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
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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.
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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
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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
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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