FDA Approves New Hemophilia A Drug

The U.S. Food and Drug Administration (FDA) has approved emicizumab-kxwh for the treatment of adult and pediatric patients with hemophilia A without factor VIII inhibitors. The drug is indicated for regular prophylaxis to prevent or reduce the frequency of bleeding episodes in children and adults.

The agency’s approval is based on results from the phase III HAVEN 3 and HAVEN 4 clinical trials. In the HAVEN 3 study, adults and adolescents with hemophilia A without factor VIII inhibitors received either no treatment or treatment with emicizumab-kxwh once weekly or every other week. The weekly and biweekly arms experienced 96-percent and 97-percent reductions in treated bleeds, respectively, compared with the control group (p<0.0001).

In the single-arm HAVEN 4 trial, adult and adolescent patients with hemophilia A with and without factor VIII inhibitors were treated with emicizumab-kxwh once every four weeks. The results led to a clinically meaningful control of bleeding (p value not provided).

Common adverse events occurring in both trials included injection site reactions, headache, and joint pain.

Source: FDA news release, October 4, 2018; Business Wire, October 4, 2018.