Genentech has announced interim results from the Phase III HAVEN 2 study evaluating emicizumab prophylaxis in children less than 12 years of age with hemophilia A and inhibitors to factor VIII.
Interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time. These findings are consistent with results from the Phase III HAVEN 1 study in adults and adolescents (12 years of age or older) with hemophilia 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 HAVEN 2 study were injection site reactions and nasopharyngitis (common cold symptoms).
“Managing hemophilia 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,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “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 program 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.