FDA accepts sBLA for Hemlibra to treat hemophilia A without factor VIII inhibitors.- Genentech/Roche.

Genentech, a member of the Roche Group announced that the FDA has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for Hemlibra (emicizumab-kxwh) for adults and children with hemophilia A without factor VIII inhibitors. The sBLA is based on data from the Phase III HAVEN 3 study. The FDA is expected to make a decision on approval by October 4, 2018.

In the HAVEN 3 study, adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors who received Hemlibra prophylaxis every week or every two weeks showed a 96 percent (p<0.0001) and 97 percent (p<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis. In an additional arm of the study, people who had previously received factor VIII prophylaxis in a non-interventional study switched to Hemlibra prophylaxis, allowing for an intra-patient comparison of two prophylaxis regimens. Based on the intra-patient comparison, Hemlibra demonstrated a statistically significant reduction of 68 percent (p<0.0001) in treated bleeds, making it the first medicine to show superior efficacy to prior treatment with factor VIII prophylaxis, the standard of care. There were no unexpected or serious adverse events (AEs) related to Hemlibra in the HAVEN 3 study.