Slide 1: Second Quarter 2026 Financial Results
Second Quarter 2026 Financial Results August 6, 2026 1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expecta
Slide 3: Q2'26 Financial Results Agenda
Q2’26 Financial Results Agenda: 1 Q2’26 Results and Amicus Integration Update 2 Commercial Update 3 Research & Development Update 4 Financial Results 5 Q&A Alexander Hardy Cristin Hubbard Greg Friberg Brian Mueller Chief Executive Officer Chief Commercial Officer Chief R&D Officer Chief Financial Of
Slide 4: Q2'26 Key Business and Amicus Integration Updates
Q2’26 Key Business and Amicus Integration Updates Alexander Hardy Chief Executive Officer 4
Slide 5: Q2: Strengthened Rare Disease Leadership Accelerated Growth
Q2: Strengthened Rare Disease Leadership Accelerated Growth STRONG Q2’26 EXECUTION AMICUS VALUE CREATION PIPELINE MOMENTUM $990M ▲ 20%Y/Y ~$1.4B ~$1.2B Phase 3 Win GALAFOLD POMBILITI + OPFOLDA Hypochondroplasia (HCH) Potential to Expand Total Revenue Growth Peak Revenue Peak Revenue VOXZOGO Market ■
Slide 6: Accelerating BioMarin's Growth Trajectory through Mid-2030s
Accelerating BioMarin’s Growth Trajectory through Mid-2030s • Significant Cost Synergies Expected to be Fully Realized in 2028 • GALAFOLD and POMBILITI + OPFOLDA Peak Revenue • Expanding Combined Portfolio Non-GAAP Operating Margin • Generating Significant Operating Cash Flow 6
Slide 7: Accelerating the GALAFOLD Peak Revenue Outlook
Accelerating the GALAFOLD Peak Revenue Outlook Growth and Acceleration Levers ~10% CAGR ('27-'32) Peak Revenue Driving Diagnosis and Treatment Estimate • Globally, scale AI-enabled patient finding, genetic testing, newborn screening, and family cascade screening $1.4B • Goal to more than double numb
Slide 8: Accelerating the POMBILITI + OPFOLDA Peak Revenue Outlook
Accelerating the POMBILITI + OPFOLDA Peak Revenue Outlook Growth and Acceleration Levers ≥ 20% CAGR ('27-'32) Peak Revenue Driving Switch and Global Expansion Estimate • HCP and patient education; real-world-evidence generation to demonstrate the benefit of switching $1.2B • Leverage AI patient find
Slide 9: ~$220M in Amicus Cost Synergies Expected
~$220M in Amicus Cost Synergies1,2 Expected to Result in ~50% Reduction from Amicus-reported 2025 Non-GAAP Operating Expenses1 Cost Synergies Split Key Synergies Drivers • Significant reductions in R&D and G&A • S&M retained; maintaining HCP and patient- facing capabilities to drive commercial expan
Slide 10: Creating Significant Value Near-term and Beyond
Creating Significant Value Near-term and Beyond 2027 2028 Beyond 2030 Cost Synergies1,2 Cost Synergies Peak Revenue Outlook Ramping Fully Realized GALAFOLD ~$220M 2 $1.4B by mid-2030s (~10% CAGR ’27-’32) Annual run-rate cost reduction Gross Leverage1 POMBILITI + OPFOLDA < 2.5x ~50% Reduction from Am
Slide 11: Commercial Update
Commercial Update Cristin Hubbard Chief Commercial Officer 11
Slide 12: Acceleration of Galafold and Pombiliti + Opfolda Underway
Metabolic GROWTH Conditions Acceleration of Galafold and Pombiliti + Opfolda Underway Revenue Revenue $ in millions $ in millions + ~10% +16% + > 65% +62% $129 $111 $26 $16 1 1 Q2 2024A Q2 2025A Q2 2026A Q2 2024A Q2 2025A Q2 2026A FY 2024A: FY 2025A: FY 2024A: FY 2025A: $458M $522M $70M $113M Charts
Slide 13: Metabolic Conditions: More Diversified Portfolio Drove Strong Q2'26
Metabolic GROWTH Conditions Metabolic Conditions (Formerly Enzyme Therapies): More Diversified Portfolio Drove Strong Q2’26 Metabolic Conditions Q2’26 Revenue Q2’26 Dynamics $ in millions • 25% Y/Y revenue growth, inclusive of GALAFOLD and $695M, +25% Y/Y POMBILITI + OPFOLDA • Patients on therapy gr
Slide 14: Skeletal Conditions: Strong Demand in Q2'26 for VOXZOGO for ACH
Skeletal Conditions Skeletal Conditions: Strong Demand in Q2’26 for VOXZOGO for ACH Revenue Q2’26 Dynamics $ in millions • Double-digit U.S. and OUS revenue growth and strong 2H outlook led to increased FY’26 VOXZOGO guidance • Approximately three-quarters of VOXZOGO revenue generated OUS +14% • Chi
Slide 15: Research & Development Update
Research & Development Update Greg Friberg Chief R&D Officer 15
Slide 16: 2026 R&D Highlights: Recent Updates and Anticipated Milestones
2026 R&D Highlights: Recent Updates and Anticipated Milestones Program Status and Upcoming Milestones Recent Updates Completed in April – July 2026 Candidate Condition Key Highlights • sNDA submitted; subject to FDA review and VOXZOGO Phase 3 topline data for acceptance ✓ hypochondroplasia; sNDA sub
Slide 17: Potential Upside with BMN 820 for FSGS
Potential Upside with BMN 820 for Focal Segmental Glomerulosclerosis (FSGS); Phase 3 Endpoint Agreed with FDA FSGS Background and BMN 820 Opportunity BMN 820 Total Addressable Patient Population1 in the United States: ~30,000 FSGS Background • FSGS is a progressive kidney disease in which scarring o
Slide 18: Q2'26 Financial Results
Q2’26 Financial Results Brian Mueller Chief Financial Officer 18
Slide 19: Q2 Financial Highlights
Q2 Financial Highlights Q2 2026 Results Non-GAAP R&D and SG&A In millions, except percentages and per Y/Y • Y/Y increases reflect Amicus operating expenses, Q2’26 share amounts Change continued pipeline investment and commercial execution Total Revenues $990 +20% • GAAP SG&A includes ~$84M of transa
Slide 20: Updated Full-year 2026 Guidance
Updated Full-year 2026 Guidance Prior Guidance Updated Guidance Midpoint Growth (In millions, except per share amounts) As of May 4, 2026 As of August 6, 2026 (Y/Y) Total Revenues $3,825 to $3,925 $3,875 to $3,925 21% Metabolic Conditions $2,725 to $2,775 Unchanged 31% VOXZOGO $975 to $1,025 $1,
Slide 21: IOMARIN
IOMARIN B <x 08 S) ee ee
Slide 22: Appendix
Appendix Reconciliation of GAAP Reported to Selected Non-GAAP Adjusted Information
Slide 23: Reconciliation of GAAP Reported Net Income to Non-GAAP Income
Reconciliation of GAAP Reported Net Income to Non-GAAP Income(1) (In millions of U.S. dollars) Please refer to slide 27 for footnotes to the table 23
Slide 24: Reconciliation of GAAP and Non-GAAP COS, R&D, and SG&A Expenses
Reconciliation of GAAP and Non-GAAP COS, R&D, and SG&A Expenses(1) (In millions of U.S. dollars) Please refer to slide 27 for footnotes to the table 24
Slide 25: Reconciliation of GAAP Income from Operations to Non-GAAP Income from Operations
Reconciliation of GAAP Income from Operations to Non-GAAP Income from Operations(1) (In millions of U.S. dollars) Please refer to slide 27 for footnotes to the table 25
Slide 26: Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS
Reconciliation of GAAP Diluted EPS to Non-GAAP Diluted EPS(1) (In U.S. dollars) Please refer to slide 27 for footnotes to the table 26
Slide 27: Reconciliation of GAAP Weighted-Average Diluted Shares Outstanding
Reconciliation of GAAP Weighted-Average Diluted Shares Outstanding to Non-GAAP Weighted-Average Diluted Shares Outstanding(1) (In U.S. dollars) (1) Certain amounts may not sum or recalculate due to rounding. (2) Stock-based compensation expense recorded in SG&A for the three and six months ended Jun
Slide 28: Amicus Therapeutics, Inc. Reconciliation of Non-GAAP Financial Measures
Amicus Therapeutics, Inc.(1) Reconciliation of Non-GAAP Financial Measures (In thousands of U.S. dollars) (1) The above historical reconciliation is reproduced from Amicus’ earnings release furnished as Exhibit 99.1 to its Current Report on Form 8-K dated February 20, 2026 and reflects Amicus’ histo