FDA Approves Biogen Idec's Eloctate, First Hemophilia A Therapy to Extend the Interval between Prophylactic Infusions, for Both Adults and Children

Biogen Idec has announced that FDA has approved Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia A.

Eloctate is the first recombinant hemophilia A therapy with prolonged circulation in the body. It is the only treatment for hemophilia A to reduce the frequency of bleeding episodes with prophylactic infusions every 3–5 days, offering people with hemophilia A the potential to extend the interval between prophylactic infusions.

“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,” said George A. Scangos, PhD, chief executive officer of Biogen Idec. “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.”

The recommended starting prophylactic regimen for Eloctate is 50 IU/kg every four days. Based on clinical response, the regimen may be adjusted in the range of 25 to 65 IU/kg and every 3–5 days.
In clinical trials, Eloctate was effective for both routine prophylaxis and to treat acute bleeding episodes with a favorable safety and tolerability profile. It was developed using a process called Fc fusion, which is designed to prolong the therapy’s circulation in the body using a natural pathway. The FDA’s approval is the first regulatory approval worldwide for Eloctate, and the therapy is currently under review by regulatory authorities in several other countries, including Canada, Australia and Japan. This FDA action follows regulatory approvals of Biogen Idec’s hemophilia B therapy, Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein], in the US, Canada and Australia.

“Prophylactic treatment is recommended for people with severe hemophilia, and following a protective regimen can be burdensome given the frequency of infusions required,” said Patrick F. Fogarty, MD[1], 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.”

Therapies for hemophilia A, the most common form of hemophilia, can be administered either on a schedule to help prevent or reduce bleeding episodes (prophylaxis), or to control bleeding when it occurs (on-demand). The NHF recommends routine prophylaxis as optimal for the treatment of people with severe hemophilia. In recent years, regimens have shifted from on-demand treatment to routine prophylaxis because of observed improvement in long-term clinical outcomes, such as joint damage.

“We are encouraged by the arrival of new and innovative therapies, which may help address treatment gaps for the approximately 16,000 adults and children living with hemophilia A in the United States,” said Val Bias, chief executive officer of the NHF. “These advances, in conjunction with continued community education and empowerment, are critical to effectively serving the needs of people with the disorder.”

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[1] Editor’s Note: Dr Fogarty has received researched support, and fees for consulting, from Biogen Idec.