BioMarin’s Growth, Pipeline and Amicus Integration Highlighted at Wells Fargo Conference

At the Wells Fargo 21st Annual Healthcare Conference in September 2026, BioMarin Pharmaceutical Inc. (BMRN) detailed its financial trajectory, therapeutic pipeline, and strategic integration of Amicus Therapeutics, a move that could bolster its metabolic conditions business. The company’s metabolic segment grew at a 9% compound annual growth rate (CAGR) through 2023, reaching $2.1 billion in full-year revenue, with two of its top products—NAGLAZYME and VIMIZIM—generating $500 million and $800 million, respectively, in 2023 alone. VOXZOGO (vosoritide), launched for achondroplasia, posted 14% year-over-year revenue growth and 20% patient growth in its latest quarter, with global sales guidance raised to $1 billion at the midpoint. The drug’s revenue is 75% outside the U.S., reflecting its broad market appeal. BioMarin also acquired Amicus Therapeutics, which holds assets with projected peak revenues of $1.4 billion for GALAFOLD and Fambril (Fabry disease) and $1.2 billion for POMBILITI and OPFOLDA (Pompe disease) by mid-to-late 2030s. The deal included a 20% U.S. and 18% EU/Brazil/South Korea retroactive royalty structure from Ascendis Therapeutics, a key partner in Fabry disease therapies. Meanwhile, BioMarin’s clinical pipeline remains robust. BMN 333, a next-generation CNP therapy for achondroplasia, advanced to Phase 2 with data expected in 2024, targeting a 50% improvement in annualized growth velocity—equivalent to an extra 0.75 cm/year—in a trial powered for 90% statistical significance. Hypochondroplasia data published in NEJM Evidence supported its efficacy across FGFR3 mutations, with FDA review pending. BMN 351, a Duchenne muscular dystrophy therapy, is advancing to higher-dose 12 mg/kg data in 2024, while an oral hypophosphatasia (HPP) therapy is in Phase 1, with results due in 2025. The company also completed Phase 3 enrollment for BMN 820 (formerly DMX-200), a CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS), with data readout expected in 2028. With a P/E ratio of 171, BioMarin’s stock reflects its high valuation amid its expanding pipeline and strategic acquisitions. The company operates in 80 countries, commercializing VOXZOGO in 55 markets, and Amicus’s Fabry and Pompe assets are expanding into additional geographies, underscoring its ambition to dominate metabolic disease treatments.