Genentech announced that the Food and Drug Administration (FDA) has approved Hemlibra (emicizumab-kxwh) injection for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A with factor VIII inhibitors.

Hemlibra, a bispecific factor IXa- and factor X-directed antibody, brings together factors IXa and X proteins; they are required to activate the natural coagulation cascade and restore the clotting process for hemophilia A patients. 

The Phase 3 randomized, open-label HAVEN 1 study enrolled 109 patients aged ≥12 years with hemophilia A with inhibitors. Patients who received Hemlibra prophylaxis experienced a statistically significant reduction in treated bleeds by 87% (95% CI: 72.3, 94.3; P<00001) vs. patients who did not receive prophylaxis. 

In addition, findings from an intra-patient analysis revealed Hemlibra prophylaxis statistically and significantly reduced treated bleeds of 79% (95 CI: 51.4, 91.1; P=0.0003) vs. previous treatment with bypassing agent (BPA) prophylaxis collected in a non-interventional study before enrollment. 

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The single-arm, open-label HAVEN 2 study enrolled patients <12 years with hemophilia A with inhibitors. The data showed 87% of those who received Hemlibra prophylaxis had zero treated bleeds. An intra-patient analysis (n=13) showed a 99% reduction in treated bleeds with Hemlibra prophylaxis vs. previous treatment with BPA prophylaxis or on-demand. 

Pooled safety data showed injection site reactions, headache, and arthralgia as the most common adverse events (≥10% occurrence).

“This new medicine has been shown to reduce the frequency of bleeds compared to the currently available medicines and only needs to be injected once a week. This could make a meaningful difference for these children,” said Guy Young, MD, director of Hemostasis and Thrombosis Program, Children’s Hospital Los Angeles, CA. 

Hemlibra will be available as 30mg/mL, 60mg/0.4mL, 105mg/0.7mL, and 150mg/mL strengths in single-dose vials in the near future. 

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