European Commission approves Hemlibra for prophylaxis of bleeding episodes in haemophilia A patients with factor VIII inhibitors.-Roche.

Roche announced that the European Commission has approved Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors. Hemlibra can be used in all age groups. Nearly one in three people with severe haemophilia A can develop inhibitors to factor VIII replacement therapies, putting them at greater risk of life-threatening bleeds or repeated bleeding episodes that can cause long-term joint damage. People with haemophilia A with inhibitors have a 70% increased risk of death compared to those without inhibitors.

The approval is based on two of the largest pivotal clinical studies in people with haemophilia A with inhibitors, in which Hemlibra demonstrated superior efficacy compared to prior treatment with bypassing agents (BPA) as prophylaxis or on-demand.

In the HAVEN 1 study in adults and adolescents (12 years of age or older) with haemophilia A with inhibitors, Hemlibra prophylaxis showed a statistically significant reduction in treated bleeds of 87% (risk rate [RR]=0.13, p<0.0001) compared to no prophylaxis. In a first-of-its-kind intra-patient analysis, Hemlibra prophylaxis resulted in a statistically significant reduction in treated bleeds of 79% (RR=0.21, p=0.0003) compared to previous treatment with bypassing agent (BPA) prophylaxis collected in a non-interventional study (NIS) prior to enrollment.

Interim results from the HAVEN 2 study in children younger than 12 years of age with haemophilia A with inhibitors showed that 87% (95% CI: 66.4; 97.2) of children who received Hemlibra prophylaxis experienced zero treated bleeds. In an intra-patient analysis of 13 children who had participated in the NIS, Hemlibra prophylaxis resulted in a 99% (RR=0.01, 95% CI: 0.004; 0.044) reduction in treated bleeds compared to previous treatment with a BPA.