Qfitlia approved by FDA to treat haemophilia A or B
Ella Day | March 31, 2025 | News story | Research and Development | FDA, Haematology, Sanofi, haemophilia, pharma
The US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran) as the first therapy in the US to treat haemophilia A or B, with or without inhibitors.
The approval of the antithrombin-lowering (AT) drug was based on data from the ATLAS phase 3 studies that Sanofi conducted. Sanofi is an international pharma and healthcare company, providing vaccination and treatment services. The studies demonstrated meaningful bleed protection as measured by annualised bleeding rates (ABR) across haemophilia patients with or without inhibitors.
Haemophilia A and B are rare, congenital and lifelong bleeding disorders. Both impair the ability of a person’s blood to clot, leading to excessive bleeds and spontaneous bleeds into joints. These can result in joint damage and chronic pain. Haemophilia A and B are caused by a deficiency of factor VIII and IX, respectively, resulting in insufficient thrombin generation and ineffective clot formation.
Qfitlia prevents or reduces the frequency of bleeding episodes. By lowering AT (a protein that inhibits blood clotting), it increases thrombin generation to restore haemostasis in haemophilia patients. It offers consistent protection with as few as six injections a year through a pre-filled pen, vial or syringe.
Brian Foard, executive vice president, head of speciality care at Sanofi, stated: “Qfitlia has the potential to meaningfully change the haemophilia landscape through effective bleed protection, infrequent dosing and simplified administration.”
Guy Young, director of Hemostasis and Thrombosis center at Children’s Hospital, Los Angeles, US, commented: “Qfitlia delivers the fewest doses of any prophylactic therapy in haemophilia, and its unique mechanism allows it to be used to treat all types of haemophilia, including with inhibitors and haemophilia B, where unmet medical needs remain.”
Ella Day
31/3/25
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