BioMarin Pharmaceutical Inc. (BMRN) reported that its growth drug VOXZOGO is on track to generate between $1.0 billion and $1.05 billion in full-year 2026 revenue, positioning the CNP analog as the company's first blockbuster product. The figures were discussed Thursday at the Citi Biopharma Back to School Summit 2026.
Chief Commercial Officer Cristin Hubbard said four products are tracking toward blockbuster status, with VOXZOGO leading the way. The company expects trailing twelve-month revenue of $3.4 billion and generates roughly $1 billion in annual free cash flow against a market capitalization of $12.6 billion.
VOXZOGO revenue grew 14% year over year, while global patient demand rose 20%. About 75% of VOXZOGO sales come from outside the United States. The company has retained 90% of its VOXZOGO patients through July despite competition — only 10% of U.S. patients have switched to Ascendis's YORVIPATH, which launched in February 2024.
BioMarin's commercial footprint spans 55 markets, reaching patients across an 80-country presence with nine commercial therapies across skeletal and metabolic disease units. The addressable global population for achondroplasia is estimated at 24,000; hypochondroplasia affects an estimated 14,000 people worldwide with a diagnosis rate of roughly 30%.
The company completed its FDA submission for VOXZOGO's hypochondroplasia indication in just 79 days from database lock, a pace Hubbard noted would place BioMarin in the top quartile based on McKinsey benchmarking. Full approval and anticipated launch for hypochondroplasia are expected in 2027. The approved label is expected to include upwards of 10 years of safety data and approximately 1,200 patient years of clinical-trial data.
At the Citi summit, Chief R&D Officer Greg Friberg outlined several pipeline milestones. BMN 333, a next-generation long-acting CNP analog, demonstrated a 13-fold increase in area under the curve for free CNP compared with VOXZOGO and 2-to-5-times higher cyclic GMP levels. Its half-life is approximately 100 hours versus less than one hour for VOXZOGO. A Phase 3 trial is powered to detect a 50% improvement in annualized growth velocity with 60 patients per arm; data is expected in 2027.
BMN 820 (also known as DMX-200), an oral CCR2 inhibitor for focal segmental glomerulosclerosis, is expected to report Phase 3 data measuring two-year proteinuria in 2028. ALE-01, a small-molecule ENPP1 inhibitor for hypophosphatasia, has Phase 1 data publishing planned for 2025, with Phase 2 development to follow. Friberg noted the molecule's promise extends beyond bone compartments to muscle and joints affecting adult patients.
BioMarin also shared peak-revenue expectations for assets acquired from Amicus. GALAFOLD, for Fabry disease, carries a peak revenue potential of $1.4 billion in the mid-2030s. Combined peak revenue for POMBILITI and OPFOLDA in Pompe disease is estimated at $1.2 billion in the mid-to-late 2030s, with both expected to be accretive to non-GAAP earnings per share in 2027.
Under the terms of a settlement with Ascendis, BioMarin receives a 20% royalty on U.S. net sales of YORVIPATH and an 18% royalty on sales in the EU, Brazil, and South Korea. BioMarin's P/E ratio was cited at 172; InvestingPro rates the company's overall financial health as "Good" and lists it among undervalued opportunities.
