The Food and Drug Administration (FDA) has approved Eloctate, Antihemophilic Factor (Recombinant), Fc fusion protein, for use in adults and children who have hemophilia A. Eloctate is the first hemophilia A treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding. Approximately 16,000 people suffer from hemophilia A in the United States. Two months ago the FDA approved a new hemophilia treatment for hemophilia B (Alprolix), also manufactured by Biogen Idec.
Eloctate is approved to help control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes. Eloctate consists of the Coagulation Factor VIII molecule (historically known as Antihemophilic Factor) linked to a protein fragment, Fc, which is found in antibodies. This makes the product last longer in the patient’s blood.
Hemophilia A is an inherited, sex-linked, blood-clotting disorder, which primarily affects males, and is caused by defects in the Factor VIII gene. Hemophilia A affects 1 in every 5000 males born in the United States. People with hemophilia A can experience repeated episodes of serious bleeding, mainly into the joints, which can be severely damaged by the bleeding.
The safety and efficacy of Eloctate were evaluated in a clinical trial of 164 patients that compared the prophylactic treatment regimen with on-demand therapy. The trial demonstrated that Eloctate was effective in the treatment of bleeding episodes, in preventing or reducing bleeding and in the control of bleeding during and after surgical procedures. No safety concerns were identified in the trial.
Eloctate received orphan drug designation for this use by the FDA because it is intended for the treatment of a rare disease or condition.
- Food and Drug Administration (FDA). FDA approves the first antihemophilic factor, Fc fusion protein for patients with hemophilia A [news release]. June 6, 2014. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm400167.htm. Accessed June 9, 2014.