FDA Approves Eloctate for Bleeding Episodes in Hemophilia A

September 10, 2014
Hemophilia Reports, August 2014, Volume 1, Issue 2

Fewer injections and longer duration of treatment effect should lead to significant improvements in quality of life.

On June 6, 2014, the US FDA approved Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for the prevention and treatment of bleeding episodes in patientswith hemophilia A. Patients can deed for people with severe hemophilia, and following a protective regimen can be burdensome given the frequency of infusions required,” said Patrick F. Fogarty, MD, assistant professor of medicine at the Hospital of the University of Pennsylvania, and director, Penn Comprehensive Hemophilia and Thrombosis Program. “Infusion frequency is a major challenge for people with hemophilia, and I believe Eloctate begins to address this burden while protecting against bleeding episodes.”

Clinical trials evaluated the safety and efficacy of Eloctate in 164 adult patients ages 12 years and older. Patients receiving prophylactic treatment had significantly fewer bleeding episodes than patients who used on-demand therapy. In addition, one to two infusions of Eloctate were able to stop 98 percent of bleeding episodes. Adverse effects were mild and the study did not reveal any safety issues with the drug.

Eloctate, manufactured by Biogen Idec, Inc., Cambridge, MA, was designated as an orphan drug because hemophilia A is a rare disease, affecting approximately 16,000 people in the United States. A price has not yet been set for Eloctate, but the head of Biogen’s commercial operations, Tony Kingsley, said the price should be approximately the same as shorter acting therapy options. Eloctate is set to be commercially available by July 2014.