37th Annual J.P. Morgan Healthcare Conference

Jean-Jacques BienaiméChairman and Chief Executive Officer

BioMarin Pharmaceutical Inc.

2019

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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.

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News Today: Progress of 3 Transformative Products Anticipated in 2019

• CHMP opinion anticipated 1Q19; Potential EU approval 2Q19 (new!)

• 2019 FY revenues expected to be between $70M-$100M (new!)

Vosoritide• Global Phase 3 enrolled; data expected YE 2019

• 0-5 y/o study enrollment on track and generally well-tolerated in early

dosing (new!)

(Adult phenylketonuria)

(achondroplasia)

Valoctocogene

Roxaparvovec

• Enrollment complete for Accelerated filing requirements (new!)

• Accelerated Approval filing decision tracking to 2H19

(severe Hemophilia A)

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7 Approved Products Expected to Deliver $2B in Revenues in 2020 Palynziq approved 2Q18 by FDA, potential EU approval 2Q19

Commercialized Products

Vosoritide for Achondroplasia

Valoctocogene Roxaparvovec for Hemophilia A (under AA)

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

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Demonstrated Track Record of Consistent and Growing RevenuesExpect commercial base to drive 15%+ top-line growth through 2020 followed by acceleration

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

$26

$325$297

$122

$441$376

$84

$501$549

$751

$890

$1,117

$1,313

$1,470 -

$1,530

$460-

$500

$325-

$355

$440-

$480

$35-

$55

(Revenues in millions)

Brineura

Vimizim

Naglazyme

Kuvan

Aldurazyme + Other

• CHMP opinion anticipated 1Q19; Potential EU approval 2Q19 (new!)

• 2019 FY revenues expected to be between $70M-$100M (new!)(Adult phenylketonuria)

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Strong Initial US Launch Continues; Metrics as of December 31, 2018 (new!)PKU 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

72

252 = 112 140

154

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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

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Palynziq CHMP Opinion Anticipated 1Q19 (new!)Potential EU approval 2Q19

Adult PKU Patients in Europe and Turkey

PKU patients defined as patients diagnosed through newborn screening

EU Market Attributes:• Large initial commercial market of

4,900 in-clinic adult PKU patients

• 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

Vosoritide• Global Phase 3 enrolled; data expected YE 2019

• 0-5 y/o study enrollment on track and generally well-tolerated in early

dosing (new!)(achondroplasia)

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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

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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

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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) enrolling well and generally well-tolerated in early dosing with no symptomatic AEs identified

• Large contemporaneous Natural History Data to Assess Final Adult Height

NEXT STEPS: Phase 3 data YE 2019

Valoctocogene

Roxaparvovec

• Enrollment complete for Accelerated filing requirements (new!)

• Accelerated Approval filing decision tracking to 2H19

(severe Hemophilia A)

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Valrox Targets Substantial Improvement over Standard of Care in Hemophilia A

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 life

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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ValRox 2019: Key Anticipated Milestones

Phase 1/2 data with 6e13 dose

• 3-year update “mid-year” at a key medical meeting; potential top-line data update prior to that

• Data to be included in expedited review package with goal of continued hemostasis control

Phase 3 with 6e13 dose

• Powered to demonstrate superiority vs. Standard of Care

• Enrollment completion (includes 6-month run-in; N=130) expected in the third quarter

• Regulatory requirements for durability for full approval will be established over the year following dosing

Accelerated Approval Pathway

• AA filing decision tracking to 2H19 and will include cohort of Phase 3 subjects already enrolled

• Regulatory requirements for durability for AA will be established within one year following dosing in a smaller subset of patients from Phase 3 cohort

• Comprehensive CMC package to be included

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Key Considerations for Expedited Valrox Registration in the US 2019

Key Filing Elements for Accelerated Approval

Choice of Factor VIII Assay Chromogenic acceptable

FVIII in normal range Acceptable primary endpoint for registration

ABR FVIII levels reasonably likely to predict reduction

Longer-term data 3.5 years at time of filing

Comprehensive CMC Package Must use to-be-commercialized materials in trials

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FVIII Activity Levels in Normal Range with Chromogenic AssayValrox 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

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Sustained Reduction of Annualized Bleed Rates 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

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Pre-infusion Post-infusion(52 weeks)

Post-infusion(104 weeks)

AB

R (

ep

iso

de

s/ye

ar)

median mean

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Global Hemophilia A Market in 2016 was $8.4B1

Fully-compliant, WAC pricing for FVIII replacement in adults is $403K-$674K per year 2

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 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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In-house Manufacturing Capabilities Support more Rapid Development Program

Reduces Risk By Developing Commercial Ready Processes To Support Pivotal Clinical StudiesBMN 270 Phase 3 Studies Being Conducted With Material Made At Commercial Scale in the to be Commercial FacilityBMN 307 Clinical Studies Will Be Conducted With Material Made At Commercial Scale in the to be Commercial Facility

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 Facility

BioMarin’s

What’s Next?

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

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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

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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

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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

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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

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2018 Final Financial Guidance Largely in-line with Prior Guidance

Revenue Guidance ($ in millions)

Item 2018 Guidance Final 2018 Guidance

Total BioMarin Revenues $1,470 to $1,530 Unchanged

Vimizim Net Product Revenue $460 to $500 Unchanged

Naglazyme Net Product Revenue $325 to $355 Unchanged

Kuvan Net Product Revenue $440 to $480 $430 to $450

Brineura Net Product Revenue $35 to $55 Unchanged

Palynziq Net Product Revenue $10 to $14 Unchanged

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

Item 2018 Guidance

Cost of Sales (% of Total Revenue) 20.0% to 21.0% 20.25% to 21.25%

SG&A Expense $575 to $615 Unchanged

R&D Expense $680 to $710 Unchanged

Non-GAAP Net Income $100 to $140 $90 to $105

GAAP Net Loss $(115) to $(165) $(100) to $(115)All Financial Guidance items are calculated based on Generally Accepted Accounting Principles (GAAP) with the exception of Non-GAAP Income. Refer to Non-GAAP Information beginning on page 10 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the comparable GAAP reported information.

Reaffirmed Y/Y revenue growth of approximately 15% through 2020; $2B in 2020

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THANK YOU