In Major Advance, Biogen Submits BLA to FDA for Hemophilia A Treatment

Biogen Idec announced that it has submitted a Biologics License Application (BLA) to the FDA for the marketing approval of recombinant factor VIII Fc fusion protein (rFVIIIFc) for the treatment of hemophilia A. Recombinant FVIIIFc is the first hemophilia A product candidate in a new class of long-lasting clotting factor therapies which makes use of a naturally occurring pathway that delays the destruction of factor and cycles it back into the bloodstream, resulting in a longer circulating half-life.

Approval of rFVIIIFc will make it the first major advance in hemophilia A treatment in more than two decades. Regulatory submission for rFVIIIFc was based on results from A-LONG, the largest registrational phase 3 clinical study in hemophilia A.

Prophylaxis in hemophilia A typically requires injections three times a week or every other day to maintain adequate circulating clotting factor levels to provide protection from bleeding. Patients with hemophilia A can require approximately 150–180 injections annually for prophylaxis with commercially available factor VIII products. Without prophylactic treatment, hemophilia A patients remain at risk for bleeding episodes which can cause irreversible joint damage and life threatening hemorrhages. Recombinant FVIIIFc is designed to provide long-lasting protection from bleeding and reduce the treatment burden associated with hemophilia A.

On March 4, 2013, Biogen Idec announced the FDA had accepted for review the company's BLA for its factor IX candidate, rFIXFc, for use in patients with hemophilia B.

For more information visit www.biogenidec.com.