The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to emicizumab-kxwh for the treatment of patients with hemophilia A without factor VIII inhibitors.
The FDA based its decision on data of the randomized phase III HAVEN 3 study of 152 patients (aged 12 years or older) with hemophilia A without inhibitors who previously received factor VIII therapy either on-demand or for prophylaxis. In the trial, prophylactic emicizumab-kxwh administered subcutaneously every week or every two weeks led to a statistically significant reduction in the number of treated bleeds, compared with no prophylaxis.
The most common adverse events with emicizumab-kxwh were injection-site reactions, and no new safety signals were observed. No thrombotic microangiopathy or thrombotic events occurred in this study.
The FDA approved emicizumab-kxwh for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors in November 2017, based on results from the HAVEN 1 and HAVEN 2 studies.
Source: Genentech press release, April 16, 2018.