Recent Developments of Coagulation Factor Products for Hemophilia Treatment

Dear Colleagues,

Hemophilia management first started in the 1970s upon the introduction of plasma-derived concentrates of coagulation factor VIII and factor IX, which provided effective treatment of the bleeding episodes in patients with hemophilia A and B, respectively. During 1990s, the management of hemophilia was transformed from treating bleeding events using plasma concentrates to prophylactically preventing them by using recombinant coagulation factors. Importantly, the last decade has witnessed further progress toward better therapeutic interventions for hemophilia as represented by the introduction of extended half-life recombinant coagulation factors (Fc-fused FIX eftrenonacog alfa and Fc-fused FVIII efmoroctocog alfa among others). Despite such progress, certain needs remained unmet. Particularly, hemophilia patients with FVIII inhibitors are not good candidates for prophylaxis. Therefore, non-factor therapeutic interventions were developed. Some of those interventions are already approved, such as emicizumab, while others are in advanced clinical development. Emicizumab is a bispecific monoclonal antibody that facilitates interactions between factor IXa and factor X; thus, it promotes thrombin generation by mimicking factor VIIIa activity irrespective of factor VIII deficiency or the presence of its inhibitors. With this breakthrough in the way that different types of hemophilia are being treated, the horizon for new breakthrough interventions appears to be more promising.

This Special Issue titled “Recent Development of Therapeutics for Hemophilia Treatment” invites authors to contribute reviews or original research articles dedicated to the most recent advances on therapeutics for hemophilia treatment. The contributions include the discovery of new therapeutic entities, as well as their assessment in the context of hemophilia. Studies concerning the elucidation of mechanisms of action of new therapeutics are also welcomed.

Dr. Rami A. Al-Horani
Guest Editor

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