Biogen Idec, a compnay discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, haemophilia and autoimmune disorders, recently submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of haemophilia B.

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 III clinical study in haemophilia B to date.

“The submission to FDA of rFIXFc is an important milestone towards the introduction of a long-lasting factor IX therapy which is the first major treatment advance for the haemophilia B community in 15 years,” said Glenn Pierce, MD, Ph.D., senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec’s hemophilia therapeutic area. “Our Fc fusion technology uses a natural pathway in the body to keep factor in circulation longer. 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 haemophilia B, helping patients prevent bleeding episodes and potentially reduce their long-term consequences.”

Biogen Idec’s BLA submission to the US FDA for rFVIIIFc for use in patients with haemophilia A is on track for filing in 1H 2013.

rFIXFc is a clotting factor developed using Biogen Idec’s novel and proprietary 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 haemophilia B, which currently can require more than 100 injections annually for prophylaxis with commercially-available factor IX products. Fc fusion technology is used in seven FDA-approved products for the long-term treatment of chronic diseases including rheumatoid arthritis, psoriasis and platelet disorders.

Haemophilia B is a rare, inherited disorder in which the ability of a person's blood to clot is impaired. Haemophilia 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. People with haemophilia B therefore need injections of factor IX to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages. The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia B. Currently, prophylaxis in haemophilia B typically requires injections up to three times per week to maintain a sufficient circulating level of clotting factor.

Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of rFIXFc in haemophilia B and rFVIIIFc in haemophilia A. Biogen Idec leads development, has manufacturing rights, and has commercialization rights in North America and all other regions excluding the Sobi territory. Sobi has the right to opt in to assume final development and commercialisation in Europe including Russia, the Middle East and Northern Africa.