BioMarin Pharmaceutical Inc.

What they do

BioMarin discovers, develops and commercializes medicines for genetically defined conditions, with eight commercial therapies as of the 2025 10-K and nine as of the second quarter of 2026 after adding GALAFOLD and POMBILITI + OPFOLDA through the Amicus acquisition. Products span skeletal conditions such as VOXZOGO for achondroplasia, metabolic conditions including ALDURAZYME, BRINEURA, NAGLAZYME and VIMIZIM, and PKU therapies KUVAN and PALYNZIQ. The company is based in San Rafael, California, was founded in 1997, and reported $3.2 billion in total revenues for 2025.

Revenue drivers

• VOXZOGO (vosoritide) — Achondroplasia therapy that management says drove a significant portion of 2025 growth; second quarter 2026 guidance was raised to at least $1 billion of full-year 2026 revenue.
• Amicus-acquired products (GALAFOLD and POMBILITI + OPFOLDA) — Added on April 27, 2026 for Fabry disease and late-onset Pompe disease; the company expects these to accelerate revenue growth and non-GAAP EPS accretion into the mid-2030s.
• Metabolic conditions portfolio — Includes ALDURAZYME (MPS I), BRINEURA (CLN2), NAGLAZYME (MPS VI), VIMIZIM (MPS IVA) and the PKU therapies KUVAN and PALYNZIQ.
• Total company revenues — Revenue grew from $1.85 billion in 2021 to $3.22 billion in 2025; second quarter 2026 revenue was $989.7 million, up 20% from $825.4 million a year earlier.

Recent performance

Second quarter 2026 total revenues were $989.7 million, up 20% from $825.4 million in the second quarter of 2025, with first half 2026 revenues of $1,755.9 million versus $1,570.6 million. Second quarter net income fell to $44.8 million from $240.5 million a year earlier, and first half net income was $150.3 million versus $426.2 million, reflecting acquisition-related costs. Second quarter SG&A rose to $395.5 million from $232.3 million, intangible asset amortization rose to $73.5 million from $4.8 million and interest expense rose to $63.3 million from $2.7 million. Full-year 2025 revenue was $3.22 billion with net income of $348.9 million, down from $426.9 million in 2024, while operating cash flow rose to $828.0 million from $572.8 million.

Strategy

BioMarin completed the acquisition of Amicus Therapeutics on April 27, 2026, adding two commercial therapies for Fabry disease and late-onset Pompe disease and stating it expects cost synergies and operating cash flow growth into the mid-2030s. It is expanding VOXZOGO beyond achondroplasia, with an sNDA submitted for hypochondroplasia and an FDA-accepted sNDA for full approval in achondroplasia with a PDUFA date of February 28, 2027. The company added BMN 820, a first-in-class oral CCR2 inhibitor for FSGS, with exclusive U.S. commercialization rights and an addressable U.S. population of about 30,000. Earlier pipeline expansion included the July 2025 acquisition of Inozyme, adding BMN 401 for ENPP1 deficiency. In December 2025 the company committed to voluntarily withdraw ROCTAVIAN from the market and recorded approximately $240.0 million of restructuring charges in 2025.

Risks

• Integration of Amicus — BioMarin closed the Amicus acquisition in April 2026 and must integrate an expanded commercial portfolio and operations, and the 10-K risk factors state acquisitions may divert management attention or fail to achieve anticipated benefits.
• Increased leverage and interest expense — Long-term debt was $3.53 billion at June 30, 2026 and quarterly interest expense rose to $63.3 million from $2.7 million, related to financing including $850.0 million of 5.5% senior unsecured notes due 2034 issued in February 2026.
• Concentration in small patient populations — The 10-K risk factors note that target patient populations for the products are relatively small, requiring significant commercial effort to generate sufficient sales.
• Reimbursement and payer coverage — The 10-K states that failing to obtain and maintain adequate coverage and reimbursement from third-party payers would adversely affect product sales or leave no commercially viable market.

Outlook

Management raised full-year 2026 guidance for total revenues, VOXZOGO and non-GAAP diluted EPS, with VOXZOGO expected to contribute at least $1 billion in 2026. It expects the addition of GALAFOLD and POMBILITI + OPFOLDA, with cost synergies, to accelerate revenue growth, non-GAAP diluted EPS accretion, non-GAAP operating margin expansion and operating cash flow through the mid-2030s. If approved for hypochondroplasia, VOXZOGO would be the first targeted therapy for that indication with a potential 2027 launch; the company plans to update on the application status at its third quarter earnings.