血友病创新疗法芬妥司兰钠注射液获批，一年最少6针皮下注射

Core Viewpoint - Sanofi's innovative siRNA therapy, Fintorisan Sodium Injection, has received approval from the National Medical Products Administration (NMPA) in China for the treatment of severe hemophilia A and B in patients aged 12 and above, marking a significant advancement in hemophilia treatment options [1][2][3] Group 1: Product Approval and Indications - Fintorisan Sodium Injection is the world's first and only siRNA therapy for hemophilia, approved for routine prophylactic treatment to prevent bleeding in patients with or without inhibitors [1] - The therapy is specifically indicated for severe hemophilia A patients with or without factor VIII inhibitors and severe hemophilia B patients with or without factor IX inhibitors [1] Group 2: Patient Demographics and Treatment Challenges - Hemophilia is one of the first diseases included in China's rare disease directory, with approximately 40,000 registered patients in the country [1] - Patients face significant risks of spontaneous or excessive bleeding due to a congenital lack of specific clotting factors, which can lead to severe complications if not properly managed [1] Group 3: Clinical Research and Efficacy - The approval of Fintorisan was primarily based on data from the ATLAS Phase III clinical trial, which demonstrated significant efficacy in reducing annualized bleeding rates (ABR) by 73% for patients with inhibitors and 71% for those without inhibitors [2] - The therapy offers effective bleeding protection across a wide range of hemophilia patients and has shown good safety profiles [2] Group 4: Treatment Paradigm Shift - The introduction of Fintorisan represents a new era in hemophilia treatment, moving towards non-factor therapies that enhance treatment convenience and adherence [3] - The therapy requires a minimum of six subcutaneous injections per year, significantly reducing the burden of frequent intravenous infusions associated with traditional factor replacement therapies [3] - This innovation fills a critical treatment gap for patients with inhibitors and provides a more efficient and sustainable treatment option for all hemophilia patients, making regular treatment more accessible [3]