AMICUS THERAPEUTICS Business Summary

Amicus Therapeutics is a global, commercial-stage biotechnology leader specializing in high-growth precision medicines for rare lysosomal storage disorders. The company generates revenue primarily through the direct sale and commercialization of its two flagship franchises: Galafold (migalastat), the first oral precision medicine for Fabry disease, and the Pombiliti + Opfolda combination therapy for late-onset Pompe disease. Operating as a fully integrated biopharmaceutical entity, Amicus utilizes a proprietary "chaperone-advanced" technology platform designed to stabilize enzymes and improve cellular uptake. Its business model is centered on the Global Metabolic segment, targeting high-value orphan drug markets with significant barriers to entry. In the competitive landscape, Amicus contends with Sanofi (Nexviazyme, Fabrazyme), Takeda (Replagal, Vpriv), and Chiesi Global Rare Diseases (Elfabrio). Amicus maintains a unique market position as the only provider of an oral alternative for Fabry patients with amenable mutations and a differentiated two-component therapeutic approach for Pompe disease that addresses the limitations of traditional monotherapy. The leadership team is anchored by President and CEO Bradley Campbell, who has spearheaded the company’s transition to sustained profitability since succeeding founder John F. Crowley in 2022. Crowley, whose high-profile journey to find a cure for his children’s Pompe disease served as the company’s founding catalyst, continues to provide strategic oversight as Executive Chairman. The executive suite is further strengthened by Chief Development Officer Dr. Jeff Castelli, the primary architect of the Pompe program, and Chief Financial Officer Simon Harford, who manages the company's disciplined capital allocation and global financial operations. The company’s strategic direction is supported by a board of directors featuring industry veterans such as Margaret McGlynn (formerly of Merck) and Michael Raab (CEO of Ardelyx). Amicus maintains a strong institutional investor base, with significant positions held by Perceptive Advisors and BlackRock, reflecting market confidence in its specialized rare disease pipeline and commercial execution.