Advances in biopharmaceutical products for hemophilia

Abstract

Hemophilia is caused by the deficiency of clotting factors due to a single genetic abnormality. Replacement therapies have evolved from plasma-derived to recombinant coagulation factor concentrates but continue to have certain limitations. Monoclonal antibodies are clinical prophylactic treatment options unaffected by inhibitors and have better compliance than coagulation factor concentrates for patients with hemophilia. Gene therapy is a breakthrough in hemophilia treatment, as it drives the hepatic expression of factor VIII or factor IX and requires only a single administration to enable long-term replacement treatment in adult patients. Furthermore, biopharmaceutical products that target new pathways unaffected by inhibitors, including tissue factor pathway inhibitors, activated protein C, and antithrombin, as well as pharmaceutical technology advances to reduce dosing frequency, have demonstrated promising clinical results. This review provides a comprehensive overview of these biopharmaceutical products and explores the future of hemophilia treatment.