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.

FRSW117

FRSW117 is the world’s innovative, latest-generation ultra-long-acting recombinant coagulation factor VIII product that incorporates two technologies (Fc fusion and PEG technologies),enabling a “once-a-week” dosing frequency. The preventive treatment strategy with a “once-a-week” dosing frequency enables hemophilia A patients to return to normal life and achieve “functional cure”. Data from completed clinical phase II studies showed that FRSW117 has a good safety profile; the trough level of its drug activity was 3% at 7 days post dosing. It suggests that once-weekly administration of FRSW117 maintains FVIII activity at a high level, provides better bleeding protection, and reduces the annualized bleeding rate; it also significantly reduces the dosing frequency and the intravenous load. A clinical Phase III trial was initiated in December 2023 for this product in China, and in November 2023, and it obtained the implied approval for clinical trials from FDA.

Gensciences

Founded in 2019, Gensciences is a China-based, globally oriented innovative pharmaceutical company for hemophilia. Relying on our leading long-acting biotechnology platform, we focus on the unmet clinical needs of patients with hemophilia and continue to iteratively develop hemophilia drugs with Global Best-In-Class potential, aiming to help hemophilia patients live a normal life.