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FDA Approval Sought for Hemophilia Therapy

Recombinant fusion protein would be first major advance in more than 20 years (Mar. 12)

A biologics license application (BLA) has been submitted to the FDA for the marketing approval of a recombinant factor VIII Fc fusion protein (rFVIIIFc, Biogen Idec) for the treatment of hemophilia A. If approved, rFVIIIFc would be the first major advance in the treatment of hemophilia A in more than two decades. The regulatory submission was based on results from the phase III A-LONG trial.

Typically, prophylaxis in hemophilia A requires injections three times per week or every other day to maintain a sufficient circulating level of clotting factor to provide protection from bleeding. Without prophylactic treatment, patients with hemophilia A remain at risk of bleeding episodes that can cause irreversible joint damage and life-threatening hemorrhages.

Recombinant FVIIIFc is a clotting factor developed using a monomeric Fc fusion technology, which delays the factor’s destruction and cycles it back into the bloodstream, resulting in a longer circulating half-life. The prolonged half-life of rFVIIIFc may allow prophylactic dosing once or twice weekly.

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.

Hemophilia A is a rare, inherited disorder in which the ability of a person’s blood to clot is impaired. Hemophilia A occurs in about one in 5,000 male births annually and is caused by having substantially reduced or no factor VIII activity, which is needed for normal blood clotting. People with hemophilia A therefore need injections of factor VIII to restore the coagulation process and to prevent frequent bleeds. The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia A.

Source: Biogen Idec; March 12, 2013.

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