The European Commission has approved emicizumab for routine prophylaxis of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended granting marketing authorization to emicizumab (Hemlibra) in January 2018 and it was approved by the Food and Drug Administration in November 2017.
The approvals are based on findings from the HAVEN 1 trial (in 109 adults and adolescents) and the HAVEN 2 trial (in children younger than age 12 years), according to the drug’s maker, Roche. In HAVEN 1 (NCT02622321), emicizumab showed a significant reduction in treatment bleeds (87%), compared with no prophylaxis. And the drug reduced treated bleeds by 79%, compared with previous treatment with bypassing agent prophylaxis. An interim analysis of HAVEN 2 (NCT02795767) of 23 children, showed that 87% of children who received emicizumab prophylaxis had zero treated bleeds, according to Roche.