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FDA grants breakthrough therapy designation to Helimbra for hemophilia A without inhibitors
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The FDA granted breakthrough therapy designation to emicizumab-kxwh for the treatment of patients with hemophilia A without factor VIII inhibitors, according to the agent’s manufacturer.
Emicizumab-kxwh (Helimbra; Chugai Pharmaceutical, Roche/Genentech) — a bispecific factor IXa- and factor X-directed antibody — is designed to bring together factor IXa and factor X to activate the natural coagulation cascade and restore the blood clotting process.
“Helimbra is the first medicine to show superior efficacy compared [with] factor VIII prophylaxis, the standard of care for people with hemophilia A without inhibitors, in an intrapatient comparison,” Sandra Horning, MD, chief medical officer and head of Global Product Development, said in a company-issued press release. “We look forward to working with health authorities to make Helimbra available to people without inhibitors as soon as possible, and we are excited to share this news with the community as we join in celebrating World Hemophilia Day.”
The FDA’s decision was based on data from the randomized phase 3 HAVEN 3 study of 152 patients aged at least 12 years with hemophilia A without inhibitors who previously received factor VIII therapy either on-demand or for prophylaxis.
Patients who received emicizumab-kxwh prophylaxis subcutaneously every week or every 2 weeks achieved a significant and clinically meaningful reduction in treated bleeds compared with patients who did not receive prophylaxis.
Also, an intrapatient comparison showed once-weekly emicizumab-kxwh prophylaxis was superior to prior factor VIII prophylaxis by leading to a significant and clinically meaningful reduction in treated bleeds.
Injection site reactions were the most common adverse event associated with emicizumab-kxwh. Researchers observed no thrombotic microangiopathy or thrombotic events.
The FDA approved emicizumab-kxwh last year for routine prophylaxis to prevent or reduce the frequency of bleeding episodes among adults and children with hemophilia A and factor VIII inhibitors based on the HAVEN 1 and HAVEN 2 studies.