Emicizumab: the hemophilia A game changer

Abstract

In hemophilia, the unmet needs regarding adherence to prophylaxis and lack of effective longterm prophylaxis regimens, especially in patients with inhibitors, led to the production of emicizumab, the first non-factor medicine for subcutaneous administration in patients with severe and moderate hemophilia A with or without factor VIII inhibitors. This article describes the research steps behind the development of this game-changer medication, its success for the prophylaxis of bleeding episodes as witnessed by the results of pivotal clinical trial but also by real life use in the frame of a still expanding global market. We shall also discuss potential and actual adverse events and the nuances related to clinical use such as laboratory monitoring, development of neutralizing anti-drug-antibodies, risk of thrombosis/hypercoagulability and use in the management of surgical operations. The potential of using emicizumab to prevent bleeding in other congenital and acquired coagulation disorders will also be sketched.