Emicizumab for mild or moderate haemophilia A

Emicizumab is currently in development for the treatment of congenital mild or moderate haemophilia A, without factor VIII inhibitors. Haemophilia A is a genetic bleeding disorder caused by insufficient levels of a blood clotting factor, called factor VIII (eight). This causes poor blood clotting, which results in difficulty in stopping the flow of blood from a wound, causing prolonged bleeding. There is currently no cure for haemophilia A. It is managed by lifelong prophylactic (preventative) treatment to prevent bleeding episodes from occurring. Bleeds can be prevented or reduced by injections of factor VIII into the vein. However, the half-life of factor VIII means >2 infusions per week are necessary; resulting in a substantial treatment burden, thus treatments with a high efficacy and reduced burden are needed