BioMarin Pharmaceutical Inc.

2Q19

2

Safe Harbor Statement

This non-confidential presentation contains‘forward-looking statements’about the business prospects of BioMarin Pharmaceutical Inc., including potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin’s product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission such as 10-Q, 10-K and 8-K reports.

3

7 Approved Products Targeted to Deliver $2B in Revenues in 2020

Commercialized Products

Vosoritide for Achondroplasia

Valoctocogene Roxaparvovec for Hemophilia A (under AA scenario)

Tralesinidase Alfa for MPS IIIB, or Sanfilippo Type B

PHASE 1 PHASE 2 PHASE 3 BLA/NDA/MAAProduct Development Pipeline

IND/CTA

BMN 290 for Friedreich’s Ataxia

BMN 307 Gene Therapy for PKU

4

Demonstrated Track Record of Growing Revenues

2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019E

$26

$325$297

$122

$441$376

$84

$501$549

$751

$890

$1,117

$1,491

$530-

$570

$350-

$380

$420-

$460

$1,313

(Revenues in millions)

Palynziq

Brineura

Vimizim

Naglazyme

Kuvan

Aldurazyme + Other

$70- $100

$55- $75

2019 FY Guidance

$1,680 - $1,750

Commercial base business expected to drive 15%+ top-line growth Y/Y in 2019

5

Late-stage Product Highlights and Anticipated Near-term Catalysts

• EU approval received May 6, 2019

• US launch drives 414 patients on reimbursed Palynziq at end of 1Q19

• 2019 FY revenues expected to be between $70M-$100M

Vosoritide• Global Phase 3 enrolled; data expected YE 2019

0-5 year-old study underway:

• Cohort 1 (24 to 60 month olds) nearly finished enrolling;

• Cohort 2 (6 to 24 month olds) enrolling

(Adult phenylketonuria)

(achondroplasia)

Valoctocogene

Roxaparvovec

• Manufacturing campaigns and enrollment for AA filing completed

• Complete enrollment in Phase 3 for “Full” Approval anticipated 3Q19

• Accelerated Approval filing decision tracking to 2H19(severe Hemophilia A)

Valoctocogene

Roxaparvovec

• Manufacturing campaigns and enrollment for AA filing completed

• Complete enrollment in Phase 3 for “Full” Approval anticipated 3Q19

• Accelerated Approval filing decision tracking to 2H19(severe Hemophilia A)

7

ValRox 2019: Key Development/Regulatory/Commercial Activities Underway

• Accelerated Approval Filing

• Full Approval Filing

• Regulatory Agency Interactions and Timelines in US and EU

• Pricing/Reimbursement: based on Clinical Outcomes and Current Established Costs

• Commercial Pre-launch Preparations and Readiness

Focus on components for both expedited and full registrations to support potential approval(s) and launch

8

ValRox 2019: Accelerated Approval Elements Tracking to Plan

Accelerated Approval considerations and activities underway:

• Phase 3 study cohort required for AA filing enrolled

• FDA considerations based solely on FVIII above 40 IU/dL through 12 months

• Manufacturing campaigns required for AA filing completed

• Longer-term data from ongoing Phase 2 study anticipated by June 7

• Chromogenic FVIII assay accepted as appropriate measure of activity levels with valrox to support accelerated approval

Key activities and read-outs to enable Accelerated Approval filing decision 2H19

9

FVIII Activity Levels in Normal Range with Chromogenic Assay at 1-yearValrox Phase 1/2 data conforms to regulatory requirements for expedited registration

The upper and lower box bounds represent 25th and 75th percentiles. The whisker lines represent the minimum and maximum values.

Expedited Registration:

Powered based on Phase 1/2

Valrox 6e13 vg/kg dose results to 52 weeks

50%-150% “normal range”

> 40% “non-hemophilic” range;

threshold for Accelerated Approval

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Sustained Reduction of Annualized Bleed Rates 2-years Post Valrox TreatmentValrox 104 week Phase 1/2 ABR data superior to Standard of Care

ABR results with 6e13 dose through week 104

% Patients Bleed Free

97% REDUCTION in MEAN ABR

Baseline Year 1 Year 2

14% 71% 86%

All patients off prophylaxis

100% resolution in target joints

As presented at WFH, May 22, 2018

16.5

0 0

16.3

0.9 0.50

5

10

15

20

Pre-infusion Post-infusion(52 weeks)

Post-infusion(104 weeks)

AB

R (

ep

iso

de

s/ye

ar)

median mean

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Valrox Substantially Improves Quality of LifeValrox 104 week Phase 1/2 QoL data superior to Standard of Care

As presented at WFH, May 22, 2018

Grey line indicate distribution based minimally clinical important difference (MCID) at lower threshold

* Pocoski J et al., 2014

Me

an

Ch

an

ge

in

Ha

em

o-Q

oL

-A T

ota

l S

co

re

6e13 vg/kg cohort

4e13 vg/kg cohort

Spinart result at 3 years*

0

2

4

6

8

10

12

14

16

18

Week 16 Week 28 Week 52 Week 78 Week 104

7.2

4.76.3

3.8

13.4 12.9

9.6

16.617.3

• QOL improvement

observed in all 6 domains:

‒ Consequences of

Bleeding

‒ Emotional Impact

‒ Physical Functioning

‒ Role Functioning

‒ Treatment Concern

‒ Worry

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Hemophilia B: Sustained FIX Levels Result in Significant Bleed Control8 year follow up from Dr. Amit Nathwani’s FIX gene therapy study in subjects with severe Hemophilia B

Nathwani et al. ASH 2018

0 2 4 6 8

0

2

4

6

Years

FIX

(%

of

no

rmal)

Low (N=2) Mid (N=2) High (N=6)

P<0.0001

5.1 1.4 IU/dL2.3 0.3 IU/dL1.9 0.6 IU/dL

FIX activity levels (mean, SD):

16.5 10

All-

Pre

All-

Post

HD-P

re

HD-P

ost

0

10

20

30

40

An

nu

alised

ble

ed

rate

(N

o/Y

ear)

p = 0.002 p = <0.001

2.2 2 21.9 11 2.3 2

Annualized bleed rateFIX levels through years 6 and 8

(Through year 6) (Through year 8)

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AAV Durability for the Life of Canine Subjects as Measured by Chromogenic Assay

BioMarin data

FVIII expression post AAV gene therapy in canine hemophilia

Chromogenic FVIII activity measured using a canine normal pooled plasma standard. Source: Batty, P. et al unpublished data

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Similar FVIII Expression Profile seen with AAV5 in Non-human Primates

BioMarin data

0 10 20 30 40 50 60 70 80 90 100 110 120 130 140 150 160 170 180 190 2000

20

40

60

80

100

120

140

160

180

200

220

240

260

280

300

Study Day

FV

III P

rote

in (

ng

/mL

)

• Independent of capsids, promoter, transgenes• Dose-dependent

Non-human primates

(cynomolgus monkeys)

* Immunosuppression regimen included in study protocol

15

Circular Episomes Persist 1 Year After Treatment; ASGCT data May 2019

• Circular episomes have been shown to be the long-lived gene-expressing form in multiple pre-clinical models of AAV gene therapy

• BioMarin assays can detect structural forms of FVIII transgene at single molecule sensitivity, using blood cells as a non-invasive alternative to liver biopsies

• Long term FVIII transgene from a liver-directed promoter is associated with circular, episomal forms inside cells• Past the time of red cell clearance, vector persists in peripheral blood mononuclear cells as circular episomes• We speculate that similar patterns of vector loss from the liver will depend on the rate of liver cell turnover

Weeks after infusion of BMN 270

Cellular Episomes Vector Cleared Late through Senescence 52 week whole blood DNA is primarily episomes in PBMCs

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ValRox 2019: Full Approval Elements Tracking to Plan

Full Approval considerations and activities with GENEr8-1 Phase 3 study:

• 95% of subjects interested in 6e13 dose study

• N=130, goal to prove “Superiority” in bleed reduction vs. Standard of Care (prophylaxis)

• 52-week study, endpoints include reduction in bleeds and FVIII usage

• Global Phase 3 study underway in 13 countries

• Enrollment completion expected 3Q19

Key activities to support full approval path forward

17

ValRox 2019: Filing Agreements Reached with Regulatory Authorities

Key agreements have been reached regarding plans for marketing application submissions in US and EU, including:

• Success criteria for Phase 3 study Interim Analysis to support US Accelerated Approval

• Structure, format, and content for all modules (CMC, Nonclinical, and Clinical)

• Data-cuts and size of the safety database

• Final results from Phase 3 study to serve as confirmatory evidence for Full Approval

Next Steps:

• Meetings with US and EU regulators planned for mid-2019, following availability of Phase 3 study interim analysis and Phase 2 study 3-year data

• Decision to pursue accelerated marketing application on track for 2H19

Breakthrough Designation and PRIME status have facilitated frequent interactions with US and EU regulators

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ValRox 2019: Pricing & Reimbursement will be Based on Clinical Outcomes Valrox targets substantial improvement over Standard of Care

High Unmet Needs with Current Standard of Care

Recurrent joint bleeds

Deterioration of target joints

Burdensome weekly infusions

Limited physical activity

Peaks and troughs

High costs for lifetime therapy

Valrox Cumulative Value Over Standard of Care

Elimination of bleeds

Resolution of target joints

One-time infusion

More active lifestyle

Meaningful QOL improvements

Cost offsets of millions over lifetime

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Recent Survey: Speedy Uptake >40% Market Share in Severe Population in 5 Years

• Hematologists anticipate robust uptake; among eligible severe hemophilia patients (see Q18) ~25% immediately after approval and ~45% after 5 years

• Of note, ~45% (severe) and ~30% (moderate) penetration rates are higher than our estimates of 15-30% in hemophilia A (Valrox, SPK-8011)

• Hematologists also projected gene therapy use among moderate hemophilia patients

Hemophilia A

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Global Hemophilia A Market in 2016 was $8.4B1

Estimated WAC pricing for Hemlibra in non-inhibitor adult patients is between $600K - $800K per year2

Fully-compliant, WAC pricing for FVIII replacement in adults is $403K-$674K per year 3

An Estimated 117K Hemophilia A Patients in our Territories 3

NORAM total: ~18,000

LATAM total: ~22,000

EUMEA total: ~64,000

APAC: ~13,000

1 Evaluate Pharma; 2 PriceRx IHA Global insight Oct. 2015-Oct. 2016 (WAC price reflects cost of Factor VIII replacement for an adult on prophylaxis)

2 Based on WAC price of $99.20 per mg $482K/year for a 58 kg subject; Centers for Disease Control and Prevention (CDC) estimates the average adult American male weighs >90 kg

3 EPI Data from 2016 WFH Annual Survey; NHF website: http://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A;

Hemophilia A Severity

Source: WFH 2016

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Multiple Lifecycle Management Opportunities Exist to Capture Majority of 117K Hemophilia A Patients in our Territories

Goal to expand into additional Hemophilia A patient subpopulations over time

• All levels of

severity

• All Ab-status

• All inhibitor status

AAV5 Ab+

All prophy

subjects

Adolescents

AAV5 Ab free, >

18 y/o, severe

• AAV5 Ab free, >

18 y/o, severe

• AAV5 Ab+

• All prophy

subjects

• Adolescents

AAV5 Ab free, >

18 y/o, severe

Valoctocogene

Roxaparvovec

Initial label

Valoctocogene

Roxaparvovec

Future label

~30,000 patients

~50,000 patients

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Annual Valrox Market OpportunityAverage price based on cost of either rFVIII or Hemlibra for a severe Hemophilia A patient

Patients Treated/ Year

Average Price: $1M(1)

Average Price: $2M(2)

Average Price: $3M

1,000 patients $1B $2B $3B

2,000 patients $2B $4B $6B

3,000 patients $3B $6B $9B

4,000 patients $4B $8B $12BBMRN current manufacturing capacity of 4,000 patients; easily scales to 5,000 patients

Initial target market: 30,000 (severe Hem A; 18 y/o +; AAV5 Ab free)

(1) 1.5 years of Hemlibra (non-inhibitor price)

(2) 3 years of Hemlibra (non-inhibitor price)

23

In-house Manufacturing Capabilities to Support Seamless Launch

FULLY INTEGRATED VECTOR PRODUCTION FACILITY

• Facility Design Vetted with Health Authorities

• Single Use Technology Throughout

• Multi-Product Production

• Supports Multiple 2000L Bioreactors

• Supports 4000 Patients/year at Highest Dose

• ISPE 2018 Facility of the Year – Project Execution

Biologics Facility Gene Therapy FacilityBioMarin’s

Ready to supply 4000-5000 patients with commercial valrox at launch; plans for next GT facility being finalized

Vosoritide(achondroplasia)

• Global Phase 3 enrolled; data expected YE 2019

0-5 year-old study underway:

• Cohort 1 (24 to 60 month olds) nearly finished enrolling

• Cohort 2 (6 to 24 month olds) enrolling

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About AchondroplasiaSpontaneous mutation that occurs in 80% of cases from parents of average stature

In addition to short stature, serious medical complications include:

• foramen magnum compression

• sleep apnea

• bowed legs

• permanent sway of the lower back

• spinal stenosis

• obesity

~ 24,000 children with achondroplasia in our global territories

26

Growth Characteristics in Achondroplasia

AchondroplasiaAverage stature

Hoover Fong et a. J Clin. Nut. 2008

4 cm/year 6 cm/year

Children with Achondroplasia Grow an Average of 4cm/year

vs. 6cm/year for Average Height Children

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DIAGNOSIS HEIGHT SD(Z-score)

ACHONDROPLASIA -6.0GH DEFICIENCY -2.7

IDIOPATHIC SHORT STATURE -2.6

TURNER SYNDROME -2.8

SMALL FOR GESTATIONAL AGE

-2.5

NOONAN SYNDROME -2.3

Magnitude of Height Deficits in Various Short Statural Conditions

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Durable Growth Sustained through 42-months with Vosoritide 15µg/kg Dose

42 Month Additional Height Gained is 5.7cm with Vosoritide

Sustained elevation of AGV shown in sequential 6-month time periods in ongoing Phase 2 study

Average Stature AGV

Baseline ACH AGV

Vosoritide (15µg/kg) n=8

29

Executing 4 Pillar Strategy to Demonstrate Improved Clinical Outcomes with Vosoritide

Comprehensive Global Development Program:

• Phase 3: Placebo-controlled, global trial (over)enrolled; 121 subjects enrolled

• Phase 2: (5 to 14 years) demonstrating additional height gain of 5.7cm at 42 months

• Phase 2: (0 to <5 years) Cohort 1 (24 -60 months) enrolled; Cohort 2 (6 -24 months) underway and generally well-tolerated in early dosing with no symptomatic AEs identified; Cohort 3 to begin enrollment once Cohort 2 complete

• Large contemporaneous Natural History Data to Assess Final Adult Height

NEXT STEPS: Phase 3 data YE 2019

(Adult phenylketonuria)

• EU approval received May 6, 2019

• US launch drives 414 patients on reimbursed Palynziq at end of 1Q19

• 2019 FY revenues expected to be between $70M-$100M

31

Palynziq Approved in the EU May 6; Preparing for Launch 2H19

PKU patients defined as patients diagnosed through newborn screening; EUMEA includes Europe, Turkey, Russia and Middle East

Market Attributes:

• Large initial commercial market of 5,800 in-clinic PKU patients aged 16 and older

• 3 years of direct experience working with PKU community to prepare for launch

• Anticipate meaningful revenue in EU starting 2020 following usual pricing and reimbursement process by country

Out of Clinic On Kuvan In-Clinic

Total: 18,000

~500

~11,700

~5,800

PKU Patients 16 years + in EUMEA

32

Strong Initial US Launch Continues; Metrics as of March 31, 2019PKU represents BioMarin’s largest US patient population opportunity

>11,000 adult patients with PKU, 3,900 actively managed in-clinic

Sites with ≥1

complete enrollment

Patients on

reimbursed Palynziq

Clinical Study

Patients

Formerly Naïve

Patients

+

Patients enrolled but not yet

reimbursed/on therapy

Breadth and depth of adoption

across key clinics, both clinical trial

sites and naïve clinics

Positive payer coverage at launch

leading to strong uptake of

reimbursed patients

Leading indicators point toward

continued uptake acceleration in

2019

89

414 = 136 278

140

33

3 Year Durability with Palynziq Strengthens EU Plans< 600 µmol/L - EU PKU guideline recommendation

< 360 µmol/L - US guideline recommendation< 120 µmol/L - physiologically normal

Proportion of Subjects Reaching Blood Phe Threshold over Time(doses up to 60mg/day) (n=285)

42%

57%

71% 74%

29%

46%

63%67%

22%

35%

54%59%

6 months 12 months 24 months 36 months

≤600 umol/L ≤360 umol/L ≤120 umol/L

Subjects reflect general adult PKU population with mean baseline blood Phe 1233µmol/L

What’s Next?

BioMarin’s Formula for “Medium Rare” Disease Drug Development

35

Our Enriched Approach for Speed and Success4 Key Criteria Guide Discovery and Development at BioMarin

• IND to approval in 4-6

years for 5 out of 7

products

• Hem A, PKU, MPS:

Rapidly gauge efficacy with

relevant endpoints

• Gene therapy to restore

FVIII expression in Hem A

and PAH activity in PKU

• Hem A, PKU, CLN2,

MPS I, IVA, VI,

achondroplasia

• High unmet need and rapid

development

• Diseases with genetic

mechanisms

• Target epicenters and drive for

normalization

• Discern outcomes through

sensitive endpoints

3

FVIII

Phe

Phe

Tyr

PAHx

1

2

3

4

36

BMN 307: Leveraging Our Leading Gene Therapy Capabilities

Pre-existing

immunity

Vector

optimization

Expression

Manufacturing

Tissue

tropism

AAV BMRNAAV9

Improved tissue tropism with novel

BMRN AAVs – muscle example

Core gene therapy expertise

across 5 domains

1

2

34

5

Reference: Data on file, BioMarin (2018)

Novel BMRN AAVs with less

susceptibility to pre-existing immunity

37

BMN 307 and the PKU Model Used for Development

Validated mouse model of PKU (the ENU2 model)

‒ Mice have no detectable PAH catalytic activity and high Phe levels

Model recapitulates many aspects of the human PKU phenotype, including:

‒ High plasma/tissue Phe

‒ Reduced neurotransmitters

PKU mice also have a light coat color

Acts as a readily detectable biomarker of

therapeutic response

BMN 307: Liver-directed gene therapy (AAV5 PAH)

IND filing in 2H19 (Commercial scale material available in 2H19)

ENU2WT

38

Lifetime Phe Correction seen in Treated PKU Mice with BMN 307; IND 2H19

Reference: Data on file, BioMarin (2018)

Phenylalanine reductions seen in ENU2 mice

Phe in µM

2 weeks

ENU2 vehicle

ENU2 + AAV5 muPAH

WT vehicle

ENU2 + AAV5 PAH

• AAV5-PAH

normalizes Phe in

ENU2 mice

• Levels

indistinguishable

from WT after 2

weeks

• Efficacy

sustained at 80

weeks

39

2019 Financial Guidance

Revenue Guidance ($ in millions)

Item 2019 Guidance

Total BioMarin Revenues $1,680 to $1,750

Vimizim Net Product Revenue $530 to $570

Kuvan Net Product Revenue $420 to $460

Naglazyme Net Product Revenue $350 to $380

Palynziq Net Product Revenue $70 to $100

Brineura Net Product Revenue $55 to $75

Selected Income Statement Guidance ($ in millions, except percentages)

Item 2019 Guidance

Cost of Sales (% of Total Revenue) 20% to 21%

SG&A Expense $650 to $690

R&D Expense $740 to $780

Non-GAAP Net Income $130 to $170

GAAP Net Loss $(45) to $(85)

15% Revenue Growth Anticipated Y/Y in 2019; and $2B in Revenue Targeted in 2020

40

THANK YOU

as of May 13, 2019