Emicizumab for haemophilia without FVIIIDecember 13, 2018
The US FDA has approved emicizumab-kxwh(Hemlibra) for routine prophylaxis to reduce the frequency of bleeding episodes in adults and children with haemophiliaA without factor VIII inhibitors.
Emicizumab is now the only prophylactic treatment for people with haemophilia A with and without factor VIII inhibitors that can be administered subcutaneously and at multiple dosing options, Roche said.
This approval is based on positive results from the phase III Haven 3 and Haven 4 studies. Emicizumab prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis and across all other bleed-related endpoints in the Haven 3 study, and showed control of bleeding in the Haven 4 study.
Haven 3 is a randomised, multicentre, open-label, phase III study evaluating the efficacy, safety andpharmacokinetics of emicizumabprophylaxis versus no prophylaxis in people with haemophilia A without factor VIII inhibitors. The study included 152 patients with haemophiliaA who were previously treated with factor VIII therapy.