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Emicizumab prophylaxis reduces bleeds in hemophilia A
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A randomized phase 3 study designed to evaluate emicizumab prophylaxis in patients aged 12 years or older with hemophilia A and inhibitors to Factor VIII met its primary endpoint, according to the agent’s manufacturer.
The HAVEN 1 study showed patients who underwent emicizumab (ACE910, Genentech) prophylaxis experienced a statistically significant reduction in number of bleeds compared with those who received no prophylaxis.
“The development of inhibitors that render Factor VIII replacement less effective — or ineffective — is one of the greatest challenges in the treatment of hemophilia A today, putting patients at high risk for life-threatening bleeds and repeated bleeds that may cause long-term joint damage,” Sandra Horning, MD, chief medical officer and head of global product development at Genentech, said in a company-issued press release.
The multicenter, open-label study evaluated the safety and efficacy of emicizumab prophylaxis in 109 patients with hemophilia A with inhibitors to Factor VIII who were previously treated with episodic or prophylactic bypassing agents.
Researchers randomly assigned patients 2:1 to emicizumab prophylaxis or no prophylaxis. Patients previously treated prophylactically with bypassing agents also received emicizumab prophylaxis.
Individuals assigned emicizumab prophylaxis demonstrated a significant reduction in the number of bleeds over time compared with those in the control arm.
The study also met all secondary endpoints. These included bleed rate, joint bleed rate, spontaneous bleed rate, target joint bleed rate, health-related quality of life/health status. Researchers also reported improvement in an intrapatient comparison to their bleed rate on their prior prophylaxis regimen with bypassing agents.
The most common adverse events were injection site reactions, and this was consistent with prior studies.
“We are pleased to see that, in our first pivotal trial, emicizumab prophylaxis significantly reduced the number of bleeds over time in people in this difficult-to-treat setting,” Horning said. “We look forward to working with health authorities to bring this treatment to the hemophilia community as soon as possible.”