Amicus Therapeutics is a rare disease biotech with two marketed therapies targeting lysosomal storage disorders. Galafold (migalastat) is an oral precision medicine for Fabry disease, a rare genetic disorder causing progressive damage to the heart, kidneys, and nervous system. Galafold works by stabilizing a patient's own defective enzyme, but only works in patients with genetically amenable mutations — roughly 35-50% of Fabry patients. Galafold is approved in over 40 countries and holds roughly 69% global market share among amenable patients. Pombiliti + Opfolda is a two-component enzyme replacement therapy for late-onset Pompe disease, a disorder that causes progressive muscle weakness. The combination is designed to deliver more active enzyme to disease-relevant tissues than prior therapies. Approved in the U.S., EU, and several other markets in 2023, it is in active global launch with reimbursement secured in 15 countries. Both products are sold directly to hospitals and specialty treatment centers, targeting rare disease physicians. Revenue is driven by patient volume — patients generally stay on therapy for life — premium pricing typical of orphan drugs, and geographic expansion as new markets secure reimbursement. Roughly 58-60% of revenue comes from outside the U.S. Amicus pays mid-teen royalties to GSK on Galafold sales in major ex-U.S. markets. Beyond its two marketed products, Amicus has licensed U.S. rights to DMX-200, an oral therapy in Phase 3 for FSGS, a rare kidney disease. Amicus is pending acquisition by BioMarin, announced in December 2025 and expected to close in Q2 2026.