New therapeutic approaches for hemophilia : non-substitutive therapies

Abstract

Thanks to the great efforts of clinical research, the treatment of hemophilia has been revolutionized. Several treatments using different mechanisms have been studied. Only Emicizumab, a bispecific monoclonal antibody mimicking the activity of the factor VIII cofactor, has been granted marketing authorization in Europe, America and even Tunisia. It is currently available to clinicians for the subcutaneous treatment of hemophilia A, with or without inhibitors. Other drugs are currently being evaluated for coagulation rebalancing. They target natural anticoagulants, such as fitusiran, an antithrombin inhibitor, marstacimab or concizumab, inhibitors of the tissue factor pathway, or SerpinPC, an activated protein C inhibitor.  In this article, we will provide an overview of the main results of clinical trials related to the development of new factor-free replacement therapies.