Biogen Idec has submitted its investigational drug for the bleeding disorder haemophilia B to the US Food and Drug Administration for marketing approval.
If given the green light by the regulator, recombinant factor IX Fc fusion protein (rFIXFc) would be Biogen’s first step into haemophilia and the first drug brought to market through its partnership with Swedish Orphan Biovitrum (Sobi).
This collaboration also involves research into the experimental protein rFVIIIFc, which is in development for haemophilia A – the most common subset of the bleeding disorder.
The haemophilia B market is still a sizable one, however, and Biogen will be looking to rFIXFc to make it through the FDA’s approval process to help compensate for last week’s decision to halt development of amyotrophic lateral sclerosis treatment dexpramipexole.
Biogen expects to file haemophilia A drug rFVIIIFc during the first half of 2013, and if both products make it to market, analysts have said they expect Biogen’s haemophilia presence to be worth $1bn annually.
“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 Dr Glenn Pierce, senior VP of global medical affairs and chief medical officer of Biogen’s haemophilia therapeutic area.
Backing the submission of rFIXFc are results from B-LONG, which Biogen says is the largest phase III clinical study in haemophilia B to date.
This study demonstrated that rFIXFc was both clinically effective and safe, helping to prevent bleeding episodes and reduce long-term consequences.
The drug’s dosing regimen of once every one to two weeks also gives it a significant advantage over currently available products that need to be taken two or three times per week.
Biogen holds the manufacturing and commercialisation rights in North America for both haemophilia products under the terms of its deal with Sobi.
Elsewhere, the Swedish company retains the right to takeover final development and commercialisation of the products in Europe including Russia, the Middle East and Northern Africa.