Biogen Idec has filed the first long-lasting Factor VIII treatment for haemophilia A with the US Food and Drug Administration.
The US biotech major has submitted recombinant factor VIII Fc fusion protein (rFVIIIFc), the first haemophilia A product candidate "in a new class of long-lasting clotting factor therapies being developed with the goal of reducing the burden of treatment for this condition". If approved, rFVIIIFc will be the first major advance in haemophilia A treatment in more than two decades, Biogen claims.
The regulatory submission is based on results from A-LONG, the largest Phase III study in haemophilia A to date. Glenn Pierce, Biogen's head of global medical affairs, noted that in that trial, patients were able to inject rFVIIIFc once-weekly to twice-weekly, "which creates the potential for those currently on prophylactic treatment to reduce injections by 50 to 100 per year". Moreover, patients currently treating bleeding episodes could potentially dose once per week "and maintain significant protection from bleeding with about the same total number of injections each year they use to treat bleeding episodes today", he added.
Earlier this month, the FDA accepted for review the company’s BLA for its factor IX candidate, rFIXFc, for use in patients with haemophilia B.