FDA Approves Eloctate for Patients with Hemophilia A

The U.S. Food and Drug Administration today 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.

Eloctate is approved to help control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes (prophylaxis). 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.

“The approval of this product provides an additional therapeutic option for use in the care of patients with Hemophilia A," said Karen Midthun, M.D., director of the FDA's Center for Biologics Evaluation and Research.

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 5,000 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 to 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 treatment of a rare disease or condition.

Eloctate is manufactured by Biogen Idec, Inc., Cambridge, Mass.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.