The US Food and Drug Administration (FDA) has accepted Biogen Idec's Biologics License Application (BLA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of haemophilia B and granted the company a standard review timeline. Recombinant FIXFc is the first product candidate in a new class of long-lasting clotting factor therapies being developed with the goal of reducing the burden of treatment for haemophilia B.

“We are encouraged by the FDA’s acceptance of our application, as we believe rFIXFc has the potential to transform the care of haemophilia B by allowing for less frequent injections and helping patients to maintain low annualized bleeding rates,” said Glenn Pierce,  senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec’s haemophilia therapeutic area. “We are working with the FDA to bring the first major treatment advance for the haemophilia B community in 15 years.”

The regulatory submission was based on results from B-LONG, the largest registrational phase 3 clinical study in haemophilia B to date. The study showed that rFIXFc provides long-lasting protection from bleeding with fewer injections than are required with the current standard of care. The company’s BLA submission for rFVIIIFc for use in patients with haemophilia A is on track for filing during the first half of 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 haemorrhages. The Medical and Scientific Advisory Council of the National Haemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe haemophilia 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 commercialization in Europe including Russia, the Middle East and Northern Africa.

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