Amicus Therapeutics, Inc. Overview
Pro stress-test →Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. In 2024, Amicus Therapeutics' revenue was $528.30 million, an increase of 32.29% compared to the previous year's $399.36 million. The company is being acquired by BioMarin Pharmaceutical for $4.8 billion, with closure expected in Q2 2026.
Strategic Profile
Pro stress-test →The company's commercial products consist of Galafold, an orally administered monotherapy for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, a novel two-component treatment program for adults living with late-onset Pompe disease. The company expanded globally with Japan approval for Pompe therapy and plans to launch in 10 new European markets. Pipeline highlights include DMX-200's Phase 3 trial for FSGS and gene therapy advancements, supported by Teva's patent settlement ensuring Galafold's U.S. exclusivity until 2037.
Competitive Landscape
Pro stress-test →BioMarin, along with Sanofi, is a leader in the rare disease therapeutics market with an extensive portfolio of enzyme replacement therapies, including Palynziq, Vimizim, and Brineura. Amicus competes in rare metabolic diseases with a differentiated portfolio including the only approved oral precision medicine for amenable Fabry disease variants and emerging therapies for Pompe disease and other genetic conditions.
Industry Context
Amicus Therapeutics, Inc. operates in Biotechnology - Rare/Orphan Diseases, Lysosomal Storage Disorders.
Key facts
Founded: 2002 · Headquarters: Princeton, NJ · Employees: 499 · Revenue: $528M (2024) · Market cap: $0.7B (at $14.50/share acquisition price)