BioMarin is a rare disease biotech that develops and sells therapies for patients with genetically defined conditions. Its commercial portfolio spans eight products sold across roughly 80 countries, organized into two business units. The Enzyme Therapies unit is the larger segment, consisting of enzyme replacement therapies (ERTs) for rare lysosomal storage and metabolic disorders including MPS IVA, MPS VI, MPS I, PKU, and Batten disease. The Skeletal Conditions unit is anchored by VOXZOGO, a once-daily injectable approved for achondroplasia in children, and is BioMarin's fastest-growing product with roughly 75% of its revenues coming from outside the U.S. BioMarin sells directly to hospitals, specialty pharmacies, and government agencies in key markets, and through distributors elsewhere. ALDURAZYME is an exception: BioMarin manufactures it and supplies it exclusively to Sanofi, which handles all distribution and marketing globally, and BioMarin receives a share of worldwide net sales. BioMarin's model depends on finding and starting new patients, then maintaining them on chronic therapy — adherence is high given the severity of the underlying conditions. Growth levers include geographic expansion, indication expansion for existing drugs, and M&A. In late 2025, BioMarin agreed to acquire Amicus Therapeutics for ~$4.8B, adding two commercial rare disease products — Galafold for Fabry disease and Pombiliti+Opfolda for Pompe disease — that BioMarin expects to fold into its existing Enzyme Therapies unit and global commercial infrastructure.