BioMarin Pharmaceutical Inc. — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total revenues for the third quarter of 2026 were $1,025 million, a 32% increase year-over-year (YoY) compared to $776 million in 2025.
• Net product revenues were $1,013 million; royalty and other revenues were $11.6 million.
• GAAP net loss was $123 million, an increase from a $31 million loss in the same period in 2025.
• Non-GAAP income was $45 million, an increase from $22 million in the same period in 2025.
• GAAP operating margin was (8.0)%, compared to (6.0)% in 2025.
• Non-GAAP operating margin was 13.4%, compared to 2.8% in 2025.
• GAAP diluted EPS was $(0.63), compared to $(0.16) in 2025.
• Non-GAAP diluted EPS was $0.23, compared to $0.12 in 2025.
• For the nine months ended September 30, 2026, total revenues were $2,781 million (18% YoY growth), GAAP net income was $27 million (down from $395 million), and Non-GAAP income was $430 million (down from $525 million).
• Cash and cash equivalents were $736 million as of September 30, 2026, down from $1,312 million as of December 31, 2025.
• Total debt increased to $4.17 billion ($659 million current portion + $3.51 billion long-term) as of September 30, 2026, compared to $597 million long-term debt as of December 31, 2025.
• Net cash used in investing activities for the nine months ended September 30, 2026, was $4.68 billion, primarily driven by the business combination with Amicus Therapeutics ($5.07 billion).
• Net cash provided by financing activities for the nine months ended September 30, 2026, was $3.53 billion, driven by $3.65 billion in proceeds from borrowings.

Guidance and Future Outlook:

• Updated full-year 2026 Total Revenue guidance to $3,800 million to $3,900 million (previously $3,875 million to $3,925 million).
• Updated full-year 2026 Metabolic Conditions revenue guidance to $2,675 million to $2,750 million (previously $2,725 million to $2,775 million).
• Updated full-year 2026 VOXZOGO revenue guidance to $975 million to $1,025 million (previously $1,000 million to $1,050 million).
• Updated full-year 2026 Other Revenues guidance to $125 million to $150 million (previously $100 million to $125 million).
• Updated full-year 2026 Non-GAAP Diluted EPS guidance to $2.80 to $3.10 (previously $4.90 to $5.10).
• Guidance updates reflect order timing uncertainty from a single international customer and the impact of acquired in-process research and development (IPR&D) charges.
• The Alesta acquisition and BMN 450 license agreement are expected to result in total pre-tax charges of approximately $320 million and $83 million, respectively, for full-year 2026.
• These charges represent an unfavorable impact of approximately $1.55 and $0.32, respectively, to both GAAP and Non-GAAP Diluted EPS for full-year 2026.
• BioMarin expects approximately $280 million of GAAP cost synergies (approximately $220 million on a Non-GAAP basis) from the Amicus acquisition to be fully realized in 2028.

Business Segments and Product Lines:

• Metabolic Conditions revenue grew 44% YoY in Q3 2026 to $744 million, driven by GALAFOLD ($152 million), POMBILITI + OPFOLDA ($49 million), and VIMIZIM ($216 million).
• VOXZOGO revenue grew 12% YoY in Q3 2026 to $245 million; more than 75% of revenue was generated outside the U.S.
• GALAFOLD and POMBILITI + OPFOLDA contributed $152 million and $49 million, respectively, in Q3 2026, their first full quarter as part of BioMarin.
• The number of patients treated with GALAFOLD increased 13% YoY, and POMBILITI + OPFOLDA increased 69% YoY.
• VIMIZIM revenue grew 18% YoY to $216 million in Q3 2026.
• NAGLAZYME revenue decreased 11% YoY to $108 million due to timing of large international orders.
• PALYNZIQ revenue grew 2% YoY to $111 million.
• BRINEURA revenue grew 10% YoY to $53 million.
• ALDURAZYME revenue grew 6% YoY to $57 million.
• KUVAN revenue decreased 4% YoY to $23 million.
• ROCTAVIAN revenue was $1 million (down 67% YoY); the company announced it will no longer market ROCTAVIAN in 2026.
• Four new assets were added to the pipeline in 2026:
  • BMN 450 (HM15421/GC1134A): Investigational long-acting subcutaneous ERT for Fabry disease in global Phase 1/2.
  • BMN 440 (ALE1): Potential first-in-class oral therapy for hypophosphatasia (HPP) in Phase 1/2a; data expected in 2027.
  • BMN 430: Potential first-in-disease antisense oligonucleotide for ReNU syndrome in preclinical studies.
  • BMN 820 (DMX-200): First-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS); Phase 3 ACTION 3 trial fully enrolled with pivotal data expected in 2028.
• FDA PDUFA target action date set for May 30, 2027, for VOXZOGO expansion to treat hypochondroplasia (HCH).
• EMA validated submission for VOXZOGO to treat HCH.
• FDA is reviewing full approval of VOXZOGO for children with achondroplasia; PDUFA target action date is February 28, 2027.
• Phase 2/3 study for BMN 333 (long-acting CNP for achondroplasia) is enrolling on track; dose-response data expected in 2027.
• Five-year results for POMBILITI + OPFOLDA in late-onset Pompe disease showed durable motor function outcomes and relative stabilization of pulmonary function.
• Program update for BMN 351 (Phase 1/2 candidate for Duchenne muscular dystrophy) expected by year-end.

Market and Competitive Landscape:

• POMBILITI + OPFOLDA uptake benefited from supply constraints for other Pompe therapies.
• Children with achondroplasia in 58 countries are being treated with VOXZOGO.
• In the U.S., the majority of new VOXZOGO patient starts were in children under the age of two.
• BioMarin is building a diversified portfolio following the acquisition of Amicus Therapeutics earlier in 2026.

Risks and Challenges:

• Order timing uncertainty from a single international customer impacted Q3 2026 revenue and full-year guidance.
• Significant acquired IPR&D charges from the Alesta acquisition and BMN 450 license agreement negatively impacted earnings.
• Integration and restructuring costs related to the Amicus acquisition contributed to the GAAP net loss.
• High interest expense from debt issued to finance the Amicus transaction impacted results.
• Forward-looking statements involve risks including regulatory uncertainty, clinical trial results, manufacturing capabilities, and macroeconomic factors.

Management Commentary and Tone:

• Alexander Hardy, President and CEO, described the quarter as "strong" with revenue growth of 32%.
• Management noted that quarterly revenue exceeded $1 billion for the first time.
• Hardy highlighted the "more diversified portfolio" following the Amicus acquisition and increased global demand.
• Management expressed excitement about adding the newest asset (BMN 450) and building sustainable growth for the future.
• The tone regarding 2027 is positive, citing positioning for the next chapter of growth including the potential launch of VOXZOGO in hypochondroplasia.

Other Key Points:

• BioMarin acquired Amicus Therapeutics on April 27, 2026.
• On August 30, 2026, BioMarin announced a global settlement with Ascendis Pharma A/S regarding Yuviwel claims.
• Under the Ascendis settlement, Ascendis will pay BioMarin a royalty of 20% of net sales of Yuviwel in the U.S. and 18% in the EU, Brazil, and South Korea, retroactive to the first commercial sale, until May 2030.
• BioMarin will host a conference call and webcast on November 2, 2026, at 4:30 p.m. ET.
• The company does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP measures due to uncertainty regarding strategic portfolio reviews and potential asset impairments.