FDA Approves New Therapy for Hemophilia A and B
April 02, 2025
The Food and Drug Administration recently
approved fitusiran (Qfitlia, Sanofi) for routine prophylaxis to prevent or reduce bleeding episodes in patients 12 years and older with hemophilia A or B, with or without factor VIII or IX inhibitors.
Unlike traditional treatments that replace missing clotting factors, fitusiran reduces antithrombin levels to increase thrombin. It is administered subcutaneously once every two months, with dosing guided by the FDA-cleared Innovance antithrombin assay. Clinical trials demonstrated that fitusiran significantly reduced annualized bleeding rates in patients both with and without inhibitors.
Fitusiran carries a boxed warning for thrombotic events and gallbladder disease and requires liver function monitoring. FDA granted fitusiran
orphan drug and
fast track designations.
