(By Balaseshan) Biogen Idec Inc. (NASDAQ: BIIB) said it has recently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B.
The company said rFIXFc is the first product candidate in a new class of long-lasting clotting factor therapies that are being developed with the goals of reducing the burden of treatment for this condition and enhancing protection from bleeding.
The regulatory submission was based on results from B-LONG, the largest registrational phase 3 clinical study in hemophilia B to date. Biogen Idec's BLA submission to the U.S. FDA for rFVIIIFc for use in patients with hemophilia A is on track for filing in the first half of 2013.
Biogen said rFIXFc is a clotting factor developed using the company's monomeric Fc fusion technology, 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.
With this technology, rFIXFc is designed to provide long-lasting protection from bleeding and reduce the treatment burden associated with hemophilia B, which currently can require more than 100 injections annually for prophylaxis with commercially-available factor IX products.
The clinical efficacy and safety data for rFIXFc were robust and showed that dosing every one to two weeks provided significant protection from bleeding.
"Based on these data, we believe rFIXFc has the potential to have a major impact on the global adoption of prophylactic treatment in hemophilia B, helping patients prevent bleeding episodes and potentially reduce their long-term consequences," said Glenn Pierce, senior vice president of Global Medical Affairs and chief medical officer of Biogen's hemophilia therapeutic area.
Hemophilia B is a rare, inherited disorder in which the ability of a person's blood to clot is impaired. Hemophilia B occurs in about one in 25,000 male births annually and is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting.
Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of rFIXFc in hemophilia B and rFVIIIFc in hemophilia A.
BIIB closed Thursday's regular session at $147.86. The stock has been trading between $113.25 and $157.18 for the past 52 weeks.