FDA Approves Novo Hemophilia Prophylaxis Drug

FDA has approved Novo Nordisk’s Alhemo (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients ages 12 and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors. An agency statement says Alhemo is injected subcutaneously daily.

Alhemo’s efficacy and safety were evaluated in a Phase 3 trial with 91 adult and 42 adolescent male patients. Efficacy was determined by comparing the number of treated bleeding episodes between the Alhemo treatment group and the no prophylaxis group.

The most common adverse reactions were injection site reactions and hives. FDA says hypersensitivity reactions including erythema, rash, pruritis, and abdominal pain have occurred in patients treated with Alhemo. Treatment with the drug may increase the risk of blood clots, it says.

Alhemo received priority review and orphan drug designations for the indication.