The U.S. Food and Drug Administration (FDA) has approved Qfitlia (fitusiran) for the treatment of hemophilia A and B with or without inhibitors.
Hemophilia is an inherited bleeding disorder in which the blood does not clot normally. People with the condition will bleed more than normal after an injury, surgery, or dental procedure. In severe cases, spontaneous bleeding may occur. Bleeding into the joints, muscles, brain, or organs can cause pain and other serious complications. Hemophilia A is caused by having low levels of factor VIII protein, which is necessary for the formation of blood clots. The disorder is caused by changes in the F8 gene. Hemophilia B is caused by genetic changes in the F9 gene.
Fitusiran
Fitusiran is a small interference RNA ,first-in-class, antithrombin-lowering (AT) therapy for routine prophylaxis treatment of patients ages 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors. The therapy is a subcutaneous injection of 50 mg. By targeting the reduction of AT, thrombin generation can increase and restore hemostasis in patients with hemophilia.
Clinical Trials
The approval of fitusiran follows positive data from the phase 3 ATLAS clinical trial program. Studies in this program demonstrated fitusiran’s ability to significantly lower annualized bleeding rates in patients without inhibitors by 71% compared to on demand therapy (with clotting factor concentrate) and by 73% in patients with inhibitors compared to patients receiving on demand therapy (with bypassing agent). The median annualized bleeding rate during the open-label extension study was 3.8 in patients without inhibitors and 1.9 in patients with inhibitors. Additionally, the median annualized spontaneous bleeding rate during the open-label extension was 1.9 in both patient groups.
Fitusiran did show potential for significant adverse events such as thrombotic events, acute and recurrent gallbladder disease, and hepatotoxicity. However, the most common adverse events were viral infection, nasopharyngitis, and bacterial infection.
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To learn more about hemophilia and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/