BioMarin Pharmaceutical : Third Quarter 2025 Presentation

Third Quarter 2025 Earnings

October 27, 2025

Q3'25 Earnings Agenda:

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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

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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

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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

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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

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    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

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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

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assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.

Q3'25 Commercial Update

Cristin Hubbard Executive Vice President, Chief Commercial Officer

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