran four years of cross-country and track during high school. Meeting other people who have VWD has been a blessing because we support one another and that’s why the summer camp is so important to children.”

Leah said she wanted to raise funds to support the MHA summer camp because not all children can afford to attend.

“I have attended camp in the past,” she said. “While in camp I learned more about my disease and was surrounded by other kids with bleeding disorders. Children with bleeding disorders assist staff and volunteers at camp. Doctors, nurses and volun-teers give freely of their time during the summer to allow kids like me to be able to run and play without the fear of being hurt. I wanted to give back to the community and to help support kids to go to camp and give them the same opportunity that I was able to experience.”

David Holliday, Octapharma USA Vice President of Commercial Development, said that Octapharma was pleased to support MHA through Leah’s fundraising efforts.

“Octapharma is committed to building long-term relationships with the VWD community,” said Holliday. “Leah is an excellent role model for young people with bleeding disorders. We welcome opportunities to collaborate in innovative ways that support the community.”

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July 2014

Octapharma USA, Inc.• 121 River Street, Suite 1201• Hoboken, NJ 07030 • 201-604-1130 • www.octapharmausa.com

Octapharma USA has submitted a Biological License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the first purely human recombinant coagulation factor VIII (FVIII) from a human cell-line designed for the treatment of hemophilia A patients.

The FDA submission followed a recent recommendation from the European Union’s central regulatory body, the Committee for Medicinal Products for Human Use (CHMP), medical advisors to European Medicines Agency (EMA), which adopted a positive opinion towards Octapharma’s human cell-line recombinant human FVIII. CHMP recommended marketing authorization for the treatment and prophylaxis of bleeding (also during and after surgery) in both pediatric and adult patients with hemophilia A (congenital FVIII deficiency). Although Octapharma is optimistic about the success of this product in the U.S., there is no connection between the FDA submission and the endorsement of Octapharma’s human cell-line FVIII in Europe.

Recombinant proteins are produced in cultured human cells. Human recombinant proteins produced in human cell-lines show identical properties to the same protein derived directly from human tissues and blood plasma. They are, therefore, assumed to show improved tolerance in patients compared to currently available recombinant products derived from animal cell-lines.

Octapharma’s human cell-line recombinant factor VIII (Human-cl rhFVIII) has been developed with the primary aim of tackling the most serious risk for hemophilia A patients on coagulation FVIII replacement therapy: the development of FVIII inhibitors, or FVIII neutralizing antibodies. FVIII inhibitors may block one or more functional epitopes of coagulation FVIII, preventing the infused FVIII from restoring hemostasis. The development of FVIII inhibitors is considered to be the most serious complication and can be observed in up to 39% of hemophilia A patients treated with currently commercialized recombinant FVIII products.1

In January, Haemophilia, the official journal of the World Federation of Hemophilia, published the review article, “The first recombinant FVIII produced in human cells – an update on its clinical development programme,” revealing the results of Octapharma’s most recently completed research.

The clinical studies in adult and adolescent patients with severe hemophilia A resulted in a 96% reduction in the monthly rate of bleedings in patients on prophylactic

regimens versus those receiving on-demand treatment, the authors noted. There were no product-related serious adverse events and none of the previously treated patients treated with Human-cl rhFVIII developed inhibitors or allergic reactions, according to the article.

“There is an increasing interest in a recombinant FVIII concentrate derived from a human cell-line,” said Octapharma USA President Flemming Nielsen. “The development of inhibitors remains one of the major challenges in hemophilia care. Octapharma looks forward to addressing unmet patient needs in this area.”

Octapharma was among the first to develop human proteins based on recombinant technologies from human cell-lines. The company’s new 110,000-square-foot R&D facility in Heidelberg, Germany, which opened in 2012, is just one example of the company’s continuing commitment to recombinant science.

References 1 – Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review, published in Haemophilia 9 (2003), 418-435.

BLA Submitted for First Purely Human RecombinantFVIII Product Produced from a Human Cell-Line

Octapharma’s R&D facility in Heidelberg, Germany, where its human cell-line research is based.

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July 2014

Octapharma USA, Inc.• 121 River Street, Suite 1201• Hoboken, NJ 07030 • 201-604-1130 • www.octapharmausa.comDate of Preparation 7/14. OCTA-201-PPO

Research Presented, SymposiaOffered at Medical Congresses Octapharma USA has been active at many recent national and international medical congresses, sponsoring workshops and presenting scientific research, part of the company’s continuing commitment to share product and industry information at important provider and patient meetings.

The coagulation team participated in the World Federation of Hemophilia (WFH) Congress in Melbourne, Australia in May, sponsoring a symposium on Octapharma’s human cell-line recombinant factor VIII (Human-cl rhFVIII), the first purely human recombinant coagulation FVIII from a human cell-line. See the related story on Human-cl rhFVIII in this issue of the newsletter.

Octapharma USA presented four clinical posters at the April scientific meeting of Thrombosis and Hemostasis Society of North America (THSNA) in Chicago, emphasizing the company’s commitment to support the bleeding and clotting disorders community. The titles of the Octapharma poster presentations were:

• Global Development Plan for a Double Virus Inactivated Fibrinogen Concentrate for the Treatment of Congenital Fibrinogen Deficiency • A Clinical Study in Previously Untreated Patients with Severe Haemophilia A – Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII • Individualized prophylaxis with Human-cl rhFVIII in Adult Patients with Severe Haemophilia A • Latest Results from the PUP-GCP Clinical Trial: A Low Inhibitor Rate in Previously Untreated Patients with Severe Haemophilia A Treated with a Plasma-derived FVIII Concentrate

The critical care team participated in the South Central Association of Blood Banks 2014 Annual Meeting in Fort Worth, Texas, March 30 – April 2, including sponsoring the workshop, “A Clinical Overview of Octaplas™ [Pooled Plasma (Human), Solvent/Detergent Treated Solution for Intravenous Infusion].” Octapharma’s Andrea Neisser-Svae, MSc, PhD, presented the workshop focusing on: the rationale for developing an S/D treated, pooled plasma product; a clinical overview including the Octaplas™ manufacturing process, biochemical, safety and tolerability profiles; and a review of Octaplas™ indications and usage as well as a clinical study synopsis. A similar workshop was sponsored at The American Society for Apheresis (ASFA) & World Apheresis Association (WAA) Joint Conference in April in San Francisco.

“We find these meetings to be valuable opportunities to demonstrate the company’s leadership to both medical providers and patients,” said Octapharma USA President Flemming Nielsen. “Our goal is to keep the community informed on important trends in the industry as well as Octapharma product developments. The fascinating research presented at the THSNA meeting underscores Octapharma’s strong commitment to advancing coagulation science. Octapharma and our global research and development team is committed to creating medical advances that result in life saving therapies for people throughout our product line.”

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