FDA Approves New Prophylactic Treatment For Hemophilia A Without Factor VIII Inhibitors
October 4th, 2018
On October 4, 2018, the FDA approved Hemlibra® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors. Hemlibra is now the only medicine approved to treat people of all ages with hemophilia A with and without factor VIII inhibitors.
News in brief
- FDA approval based on positive results from Phase III studies, including the HAVEN 3 study showing Hemlibra prophylaxis significantly reduced treated bleeds compared to no prophylaxis in people with hemophilia A without factor VIII inhibitors
- First medicine to significantly reduce treated bleeds compared to prior factor VIII prophylaxis based on a prospective intra-patient comparison
- Only approved medicine that can be self-administered subcutaneously once weekly, every two weeks or every four weeks for hemophilia A with and without factor VIII inhibitors
- The efficacy and safety of Hemlibra has been demonstrated in one of the largest pivotal clinical trial programs in hemophilia A
Supporting Information
"Today’s approval of Hemlibra reflects our commitment to groundbreaking science and the development of medicines with the potential to redefine the standard of care. Hemlibra is now the only FDA-approved medicine for people with hemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across four pivotal studies. We want to thank the hemophilia community for their partnership in helping us bring this new option to everyone living with hemophilia A.”