Posted by Michael Wonder on 17 Apr 2018
FDA grants breakthrough therapy designation for Roche’s Hemlibra in haemophilia A without inhibitors
17 April 2018 - Designation based on phase III HAVEN 3 study demonstrating Hemlibra prophylaxis significantly reduced bleeds compared to no prophylaxis.
Roche today announced that the US FDA has granted breakthrough therapy designation to Hemlibra (emicizumab-kxwh) for people with haemophilia A without factor VIII inhibitors.
Hemlibra was granted its first breakthrough therapy designation in September 2015 and was approved by the FDA in November 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors based on results from the HAVEN 1 and HAVEN 2 studies. Hemlibra was also recently approved by regulatory authorities in other countries around the world, including by the European Commission in February 2018 for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors.
