The Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Hemlibra (emicizumab-kxwh; Genentech) for the treatment of hemophilia A without factor VIII inhibitors.

Hemlibra is a prophylactic, subcutaneous treatment consisting of bispecific factor IXa- and factor X-directed antibody. It is designed to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A patients. 

The FDA’s designation is based on primary results from the randomized, open-label, Phase 3 HAVEN 3 study. The trial enrolled 152 patients with hemophilia A who were previously treated, either on-demand or as prophylaxis, with factor VIII therapy. Patients who were previously treated with on-demand factor VIII were randomized to receive Hemlibra at either 3mg/kg/wk for 4 weeks, followed by 1.5mg/kg/wk until the end of study, Hemlibra at 3mg/kg/wk for 4 weeks, followed by 3mg/kg/2wks until the end of study or no prophylaxis. Those who were previously treated with factor VIII prophylaxis received Hemlibra at 3mg/kg/wk for 4 weeks, followed by 1.5mg/kg/wk until the end of study. 

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Primary results showed Hemlibra treatment was linked to a significant reduction in treated bleeds compared to no treatment. In an intra-patient comparison, once-weekly Hemlibra treatment was superior to prior factor VIII prophylaxis as demonstrated by a statistically significant and clinically meaningful reduction in treated bleeds. The full results from the HAVEN 3 trial are expected to be released in in the first half of this year. 

Hemlibra was approved last November to prevent or reduce the frequency of bleeding episodes in those with hemophilia A with factor VIII inhibitors. “We look forward to working with health authorities to make HEMLIBRA available to people without inhibitors as soon as possible,” said Sandra Horning, MD, CMO at Genentech. 

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