fitusiran

Fitusiran is a first-in-class, antithrombin-directed small interfering RNA (siRNA) therapeutic indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors. It works by silencing the gene responsible for antithrombin (AT), a liver-generated protein that inhibits blood clotting. By reducing AT levels through RNA interference, fitusiran enhances thrombin generation and restores hemostasis in people with hemophilia. Fitusiran is administered subcutaneously and was approved for medical use in the United States in March 2025[1][3][4][5][6][8].

Brand names

Qfitlia

Other names

fitusiran