Emicizumab for haemophilia A without FVIII in ECMay 8, 2019
Roche said EC cleared emicizumab (Hemlibra) for routine prophylaxis of bleeding episodes in people with severe haemophilia A without factor VIII inhibitors from EC.
Emicizumab can be used in all age groups and can also now be used at multiple dosing options for all indicated people with haemophilia A, including those with factor VIII inhibitors.
This approval is based on results from the pivotal HAVEN 3 and HAVEN 4 studies. In the HAVEN 3 study in people with haemophilia A without factor VIII inhibitors, emicizumab prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis and compared to prior treatment with factor VIII prophylaxis in a prospective intra-patient comparison.
In the HAVEN 4 study in people with haemophilia A with and without factor VIII inhibitors, emicizumab showed a clinically meaningful control of bleeding when dosed every four weeks.\
Emicizumab, was approved by the US FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with haemophilia A without factor VIII inhibitors, following Priority Review, in 2018.