[8][11][13] Concizumab is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people aged twelve years of age and older with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors; or hemophilia B (congenital factor IX deficiency) with factor IX inhibitors.
[5][6][7][8][11] The efficacy and safety of concizumab were evaluated in a multi-national, multi-center, open-label, phase III trial (NCT04083781) with 91 adult and 42 adolescent male participants with hemophilia A or B with inhibitors who have been prescribed, or are in need of, treatment with therapies that bypass the inhibitor effect.
[11] The FDA granted the application for concizumab priority review, breakthrough therapy, and orphan drug designations.
[11] In October 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Alhemo, intended for the prevention of bleeding in people with hemophilia A and FVIII inhibitors or hemophilia B and FIX inhibitors.
[9][14] Concizumab was authorized for medical use in the European Union in December 2024.