Roche has announced interim results from the phase III HAVEN 2 study evaluating emicizumab prophylaxis in children less than 12 years of age with haemophilia A and inhibitors to factor VIII.
Emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time at this interim analysis, after a median of 12 weeks of treatment.
These findings are consistent with results from the phase III HAVEN 1 study in adults and adolescents (12 years of age or older) with haemophilia A and inhibitors to factor VIII, in which emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time compared to no prophylaxis, as well as compared to prior prophylaxis with bypassing agents. The most common adverse events with emicizumab in the HAVEN2 study were injection site reactions and nasopharyngitis.
Dr Sandra Horning, Chief Medical Officer and Head of Global Product Development, Roche, said:"Managing haemophilia A with inhibitors to factor VIII is especially challenging for children and their caregivers, because bleeding is difficult to control and current treatments require frequent intravenous infusions. “We are encouraged that once-weekly subcutaneous emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time in children.”
HAVEN 2 is the second phase III study in the emicizumab clinical development programme to report results. Data from both HAVEN 1 and the interim data from HAVEN 2 studies will be presented at an upcoming medical meeting and submitted to health authorities for approval consideration.
Two additional phase III studies of emicizumab, HAVEN 3 and HAVEN 4, are ongoing.