Growing body of evidence supports Hemlibra in treatment of all patients with hemophilia A

Genentech announced positive results from the Phase III HAVEN 3 study evaluating Hemlibra (emicizumab-kxwh) in adults and adolescents (aged 12 years or older) with hemophilia A without inhibitors to factor VIII. The study met its primary endpoint, showing a statistically significant and clinically meaningful reduction in the number of treated bleeds over time in people receiving Hemlibra prophylaxis every week compared to those receiving no prophylaxis. The study also met key secondary endpoints, including a statistically significant and clinically meaningful reduction in the number of treated bleeds over time with Hemlibra prophylaxis dosed every two weeks compared to no prophylaxis.

Importantly, once-weekly Hemlibra prophylaxis was superior to factor VIII prophylaxis, as demonstrated by a statistically significant and clinically meaningful reduction in treated bleeds in an intra-patient comparison of patients receiving Hemlibra prophylaxis compared to their prior factor VIII prophylaxis. The most common adverse events with Hemlibra were injection site reactions, with no new safety signals observed. No thrombotic microangiopathy or thrombotic events occurred in this study. Data from the HAVEN 3 study will be presented at an upcoming medical meeting and submitted to health authorities around the world for approval consideration.

Comment: These results add to the growing body of evidence shown in the inhibitor population and support that Hemlibra may benefit all people with hemophilia A regardless of inhibitor status. Data from the HAVEN 1 and HAVEN 2 studies supported the recent U.S. Food and Drug Administration approval of Hemlibra for adults and children with hemophilia A with inhibitors, and are being reviewed under accelerated assessment by the European Medicines Agency.