Hemlibra Approved in Canada to Treat Hemophilia A Patients with Factor VIII Inhibitors

Health Canada has approved Hemlibra (emicizumab) as a treatment to prevent or reduce bleeding in Hemophilia A patients in Canada who have factor VIII inhibitors.

Hemlibra is an antibody therapy designed to combine factors IX and X of the blood clotting series and promote clotting. Restoring the missing function of factor VIII by an alternate method makes Hemlibra beneficial for the treatment of hemophilia A patients who develop inhibitors.

Initially created by Japan-based Chugai Pharmaceuticals, the therapy is now co-developed by Chugai, along with Roche and its subsidiary, Genentech.

“Many people with severe hemophilia A face an ongoing struggle to control their bleeds, live with pain caused by joint damage and their lives revolve around treatment infusions. Until now, treatment options have been limited for those with inhibitors to factor VIII, and there hasn’t been a new medicine in the past 20 years,” Paul Wilton, president of the Canadian Hemophilia Society, said in a press release. “Hemlibra is a significant innovation for people with inhibitors, and we are pleased that it is now approved for Canadian patients.”

Data from two Phase 3 clinical studies, HAVEN 1 (NCT02622321) and HAVEN 2 (NCT02795767) supported Health Canada’s decision. The two trials proved the effectiveness and safety of weekly Hemlibra injections in adolescents and adults (HAVEN 1) and children (HAVEN 2) with hemophilia A with factor VII inhibitors.

Both studies reported an 87% reduction in treated bleeds with Hemlibra over no treatment. Compared with other bypassing agents as preventive or on-demand treatment, there was a significant reduction in treated bleeds observed in Hemlibra-treated adolescent and adult patients (79%) and in children (99%).

Hemlibra treatment also markedly improved hemophilia-associated quality of life in adolescents and adults after 25 weeks of treatment.

“Preventing bleeds in patients with hemophilia A can be extremely challenging, usually requiring patients to self-infuse medications multiple times a week, or even daily. The development of inhibitors adds a significant challenge, with more demanding treatments that are often less effective,” said Jayson Stoffman, MD, an associate professor in the Department of Pediatrics and Child Health at the University of Manitoba and medical director of the Manitoba Bleeding Disorders Program.

“Hemlibra offers these patients the chance to effectively reduce the frequency of their bleeds with a once-weekly injection at home. This could significantly improve the quality of life for inhibitor patients, and particularly children and their families,” he said.

Two ongoing clinical trials — HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) — indicate that the therapy may be beneficial to a wider population of patients, as it was found to outperform current standard-of-care therapies both in hemophilia A patients with inhibitors and those without them.

These results were recently discussed in a Hemophilia News Today interview with Gallia Levy, MD, PhD, a hematologist and associate group medical director for Genentech.

The U.S. Food and Drug Administration approved Hemlibra in November 2017, and the European Commission followed suit in March this year.