Amicus Therapeutics is a biotechnology company focused on developing and commercializing therapies for rare and orphan diseases, primarily monetizing through high-value specialty drug sales. Its economic engine depends on securing regulatory approvals, protecting intellectual property, and driving adoption among a concentrated base of specialists and treatment centers. The moat, where it exists, comes from patent protection, regulatory exclusivity, and deep expertise in niche disease pathways that limit direct competition. Cash generation is tied to maintaining premium pricing power in small patient populations while managing R&D intensity and commercialization costs.