On March 28, 2025, the US Food and Drug Administration (FDA) approved Qfitlia (Fitusiran) for the prevention and reduction of bleeding episodes in adults and pediatric patients aged 12 and older with hemophilia A or B. According to Tanya Wroblewski from the FDA, this new treatment offers a less frequent administration schedule compared to existing options, improving the lives of those with hemophilia.
Hemophilia A and B are genetic disorders resulting from deficiencies in coagulation factors VIII and IX, respectively, leading to prolonged bleeding. Qfitlia functions by reducing proteins that inhibit thrombin, an enzyme essential for blood clotting, and is administered subcutaneously once every two months. Its dosing is monitored using an FDA-cleared companion diagnostic test that checks antithrombin activity to minimize bleeding risks.
The approval was based on two multicenter randomized clinical trials involving 177 male participants. Results showed a significant reduction in the annual bleeding rate: a 73% decrease for those with inhibitors and a 71% decrease for those without, compared to traditional on-demand treatments.
Qfitlia has boxed warnings for potential thrombotic events, gallbladder disease, and liver toxicity, with common side effects including viral infections and cold symptoms. The FDA granted Qfitlia Orphan Drug and fast track status, and it is marketed by Sanofi.
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