39th Annual J.P. Morgan Virtual Healthcare Conference

January 11, 2021

BioMarin Pharmaceutical Inc.Jean-Jacques Bienaime, Chairman and CEO

Global Leader in Rare Disease Therapeutic Discovery,

Development and Commercialization

2

Safe Harbor Statement

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

3

BioMarin is Leveraging Today’s Foundation to Drive Future Growth

Durable, scalable commercial portfolio drives stable revenue base

Sustainable, growing pipeline of products under development

Revenue from new products expected to grow margins and profitability

Legacy product revenues support expansion into pipeline of larger indications

New, transformative therapies offer blockbuster potential

Integrated, established “plug and play” manufacturing and commercial footprint

Financial stability & robust

long-term prospects

Commercial portfolio

supports pipeline of next

generation products

Proven drug development

expertise and capabilities to

facilitate future approvals

Honed and efficient R&D engine leveraging biologics and gene therapy

5-year average from Phase 1 to approvals with WW rights to all products

7/8 programs commercialized since 2005 – 87% success rate

4

BioMarin is Fully Integrated, End-to-End

Commercial infrastructure

supporting six approved therapies

✓ Serve over 75 international markets

✓ End-to-end commercial capability,

including patient identification, access,

availability and sales

High-Science, Innovative

Approach to Drug Discovery

✓ Rapidly expanding Gene Therapy

platform

✓ 5 of 6 current marketed products

were developed in-house

✓ 4 of 4 pipeline products were

discovered in-house

Highly Efficient and Effective

R&D Machine with Strong

Regulatory Capabilities

✓ Average 5 years from IND filing to

approval

✓ >87% success rate in pivotal studies

Best-in-Class Commercial

Manufacturing Capabilities

✓ 2 wholly-owned cGMP biologics

facilities

✓ First, and largest, gene therapy

manufacturing facility

5

Diversified Global Business in over 75 Countries Drives Steady and Consistent Revenue Growth

Currently Approved Products Continue

to Drive Steady Revenue Growth

FY 2018 actuals2014 2015 2016 2017 2018 2019 2020

$751$890

$1,117

$1,491

$1,313

$1,704

Global Revenue Contributions for

BMRN Marketed Products

43%

31%

14%

12%

US Europe LatAm ROWFY 2019 actuals

2020E

YTD$1,408A

(1Q-3Q

2020)

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BioMarin’s Late-stage Pipeline Addresses Significantly Larger Patient Populations within our Commercial Footprint

1. Patient population includes PKU patients ≥ 16 y/o in EUMEA, ≥ 18 y/o in U.S. 2. Patient population includes adults with severe hemophilia A, some of whom may not meet all eligibility criteria for valoctocogene

roxaparvovec 3. Population includes people <20 y/o with achondroplasia and open growth plates 4. EPI Data from 2018 WFH Annual Survey; NHF website: http://www.hemophilia.org/Bleeding-Disorders/Types-

of-Bleeding-Disorders/Hemophilia-A

Phenylketonuria1

Severe Hemophilia A2

Achondroplasia3

~12K

~12.8K

~3.5K

~3K

~13K

~5.5K

~20K

~40K

~10K~3K

~6.7K

~2K

Hemophilia A Severity4

~25%

Mild

~60%

Severe

~15%

Moderate

Growth potential across phenylketonuria, severe hemophilia A and achondroplasia patient populations

7*Patients addressable for Palynziq include people with PKU ≥ 16 y/o in EUMEA and ≥ 18 y/o in U.S. PKU patients defined as patients diagnosed through newborn screening. Out-of-clinic patients are those who have been diagnosed, but have not returned to clinic in at least 2 years

Significant Growth Opportunity Ahead for Palynziq

~1,800~1,000

~8,000

~19,200

On Kuvan On Palynziq In Clinic Out-of-Clinic$4.1

$8.1

$12.3

$18.8

$24.1

$31.7$34.6

$40.7

$46.1

$0.0

$5.0

$10.0

$15.0

$20.0

$25.0

$30.0

$35.0

$40.0

$45.0

$50.0

3Q18 4Q18 1Q19 2Q19 3Q19 4Q19 1Q20 2Q20 3Q20

Revenue

Palynziq Revenue Growth Since Launch ($ in millions)

Total Patients:

~30,000

U.S. and EUMEA Addressable Adult PKU Patients*

Palynziq and Kuvan are the only approved

products to treat PKU

In 3Q20 FDA approved label expansion to include maximum dose of 60 mg (previously 40 mg)

valoctocogene roxaparvovec (BMN 270)gene therapy for severe hemophilia A

Phase 3 Study (N=132) with More than 1 Year of Data Met all Primary and Secondary Endpoints

Demonstrating Superior Efficacy to Prior Factor VIII Prophylaxis

• Significantly Reduced Mean Annualized Bleeding Rate by 84%

from 4.8 to 0.8 (N=112) (p-value <0.0001)

• Superior to Factor VIII Replacement Therapy with 99% Reduction in Mean Annualized Factor VIII

Infusion Rate (p-value <0.0001)

• FVIII Expression at One Year is Mean 42.89 IU/dL (N=132)

• In Subset Dosed More than Two Years Ago, Slower Rate of Decline in Factor VIII Expression was

Observed Compared to Prior Study: Mean ABR in This Population was 0.9 Over These Two+ Years

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Valoctocogene Roxaparvovec Phase 3 Study

• N=132 Modified intent-to-treat (mITT), all who received valoctocogene

roxaparvovec

• N=112 Rollover subjects who completed approximately 6 months non-

interventional baseline observational study prior to receiving valoctocogene

roxaparvovec

• N=17 Subset of mITT who were non-rollover subjects and received

valoctocogene roxaparvovec at least two years prior to the November 2020

data cut

Phase 3 Patient Population Definitions

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ABR and FVIII Utilization Both Show Striking Reductions Post-Infusion

2.8

0

4.8

0.8

0

1

2

3

4

5

6

Baseline Week 5 to Last Visit

AB

R (

ble

eds/

yea

r)

Median Mean

Rollover Subjects (N=112) Rollover Subjects (N=112)

% Subjects Bleeding Free

Baseline Week 5 to Last Visit

32.1% 79.5%

% Subjects FVIII Infusions Free

Baseline Week 5 to Last Visit

0.0% 67.0%

83.8% reduction in mean ABR to 0.8

(post- vs. pre-baseline)

128.6

0

135.9

2.00

20

40

60

80

100

120

140

160

Baseline Week 5 to Last Visit

FV

III

Uti

liza

tio

n (

infu

sio

ns/

yea

r)

Median Mean

98.6% reduction in mean FVIII

utilization to 2.0(post- vs. pre-baseline)

Valoctocogene Roxaparvovec Phase 3 1-year Results

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Mean/Median Annualized Bleeding Rate (ABR) and FVIII Utilization in Phase 3 Study from Week 5 Until Week 52 and Phase 1/2 Study within the First Year

Phase 3

rollover population

On Factor VIII

prophylaxis, Before

valoctocogene

roxaparvovec

infusion

N=112

Phase 3

rollover population

After

valoctocogene

roxaparvovec

infusion

N=112

Phase 1/2

6e13 vg/kg cohort

On Factor VIII

prophylaxis,

Before

valoctocogene

roxaparvovec

infusion

N=6

Phase 1/2

6e13 vg/kg cohort

After valoctocogene

roxaparvovec

infusion

N=6

Phase 1/2

4e13 vg/kg cohort

On Factor VIII

prophylaxis,

Before

valoctocogene

roxaparvovec infusion

N=6

Phase 1/2

4e13 vg/kg cohort

After

valoctocogene

roxaparvovec

infusion

N=6

Mean

Median

Mean

Median

Mean

Median

Mean

Median

Mean

Median

Mean

Median

Annualized

Bleeding Rate

(bleeding episodes

per year)

4.8

2.8

0.8

0.0

16.3

16.5

1.3

0.0

12.2

8.0

0.9

0.0

Annualized FVIII

Infusion Rate

(infusions per year)

135.9

128.6

2.0

0.0

135.6

136.5

1.8

0.0

142.8

155.8

1.6

0.0

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FVIII Activity Levels for N= 112 Rollover Population in the Mild Hemophilic Range at 1 Year

Valoctocogene Roxaparvovec Phase 3 1-year Results

Mean

Median

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FVIII Activity Levels for Final Analysis mITT N= 17 Population Dosed ≥2 Years

Valoctocogene Roxaparvovec Phase 3 2-year Results

Mean

Median

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• In the Phase 3 study, valoctocogene roxaparvovec has been well tolerated by the participants who

received a single 6e13 vg/kg dose

• No participants developed inhibitors to Factor VIII, or thromboembolic events

• Most common side effects were infusion reactions, ALT elevation, and steroid-related side effects

with most resolved during the study

Summary of Safety

Valoctocogene Roxaparvovec Phase 3 1-year Results

valoctocogene roxaparvovec (BMN 270)gene therapy for severe hemophilia A

Next Steps:

• Expect to submit MAA in Europe 2Q21

• Share Phase 3 52-week results with FDA

vosoritide for achondroplasia

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No Approved Medical Therapies; only Treatment Available is Limb-Lengthening Surgery

Achondroplasia: The Most Common Form of Dwarfism

• Caused by a mutation in the fibroblast growth receptor 3 gene (FGFR3), a negative regulator of bone growth

• 80% of cases occur from parents of average stature (due to spontaneous mutation in FGFR3)

• In addition to short stature, serious medical manifestations include:

• Foramen magnum compression, Sleep apnea, Bowed legs, Permanent sway

of the lower back, Spinal stenosis & Obesity

• Most common skeletal dysplasia - 1:25,000 birth worldwide

• ~22,000 children with achondroplasia in BioMarin global territoriesEndochondral

bone (in blue)

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Phase 3 Study 12-Month Results of Placebo Arm After Crossover to Vosoritide1-year after switching, placebo patients had similar AGV as the active treatment arm

12-Month Interval AGV over time in Full Analysis Set (Subjects With A Height Assessment at Week 52 in 302 / Week 104 in 301)

Baseline in 301 Week 52 in 301 Week 52 in 302 / Week 104 in 301

Crossover Arm (Placebo to 15 ug/kg, N=54): Placebo 15 ug/kg

Me

an A

GV

(C

M/Y

ear

)

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Encouraging Sustained Growth Effects with Vosoritide in Phase 3 Program through 2 Years (announced December 21, 2020)

• At year 2, Phase 3 demonstrated cumulative height gain of 3.52 cm (p<0.0001) compared to untreated children

• After 1 year of treatment, Phase 3 demonstrated 1.6 cm/year (p<0.0001) placebo-adjusted additional increase from baseline in growth velocity

• The year 2, Phase 3 data demonstrated that vosoritide, administered at 15ug/kg/day was generally well tolerated with no new safety findings

2021

Catalysts:

• CHMP opinion in E.U. expected 2H21

• PDUFA Action Date in U.S. August 20, 2021

• Potential global launch 4Q21

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Broad Life Cycle Management Program with Vosoritide

Multiple Expansion Opportunities Across Current Program

Product Indication Preclinical Phase 1 Phase 2 Phase 3 BLA/MAA Submitted 2021 Goals

Vosoritide Achondroplasia 5-18 y/o Approvals in US and Europe

VosoritideGenetic forms of Short Stature (GSS) *

Ongoing Phase 2 study enrollment

Vosoritide Achondroplasia 0-5 y/o Phase 2 study completion

Vosoritide Foramen Magnum 0-1 y/o Ongoing Phase 2 study enrollment

Near-term additions to next generation portfolio:

• Enhanced drug-delivery system

• Long-acting formulation under development that could be covered by recently issued U.S. patent

Longer-term expansion within GSS indications, representing up to 275,000 patients

*Under development as part of a research collaboration with Children’s General

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Durable and Scaled

Our Business is Built for Continued Growth

Marketed Enzyme Replacement Therapy (ERT) Portfolio

Near-Term Driver

Late-Stage, Large-Market Pipeline

Horizon Value

Early-Stage Next-Generation Therapies

Today

Tomorrow

1 2 3

2020E Rev = $1.8bn+

Stable revenue,

positive operating

cash flow

Vosoritide

❖NDA and MAA opinions 2H21

Roctavian

❖Re-submit to EMA in 2Q21; share 1-year

data with FDA

Vosoritide

❖First and only non-surgical treatment for

achondroplasia

Roctavian

❖First gene therapy for

Hem A

Pipeline leveraging proven

gene therapy capabilities

PKU

Achondroplasia

MPS IVA

MPS VICLN2

Disease

Hemophilia A

BMN 307

PKUBMN 331

HAE

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BioMarin Pipeline: Built on Core Attributes and Leverages

Known Genetic MechanismMinimizes Efficacy Risk

Significant Clinical ImpactMaximizes Value

Large Effect SizesEnables Disease Normalization

Proximal EndpointsEnables Rapid Development

Pro

ba

bili

ty o

f S

ucce

ss

Sp

ee

d &

Effic

iency

Retain Core Attributes of BioMarin ProgramsTo Maximize Probability of Success and Speed

Leverage Investments, Expertise and Science

1

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FranchiseOne Disease

Multiple ProgramsLeverage scientific & commercial

expertise to benefit all patients

ExpansionOne Program

Multiple IndicationsLeverage genetics and biology to

maximize potential of a given therapy

Gene TherapyGene Therapy

Multiple DiseasesLeverage investments in gene therapy

to transform disease treatments

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Gene Therapy• ROCTAVIAN Hem A – Ph3; MAA 2021

• BMN 307 PKU – Ph1/2

• BMN 331 HAE – IND Enabling

• DiNA-001 MYBPC3 HCM – Preclinical

• Multiple Preclinical Programs

• Genetic Cardiomyopathies & Neurological Disorders

Employing Multiple Modalities to Optimally Treat Rare Disease

Peptide/Biologic Therapy• Vosoritide ACH – NDA/MAA 2020

• Vosoritide GSS – IST

• Multiple Preclinical Programs

• Genetic Cardiovascular, Neurological &

Skeletal Disorders

ACH: Achondroplasia, GSS: Genetic Forms of Short Stature, IST: Investigator Sponsored Trial (Children’s National), Hem A: Hemophilia A, PKU: Phenylketonuria, HAE: Hereditary Angioedema, MYBPC3: Myosin-Binding Protein C, HCM: Hypertrophic Cardiomyopathy, DMD: Duchenne Muscular Dystrophy

Oligonucleotide Therapy• BMN 351 DMD – IND Enabling

• Rare Disease Indications (4) - Preclinical

• Multiple Preclinical Programs

• Genetic Neurological & Neuromuscular Disorders

Small Molecule Therapy• BMN 255 – IND 2020 (Chronic Renal Disease Subset)

• Undisclosed In-licensed Asset - Preclinical Tox

• Multiple Preclinical Programs

• Genetic Cardiomyopathies

• In 2020, doubled pre-clinical pipeline via internal growth and external partnerships

• Secured 10 assets through 6 deals (2 announced)

• Balanced portfolio of gene therapies, oligonucleotide therapies, biologics and small molecules

• Therapeutic emphases on rare metabolic, cardiac, neurological/neuromuscular, skeletal disorders

THANK YOU