BioMarin Pharmaceutical : Third Quarter 2025 Presentation

Third Quarter 2025 Earnings

October 27, 2025

Q3'25 Earnings Agenda:

1

2

3

Key Business Updates Financial Results Commercial Update

4

Research & Development Update

5

Q&A

Alexander Hardy

President and CEO

Brian Mueller

Chief Financial Officer

Cristin Hubbard

Chief Commercial Officer

Greg Friberg

Chief R&D Officer

3

Q3'25 Key Business Updates

Alexander Hardy President and Chief Executive Officer

4

Building on Our Strong Foundation to Drive Growth in 2025 and Beyond

Year-to-date 2025 Total Revenues 11% Y/Y

Led by more than 20% Y/Y revenue

growth for PALYNZIQ and VOXZOGO

Enzyme Therapies 1

Executing on Focused Strategy

• Raising Full-year 2025 Total Revenues guidance to between $3,150M and

  $3,200M2; 11% FY'25 growth Y/Y3

• Reaffirming VOXZOGO FY'25 revenue

  outlook of between $900M and $935M

  $2,347M

  Q3'25 YTD Total Revenues

  VOXZOGO

  ROCTAVIAN & Other
  
  

  • Focused Portfolio Strategy; Pursuing Options to Divest ROCTAVIAN

5

1Enzyme Therapies include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ, and VIMIZIM; 2FY'25 Total Revenues guidance raised at the midpoint; 3Y/Y growth at the midpoint

Q3'25 Financial Results

Brian Mueller Executive Vice President, Chief Financial Officer

6

VOXZOGO & PALYNZIQ Strength Drive Increased FY'25 Guidance

FY'25 Total Revenues guidance increased to

$3,150M - $3,200M

Total Revenues ($M)

Y/Y Change

Q3 YTD

VOXZOGO $746 $776

+4% +11%

• Strong global demand and new patient increases Q/Q

• Q3 year-to-date revenue increased 24%, Y/Y

  190

  509

  47

  218

  516

  42

  +15%

  +1%

  (11%)

  +24%

  +8%

  (2%)

• Reaffirming $900M - $935M FY'25 revenue

  Enzyme Therapies

• Q3 year-to-date revenue increased 8%, Y/Y

• PALYNZIQ strength continues; third consecutive quarter of 20%+ Y/Y revenue growth

• Q3'25 Enzyme Therapies revenue lower than Q2'25; strong underlying demand offset by large orders for

  
  
  

  Q3 2024 Q3 2025

  NAGLAZYME and VIMIZIM in Q2

  • Higher Q3'24 ALDURAZYME volume

    VOXZOGO
    
    Enzyme Therapies
    
    ROCTAVIAN &
    
    

    Other

    7
    
    
    
    

    Q3'25 Reflects Acquired IPR&D Impact; Strong Underlying Execution

    Q3 Results Include Impact of $221M Acquired IPR&D Charges on a Pre-Tax Basis

Q3 2025 and YTD Results

Q3'25

Q3 Y/Y

YTD Y/Y

GAAP R&D $409 +121% +27%

GAAP Operating Margin (6.0%) (21.3) ppts +4.1 ppts

Non-GAAP SG&A1 $223 +23% +8%

GAAP Diluted EPS ($0.16) (129%) 31%

Operating Cash Flow $369 +66% +88%

3%

(87%)

$0.12

Non-GAAP Diluted EPS1

(24.9) ppts (1.4) ppts

2.8%

Non-GAAP Operating Margin1

+30%

+127%

$395

Non-GAAP R&D1

(5%)

6%

$268

GAAP SG&A

In millions, except percentages

Non-GAAP Operating Margin and Non-GAAP Diluted EPS

• Q3 acquired IPR&D reduced Non-GAAP Operating Margin and Non-GAAP Diluted EPS (~$1.10 per share impact)

• Year-to-date EPS growth reflects underlying strong revenue performance and operational efficiencies

• FY'25 Non-GAAP Operating Margin guidance updated to 26% - 27% and FY'25 Non-GAAP Diluted EPS guidance updated to $3.50 - $3.60

  Operating Cash Flow

• Significant operating cash flow growth continues, with

$369M generated in Q3, contributing to ~$2B cash and investments balance as of quarter-end

8

1Refer to slide 2 for more detail on Non-GAAP financial measures

Updated Full-year 2025 Guidance

Prior Guidance (In millions , except per share and % data) As of August 4, 2025	Updated Guidance As of October 27, 2025
Total Revenues 1 $3,125 to $3,200 Non-GAAP Operating Margin %2 33% to 34% Non-GAAP Diluted EPS2,3 $4.40 to $4.55	$3,150 to $3,200 26% to 27% $3.50 to $3.60

Guidance updated October 27, 2025 includes acquired IPR&D charges through Q3 2025 of

$221 million on a pre-tax basis, resulting in an approximate impact of 7% on Non-GAAP Operating Margin, or $1.10 on a per share basis

1VOXZOGO contribution to full-year 2025 Total Revenues expected to be in the range of $900 million to $935 million. 2Refer to slide 2 for more detail on Non-GAAP financial measures; 3Non-GAAP Diluted EPS guidance

9

assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.

Q3'25 Commercial Update

Cristin Hubbard Executive Vice President, Chief Commercial Officer

10

Enzyme Therapies

Enzyme Therapies Growth Reflects Broad Uptake Across Portfolio

Enzyme Therapies Q3 YTD Revenue

($M)

+8% Y/Y

(1%)

+21%

+10%

+13%

$137

$159

$308

$365

$587

+7%

Year-to-date high-single-digit growth aligned with long-term outlook

• Sustained PALYNZIQ growth driven by greater numbers of patients titrating to daily maintenance dose and strong adherence

  • Pursuing age label expansion to adolescents in

    U.S. and Europe, with potential approval in 2026

• YTD results reflect increased new patient starts across all Enzyme Therapies , despite quarter-to-quarter order timing dynamics

  
  
  11
  
  
  
  

  Skeletal Conditions

  VOXZOGO: Q3 YTD 24% Y/Y; 2025 Outlook Reaffirmed

  Revenue ($M)
  
  

  $654

  +15%

  $218

  
  
  +24%

  Q3 2025 Q3 2025 YTD

  Percentages reflect year-over-year change

  Q/Q new patient adds across all regions; Q4'25 VOXZOGO revenue expected to be highest of the year

• VOXZOGO available in 55 countries at end of Q3'25

• Continued new patient increases globally Q/Q

• ~75% year-to-date VOXZOGO revenue generated outside of the U.S. (OUS); strong uptake across international markets

• U.S. dynamics in Q3'25

  • Children under 2 years old represented of majority of Q3

    U.S. new starts

  • Due to geographical dispersion of older children, initiatives were implemented to support uptake

  • VOXZOGO prescriber base continues to expand

• VOXZOGO Q4'25 revenue expected to be highest of the year driven by timing of large OUS orders, deeper penetration in existing markets and new patient starts

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  Skeletal Conditions

  Building on our Leadership in Skeletal Conditions

  Total Addressable Patient Population (TAPP): ~420,000

  Focus ed on mos t s everely impacted s ubs et of

  Indication	TAPP
  Achondroplasia	24,000
  Hypochondroplasia	14,000
  Idiopathic short stature	190,000
  Noonan syndrome	90,000
  Turner syndrome	65,000
  SHOX deficiency	40,000

  children, repres enting modes t proportion of TAPP

  Expansion Across Multiple New Indications

• Achondroplasia (ACH): firs t of six indications for treatment with VOXZOGO or BMN 333

• Preparing for VOXZOGO in hypochondroplasia

  (HCH) launch: pivotal data readout expected 1H'26

• CANOPY Phase 2 clinical trials with VOXZOGO advancing across four additional indications : idiopathic short stature, Noonan syndrome, Turner syndrome, SHOX deficiency

  TAPP defined as the diagnosed, clinically eligible patients for a given product or program in a defined footprint; footprint for above indications (except ISS) defined as all markets included in BioMarin's internal projections for VOXZOGO revenues.

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  Idiopathic short stature (ISS) footprint is US-only. Height Z-score eligibility for TAPP: ACH (N/A); HCH (≤ -2.0 standard deviations (SDs)); ISS (≤ -2.5 SDs); Noonan syndrome (≤ -2.5 SDs); Turner syndrome (≤ -2.5 SDs); SHOX deficiency (≤ -2.5 SDs)

  
  
  

  Skeletal Conditions

  VOXZOGO for HCH: Significant Breakthrough for Patients

  Key Challenges to HCH Diagnosis Potential to Improve HCH Diagnosis

  Heterogenous presentation

  
  
  
  • VOXZOGO in ACH launch experience and commercial leadership has laid the foundation to scale and execute effectively in future indications

    Complex referral pathway

    
    
    
  • Global initiatives to improve early HCH diagnosis include:

    • Targeted genetic reclassification

    • Clinician education

      Low urgency to diagnose

      
      
      

    • Patient/caregiver awareness

    • Diagnostic pathway optimization

  • Robust engagement and enthusiasm from HCPs

    Various Barriers to

    Genetic Testing

    across specialties; reflection of the growing recognition of the unmet needs in HCH

  • VOXZOGO in HCH: Phase 3 data expected 1H'26

and potential launch in 2027

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Q3'25 R&D Update

Greg Friberg Executive Vice President, Chief R&D Officer

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Skeletal Conditions

Potential to Address Significant Unmet Need in Hypochondroplasia

Hypochondroplasia Unmet Needs

• A retrospective study of children and adults with HCH showed higher co-morbidities, procedures, and health care resource utilization as compared to the general population1

  • Prominent comorbidities included respiratory, orthopedic, mental health, and ENT

  • Overall, HCH patients had higher rates of surgeries driven by respiratory, orthopedic, and ENT

• Quality of life is affected in individuals with HCH, most notably due to impact on functional activities2,3

  • Physical impacts of disproportionate short stature included difficulty performing daily tasks and reaching for objects

  • Social and emotional impacts included poor self-esteem,

bullying, or difficulty fitting in with peers

Comorbidity Event Rates in HCH

1Mukherjee S et al. Medical Impact of HCH among children and adults in England: a 22 yr retrospective study (poster). 2Galetaki

D et al. Horm Res Paediatr 2025; 17:1-9. 3Aldegheri R et al. J Pediatr Orthop B 2001; 10; 238-47

ER reported as the number of unique comorbidity episodes per 100 PY. Only the first occurring event within a 30-day period was counted to avoid over-counting events from the same episode

Reddy et al., ICCBH 2024 (Poster P176)

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Skeletal Conditions

VOXZOGO Improves Spinal Morphology in Young Children with ACH, as Compared to Placebo Control

Positive effect of VOXZOGO on spinal measurements at week 52 compared with placebo

Positive effect of VOXZOGO on curvature of spine at week 52 compared with placebo

(mm)

(mm)

(degrees)

• VOXZOGO is the only approved therapy with data showing a positive impact on spinal morphology

• Spinal morphology is one of the factors that can lead to spinal stenosis, a leading cause of morbidity in

achondroplasia

The LSM differences are reported in millimeters for the VOXZOGO vs placebo ANCOVA-adjusted treatment groups from baseline to week 52. aTreatment difference in LSM change was

calculated as VOXZOGO − placebo. ANCOVA, analysis of covariance; CI, confidence interval; IPD, interpedicular distance; L1-L5, lumbar vertebrae 1-5; LSM, least squares mean.

Age cohorts represent age at treatment initiation. The LSM differences are reported in degrees for the VOXZOGO vs placebo ANCOVA-adjusted treatment groups from baseline to week 52. aTreatment difference in LSM change was calculated as VOXZOGO − placebo. bVOXZOGO, n = 11; placebo, n = 7. cVOXZOGO, n = 10; placebo, n = 7. dVOXZOGO, n = 18; placebo, n = 13. TLK; thoracolumbar kyphosis.

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Skeletal Conditions

BMN 333: Phase 2/3 Designed to Target Superior Profile

BMN 333 for achondroplasia: Targeting initiation of registration-enabling Ph 2/3 study, expected to begin 1H'26

Biological Rationale for

Potential Clinical Benefit

• Preclinical data: Suggest free CNP levels reached by BMN 333 may drive additional growth

• Human genetics: Pathway overexpression can drive

  significant growth without unexpected safety challenges

• Clinical data: Long-acting agents have not explored these higher exposures (BMN 333 aims to extend the dose-response curve)

Phase 2/3 Registration-

enabling Study

• Four-arm, Phase 2 dose-ranging study planned to include three dose levels of BMN 333 vs. VOXZOGO

• Followed by Phase 3 comparison of BMN 333 against active control (VOXZOGO), assessing safety, growth, and functional outcomes

BMN 333 Designed for

Superiority vs. VOXZOGO

• Aligned with regulators to pursue

  superiority for BMN 333

• Potential to set a new standard of care for the treatment of achondroplasia

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Anticipated Key Pipeline Highlights over Coming Quarters

2H 2025

1H 2026

2H 2026

Pipeline

Enzyme Therapies

Skeletal

Conditions

BMN 351 for DMD1

Clinical update (6 & 9 mg/kg cohorts)

PALYNZIQ adolescents (12-17 y/o)

U.S. and EU submissions

BMN 333 Ph1 PK data

BMN 333 Ph1 PK presentation BMN 333 Ph2/3 registration-enabling study initiation VOXZOGO for HCH Ph3 data readout		VOXZOGO for HCH health authority submissions for approval
	PALYNZIQ adolescents U.S. and EU launch
BMN 401 ENERGY 3 (1-12 y/o) data readout BMN 349 for AATD2 Phase 2 (POC) study initiation		BMN 401 ENERGY 3 U.S. and EU submissions

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1DMD = Duchenne muscular dystrophy; 2AATD = alpha-1 antitrypsin deficiency

Q&A

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Appendix

Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information

Reconciliation of GAAP Reported Net Income to Non-GAAP Income(1)

1. Certain amounts may not sum or recalculate due to rounding.

2. These amounts were included in SG&A and represent severance costs incurred in the acquisition of Inozyme in July 2025.

3. Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets.

4. These amounts were included in SG&A and represent severance and restructuring costs related to the Company's 2024 corporate initiatives and the associated organizational redesign efforts.

5. Represents impairment loss on non-marketable equity securities recorded in Other income (expense), net.

22

Reconciliation of GAAP and Non-GAAP R&D and SG&A Expenses (1)

1. Certain amounts may not sum or recalculate due to rounding.

2. These amounts were included in SG&A and represent severance costs incurred in the acquisition of Inozyme in July 2025.

(4) These amounts were included in SG&A and represent severance and restructuring costs related to the Company's 2024 corporate initiatives and the associated organizational redesign efforts.

23

Reconciliation of GAAP Income from Operations to Non-GAAP Income from Operations(1)

1. Certain amounts may not sum or recalculate due to rounding.

2. These amounts were included in SG&A and represent severance costs incurred in the acquisition of Inozyme in July 2025.

3. Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets.

4. These amounts were included in SG&A and represent severance and restructuring costs related to the Company's 2024 corporate initiatives and the associated organizational redesign efforts.

24

Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS(1)

1. Certain amounts may not sum or recalculate due to rounding.

2. These amounts were included in SG&A and represent severance costs incurred in the acquisition of Inozyme in July 2025.

3. Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets.

4. These amounts were included in SG&A and represent severance and restructuring costs related to the Company's 2024 corporate initiatives and the associated organizational redesign efforts.

5. Represents impairment loss on non-marketable equity securities recorded in Other income (expense), net.

6. Reconciliation in slide 26.

   25
   
   

Reconciliation of GAAP Weighted Average Diluted Shares Outstanding to Non-GAAP Weighted Average Diluted Shares Outstanding(1)

26

1. Certain amounts may not sum or recalculate due to rounding.