Swiss pharma giant Roche (ROG: SIX) has presented data from the largest pivotal study to date in children with hemophilia A at the American Society of Hematology Annual Meeting.
The HAVEN study evaluated Hemlibra (emicizumab) prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII inhibitors, including longer follow-up for once-weekly dosing and new data for less frequent dosing schedules.
Nearly 77% of children receiving the Roche drug once weekly experienced zero treated bleeds, and they were reduced by 99% compared to prior bypassing agents in a prospective intra-patient comparison.
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