The Food and Drug Administration has announced Monday its approval for the hemophilia A drug, Eloctate, for use in treating bleeding episodes, managing bleeding from surgical procedures, and reducing the frequency of future bleeding.
Eloctate, a recombinant drug, meaning in this case that it uses a blend of different proteins to achieve its desired effect, also consists of the Coagulation Factor VIII molecule. This molecule is linked to a protein fragment, Fc, that helps it to stay in the patient’s body longer. The FDA announced that this method of stopping bleeding is the first of its kind in treating hemophilia A — an inherited, sex-linked, blood clotting disorder.
“The approval of this product provides an additional therapeutic option for use in the care of patients with Hemophilia A,” said Dr. Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research, in the announcement.
Hemophilia A is the most common of the hereditary genetic disorders known as hemophilia. It’s marked by a deficiency in clotting factor VIII, an essential protein that helps the body seal up internal and external bleeding sites. Without it, the blood can’t coagulate, and bleeding persists. Hemophilia A affects one in every 5,000 males born in the United States. Bleeding occurs mostly into the joints, which can lead to severe damage.
Eloctate’s safety and efficacy were determined in a trial of 164 patients that compared the prophylactic treatment (prevention) regimen to on-demand therapy. The drug outperformed its competition substantially, improving preventive measures, treatment for current bleeding, and management for bleeding during and following surgical procedures.
The FDA issued Eloctate orphan drug classification. Orphan drugs are those that treat a rare medical condition, defined as occurring in 200,000 or fewer people in the U.S. Under the 1983 Orphan Drug Act, all orphan drugs may be sold free from competition for seven years. Eloctate is manufactured by Biogen Idec of Cambridge, Mass. Current treatment options for hemophilia A are limited to proteins derived from blood donations to replenish factor VIII.
“The proven ability of Eloctate to provide protection from bleeding episodes with prolonged circulation marks the first significant hemophilia A treatment advance in more than 20 years,” George Scangos, chief executive of Biogen Idec, told the pharma letter. “As a company deeply committed to improving the lives of people with hemophilia, we are excited to bring this important innovation to those living with hemophilia A.”