FDA approves Hemlibra for routine prophylaxis in haemophilia A

The FDA has approved Hemlibra (emicizumab-kxwh), from Roche, 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. Hemlibra is now the only prophylactic treatment for people with haemophilia A with and without factor VIII inhibitors that can be administered subcutaneously (under the skin) and at multiple dosing options (once weekly, every two weeks or every four weeks).

This approval is based on positive results from the phase III HAVEN 3 and HAVEN 4 studies. Hemlibra prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis (primary endpoint) and across all other bleed-related endpoints in the HAVEN 3 study, and showed a clinically meaningful control of bleeding in the HAVEN 4 study.

In the phase III HAVEN 3 study, adults and adolescents aged 12 years or older with haemophilia A without factor VIII inhibitors who received Hemlibra prophylaxis once weekly (n=36) or every two weeks (n=35) experienced a 96% (95% CI: 92.5; 98.0, p<0.0001) and 97% (95% CI: 93.4; 98.3, p<0.0001) reduction in treated bleeds, respectively, compared to those who received no prophylaxis (n=18). Hemlibra is the first medicine to significantly reduce treated bleeds compared to prior factor VIII prophylaxis, which has been the recommended standard of care, as demonstrated by a statistically significant reduction of 68% (95% CI: 48.6; 80.5, p<0.0001) in treated bleeds in a prospective intra-patient comparison (n=48) of people who previously received factor VIII prophylaxis in a non-interventional study and switched to Hemlibra prophylaxis. In the single-arm phase III HAVEN 4 study of adults and adolescents aged 12 years or older with haemophilia A with factor VIII inhibitors (n=5) and without factor VIII inhibitors (n=36), Hemlibra prophylaxis every four weeks (n=41) led to clinically meaningful control of bleeding. The most common adverse reactions occurring in 10% or more of people treated with Hemlibra in pooled studies (n=391) were injection site reactions (n=85), headache (n=57) and joint pain (arthralgia; n=59).

Comment: Hemlibra was granted Breakthrough Therapy Designation by the FDA for haemophilia A without factor VIII inhibitors. It was also granted Priority Review, a designation given to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease.