The U.S. FDA has approved fitusiran for the routine prophylactic treatment of adult and pediatric patients (aged 12 and older) with hemophilia A or B, with or without factor VIII or IX inhibitors. Fitusiran is an antithrombin-directed small interfering ribonucleic acid and the first antithrombin-lowering therapy for hemophilia A or B. Clinical evidence In clinical trials, fitusiran significantly reduced annualized bleeding rates compared to an on-demand bypassing agent (5.1 vs.
19.1) in hemophilia patients with inhibitors and to clotting factor concentrates (9.0 vs. 31.4) in those without inhibitors after nine months of treatment. Dosage and administration Administer subcutaneously Starting dose: 50 mg once every two months Contraindications None Warnings and precautions Hepatotoxicity Adverse reactions Viral infection Nasopharyngitis Bacterial infection Did you find this FDA Alert…
