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BioMarin Pharmaceutical
BioMarin Pharmaceutical (BMRN) investor relations material
BioMarin Pharmaceutical 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.44th Annual J.P. Morgan Healthcare Conference summary12 Jan, 2026
Strategic transformation and growth
Achieved a 15% CAGR from FY'23 to FY'25, with preliminary FY'25 revenue at $3.2 billion and Voxzogo revenue at $920 million, driven by innovation and business unit focus.
Completed two M&A deals in 2025, including the $4.8 billion Amicus acquisition, to supplement internal innovation and diversify revenue.
Increased profitability by over 3x compared to topline growth in FY'24 and generated $728 million operating cash flow Q3'25 YTD.
Targeting sustained double-digit CAGR into the 2030s, leveraging new assets and pipeline advancements.
Cost transformation identified $500 million in savings, improving profitability and cash flow.
Pipeline and innovation highlights
Prioritized pipeline for transformative potential, with multiple data readouts and regulatory filings expected in the next 12 months.
Announced filing for full approval of Voxzogo in achondroplasia, with post-marketing requirements completed and two label expansions expected.
BMN 333 demonstrated over 13-fold increase in free CNP AUC; phase 2/3 study to initiate in 1H'26 for achondroplasia.
BMN 401 pivotal data in ENPP1 deficiency expected 1H'26, with potential to address 2,000–2,500 patients.
PALYNZIQ label expansion for adolescents (12–17 y/o) anticipated in 2026, with significant reduction in blood Phe and increased protein intake.
Amicus acquisition and integration
Amicus acquisition brings Galafold (Fabry disease) and Pombiliti/Opfolda (Pompe disease), expanding the enzyme therapy portfolio.
Galafold to be expanded from 40 to 80 countries, aiming to increase diagnosis, treatment, and patient reach.
Pombiliti available in 15 countries, with opportunities for label expansion and increased global access.
Pombiliti + Opfolda and Galafold, acquired via Amicus, represent $1 billion peak revenue opportunities each, with global expansion plans.
Confident in achieving accretive financial impact within 12 months post-closure and substantial accretion by 2027.
BMN 351: What functional data is key for market?
VOXZOGO IP: ITC decision impact and timeline?
Galafold: How to boost diagnosis/treatment rates?
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Next BioMarin Pharmaceutical earnings date
Q4 202518 Feb, 2026
BioMarin Pharmaceutical
Next BioMarin Pharmaceutical earnings date
Q4 202518 Feb, 2026
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Frequently asked questions
BioMarin Pharmaceutical Inc is a biotechnology company focused on developing and commercializing therapies for rare genetic diseases. Its research and product development efforts target conditions with limited treatment options, primarily in the fields of enzyme replacement and gene therapy. The company serves healthcare providers and patients worldwide through approved treatments and scientific initiatives. The company is headquartered in San Rafael, California, and its shares are listed on the NASDAQ.
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