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

Sanofi US News

Qfitlia (fitusiran), the first antithrombin-lowering therapy in hemophilia, offers consistent protection with as few as six injections a year via a prefilled pen or vial and syringe, according to Sanofi.

Paris, March 28, 2025. The US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors. The approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors.

By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in people with hemophilia. Qfitlia uses small-interfering RNA technology, which enables low treatment frequency, subcutaneous dosing, and low volume injections.

Coalition for Hemophilia B CEO Kim Phelan stated, “This marks another meaningful step forward for the hemophilia community. With the FDA approval of Qfitlia (fitusiran), patients with hemophilia A or B with or without inhibitors now have access to a novel antithrombin-lowering therapy designed to reduce bleeding episodes and offer more flexibility in treatment. We’ve come a long way in advancing options for people with hemophilia, and this represents another opportunity to expand personalized care and improve quality of life. We look forward to learning more and supporting our community as this treatment becomes available.”