Roche's emicizumab shows treatment benefit in second late-stage hemophilia A study

Roche's emicizumab hemophilia drug shows positive results with children

Roche's emicizumab shows treatment benefit in second late-stage hemophilia A study

HAVEN 2 is a single-arm, multicenter, open-label clinical trial evaluating the efficacy, safety and pharmacokinetics of emicizumab in children less than 12 years old with hemophilia A and inhibitors to factor VIII who require treatment with bypassing agents. The Swiss pharmaceutical company conducted a second trial of the drug, dubbed HAVEN 2, and results showed that the medicine reduced the number of bleeding episodes in children with hemophilia under the age of 12 years. "Managing hemophilia A with inhibitors to factor VIII is especially challenging for children and their caregivers, because bleeding is hard to control and current treatments require frequent intravenous infusions", Sandra Horning, MD, chief medical officer and head of Global Product Development at Roche, said in a release.

HAVEN 3 is evaluating emicizumab prophylaxis given once a week to patients 12 years old or older with hemophilia A but without inhibitors to factor VII; and HAVEN 4, which will evaluate emicizumab dosed every four weeks in patients 12 years old or older with hemophilia A with or without inhibitors to factor VII. At this interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time. Data from both HAVEN studies will be presented at an upcoming medical meeting and submitted to regulatory agencies for approval consideration.

The drug, called ACE910, was originally developed by Roche's majority-owned Chungai, and it is created to restore the normal blood clotting process and to bind IX and X factors. Emicizumab was found to be generally well-tolerated, with injection site reactions and nasopharyngitis as the most common adverse events. Findings from the HAVEN 2 study were consistent with results from the phase 3 HAVEN 1 study in adults and adolescents 12 years or older with hemophilia A and inhibitors to factor VIII.

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