The Food and Drug Administration (FDA) has approved Hemlibra (emicizumab-kxwh; Genentech) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A with or without factor VIII inhibitors. Previously, Hemlibra, a bispecific factor IXa- and factor X-directed antibody, was approved for patients with hemophilia A with factor VIII inhibitors.
The expanded approval was based on data from two Phase 3 clinical trials: HAVEN 3 and HAVEN 4. In the HAVEN 3 study (N=89), patients with hemophilia A without factor VIII inhibitors who received Hemlibra prophylaxis once weekly or every 2 weeks experienced a 96% (95% CI: 92.5, 98.0; P<.0001) and 97% (95% CI: 93.4, 98.3; P<.0001) reduction in annualized bleed rate (ABR), respectively, compared with those who received no prophylaxis. In the HAVEN 4 study (N=48), Hemlibra prophylaxis every 4 weeks (N=41) was associated with clinically meaningful control of bleeding in patients with hemophilia A with factor VIII inhibitors and without factor VIII inhibitors (ABR: 2.4; 95% CI: 1.4, 4.3).
“Hemlibra is now the only FDA-approved medicine for people with hemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across 4 pivotal studies,” said Sandra Horning, MD, chief medical officer and head of Global Product Development.
The updated labeling includes new treatment regimens that allow for maintenance doses to be administered once weekly, every 2 weeks or every 4 weeks; selection of maintenance dose should be based on clinician preference with consideration of regimens that may increase patient adherence.
Hemlibra is available in 30mg/mL, 60mg/0.4mL, 105mg/0.7mL, and 150mg/mL strengths in single-dose vials.
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