Press Release: Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors

Core Perspective - Qfitlia (fitusiran) has been approved by the US FDA as the first antithrombin-lowering therapy for the routine prophylaxis of hemophilia A and B, providing a significant advancement in treatment options for patients with or without inhibitors [1][10]. Company Insights - Sanofi emphasizes its commitment to innovation in the treatment of rare blood disorders, highlighting Qfitlia's potential to transform hemophilia care through effective bleed protection and reduced treatment burden [3]. - The company is launching HemAssist alongside Qfitlia to offer comprehensive patient support services, including insurance assistance and educational resources [9]. Industry Impact - Qfitlia's unique mechanism allows for the treatment of all types of hemophilia, including those with inhibitors, addressing significant unmet medical needs in the market [4]. - The ATLAS clinical development program has shown that Qfitlia can achieve a significant reduction in annualized bleeding rates (ABR) by 71% for patients without inhibitors and 73% for patients with inhibitors compared to traditional on-demand treatments [7][12]. Clinical Data - In the ATLAS studies, Qfitlia demonstrated low bleed rates with as few as six injections per year, with nearly half of patients experiencing one or fewer bleeds during the open-label extension study [7][12]. - The FDA has also approved the Siemens Healthineers' INNOVANCE Antithrombin assay as a companion diagnostic for Qfitlia, allowing for AT level measurement at no cost to patients [8].