FDA OKs Genentech’s Hemlibra for Some Hep A

FDA has approved Genetech’s Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A without factor VIII inhibitors. The company says Hemlibra is the only prophylactic treatment for people with hemophilia A and without factor VIII inhibitors that can be administered subcutaneously and at multiple dosing options.

The approval was based on positive results from the Phase 3 HAVEN 3 and HAVEN 4 studies in which the drug reportedly led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis.

Hemlibra was granted breakthrough designation for hemophilia A without factor VIII inhibitors. It also was granted priority review. The product was approved by FDA 11/2017 for adults and children with hemophilia A with factor VIII inhibitors.