A day after ending development of its late-stage Lou Gehrig’s disease drug dexpramipexole, Biogen Idec has been boosted by the filing of a new haemophilia B treatment with regulators in the USA.
The company revealed it has recently submitted a Biologics License Application to the US Food and Drug Administration for its recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of haemophilia B. The therapy, which has been developed with Swedish Orphan Biovitrum (Sobi), is the first candidate in a new class of long-lasting clotting factor therapies that are being developed to reduce the burden of treatment for the condition "and enhancing protection from bleeding".
The submission is based on results from B-LONG, which Biogen says is the largest Phase III study in haemophilia B to date. Chief medical officer Glenn Pierce said the filing 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”. The rare, inherited disorder in which the ability of a person's blood to clot is impaired, occurs in about one in 25,000 male births annually.
The US biotech major also noted that a BLA submission for a long-lasting recombinant Factor VIII Fc fusion protein (rFVIIIFc) for haemophilia A, also developed with Sobi, is on track for filing in the first half of this year. The latter is estimated to be four times the size of the haemophilia B market.