September 5, 2024 (U.S. time), Gensciences received FDA orphan drug designation for its next-generation ultra-long-acting recombinant coagulation FVIII - Pegylated Recombinant Human Coagulation Factor Ⅷ-Fc Fusion Protein, intended for the treatment of hemophilia A. FRSW117 is the first recombinant FVIII product in China to be granted FDA ODD, fully demonstrating the FDA’s recognition of its unique molecular design and its superior performance in the clinic.

Orphan drugs, also known as rare disease drugs, are those used for the prevention, treatment, and diagnosis of rare diseases. The FDA orphan drug designation will help FRSW117 enjoy some policy support in the U.S. for its future R&D, registration and commercialization, including but not limited to: (1) tax credits for clinical trial costs; (2) exemption of new drug application fees; (3) seven-year market exclusivity and freedom from patent nuisance.