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Rare Disease Specialist — Growing Into a Diversified Enzyme Therapy Powerhouse

BioMarin Pharmaceutical is a San Rafael, California-based global biotechnology company singularly focused on developing and commercializing therapies for rare and ultra-rare genetic diseases.  These are mostly conditions that often affect small patient populations, frequently have no existing treatment options, and disproportionately impact children. 

Founded in 1997, BioMarin built its reputation by pioneering enzyme replacement therapies (ERTs) for lysosomal storage disorders, a class of inherited metabolic diseases caused by deficient or dysfunctional enzymes that leads to toxic accumulation of substrates in cells and progressive organ damage. Today the company markets six approved products across two commercial platforms.  BioMarin operates in over 70 countries, employs approximately 3,500 people, and generates roughly 60% of its revenue from markets outside the United States, reflecting the global patient base for the rare diseases it treats. The company's strategic identity is built around three pillars: scientific leadership in rare disease biology, deep regulatory and market access expertise in ultra-rare indications, and a commitment to reaching every eligible patient globally.

In December 2025, BioMarin announced a definitive agreement to acquire Amicus Therapeutics for approximately $3.9 billion. The transaction, financed with approximately $3.7 billion in non-convertible debt secured at favorable terms, is expected to close in Q2 2026, subject to Amicus stockholder approval and regulatory clearances. This is the most significant strategic move in BioMarin's history and directly addresses the company's need to diversify its enzyme therapy revenue base while adding two high-growth commercial products with long runway:

• Galafold (migalastat): An oral chaperone therapy for Fabry disease in patients with amenable mutations. The only oral treatment for Fabry disease; already generating meaningful revenue with significant remaining market penetration potential globally.
• Pombiliti + Opfolda (cipaglucosidase alfa + miglustat): Next-generation two-component ERT for Pompe disease (late-onset). Shown to be superior to the incumbent standard of care (alglucosidase alfa) in clinical trials. Adolescent label expansion expected in H2 2026, significantly expanding the treatable population.

Both products fit directly into BioMarin's existing enzyme therapy commercial infrastructure and rare disease go-to-market model, creating immediate synergy potential. The combined entity is expected to accelerate revenue growth and profitability well into the next decade.