Emicizumab-kxwh prophylaxis administered once every 4 weeks conferred clinically meaningful bleeding control among patients aged 12 years and older with hemophilia A, according to study results released by the agent’s manufacturer.
Emicizumab-kxwh (Hemlibra, Genentech) is a bispecific factor IXa- and factor X-directed antibody designed to bring together factor IXa and factor X, proteins necessary to restore the blood clotting process for patients with hemophilia A.
The FDA last month approved the agent to prevent or reduce the frequency of bleeding episodes among patients with hemophilia who have developed factor VIII inhibitors.
An interim analysis of the single-arm, open-label phase 3 HAVEN 4 study — performed after a median 17 weeks of treatment with emicizumab-kxwh — showed benefit with regard to bleeding control among patients with and without factor VIII inhibitors.
The results are consistent with prior studies of emicizumab-kxwh dosed once weekly or every 2 weeks, according to a Genentech-issued press release. These studies included the HAVEN 1 and HAVEN studies, which evaluated the agent in adults and children with hemophilia A with inhibitors, and the HAVEN 3 study, which included adults and adolescents with hemophilia without inhibitors.
“Current treatment regimens for hemophilia A can require frequent intravenous infusions. We are encouraged that Hemlibra prophylaxis administered by injection under the skin once every 4 weeks showed clinically meaningful bleed control in people with hemophilia A,” Sandra Horning, MD, chief medical officer and head of global product development for Genentech, said in the release. “Together with the findings from other phase 3 studies, these interim results support the potential for Hemlibra to be dosed at different schedules, allowing patients to choose the option that’s right for them.”
HAVEN 4 included 48 patients aged 12 years or older with hemophilia A, with or without factor VIII inhibitors. All patients underwent prior treatment with factor VIII or bypassing agents, either on demand or as prophylaxis.
The most common adverse event associated with emicizumab-kxwh was injection site reaction. Researchers observed no new safety signals in HAVEN 4. No thrombotic microangiopathy or thrombotic events occurred.
Additional data from HAVEN 4 will be submitted to regulatory authorities and presented at an upcoming medical meeting, according to the press release.