The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab) injection as a once-daily prophylaxis treatment to prevent or reduce frequency of bleeding episodes in patients ages 12 years and older with hemophilia A or B without inhibitors. This is an expansion on the 2024 approval of concizumab for hemophilia A or B with inhibitors.
Hemophilia is a rare bleeding disorder that slows the blood clotting process. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. The major types of this disorder are Hemophilia A and Hemophilia B. Although the two types have very similar signs and symptoms, they are caused by mutations in genes encoding for coagulation factor VIII or factor IX, respectively.
Concizumab is a tissue factor pathway inhibitor antagonist that enhances factor Xa production during initiation of coagulation, leading to improvements in thrombin production and clot formation. The approval follows results from the phase 3 explorer8 clinical trial evaluating the safety and efficacy of concizumab in patients ages 12 years and older with hemophilia A or B without inhibitors.
The trial enrolled a total of 156 male patients with hemophilia A or B with a primary objective to compare the number of treated spontaneous and traumatic bleeding episodes, measured by the annualized bleed rate (ABR), in patients receiving concizumab prophylaxis versus no prophylaxis.
Results illustrated a statistically significant reduction in ABR of 79% for patients with hemophilia B and 86% for patients with hemophilia A. Notable reductions in the average and median ABRs were also observed across patient groups. Patients with hemophilia B had an average ABR of 3.1 and a median ABR of 1.6, compared to 14.8 and 14.9, respectively, in the non-prophylaxis group. Patients with hemophilia A had an average ABR of 2.7 and a median ABR of 2.9, compared to 19.3 and 19.6, respectively, in the non-prophylaxis group.
The most common adverse events were injection site reactions and headache.
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To learn more about hemophilia and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/
