Pharmacist Medication Insights: Emicizumab (Hemlibra) for Hemophilia A

Emicizumab (Hemlibra, Roche) is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with or without factor VIII (FVIII) inhibitors.

Hemophilia A is an inherited disorder that causes an individual’s blood to not clot properly, leading to uncontrolled and spontaneous bleeding. It affects approximately 320,000 people worldwide and between 50% and 60% of these patients have a severe form of the disorder.

Patients with hemophilia A either have very little or no FVIII, a clotting factor that helps form clots and stop bleeding in healthy individuals. Although many patients with hemophilia A receive FVIII as part of their treatment, the development of inhibitors to FVIII replacement therapies can be a major complication. Inhibitors are antibodies developed by the body’s immune system that bind to and block the replacement FVIII, limiting its ability to control bleeding.

Data from the pooled, 3-year follow-up of 401 patients with hemophilia A from the HAVEN studies 1 through 4 show that emicizumab maintained low treated bleed rates and was well tolerated in patients of all ages with hemophilia A, both with and without FVIII inhibitors.