Biogen's hemophilia hopeful scores a PhIII win as FDA awaits

Biogen Idec's ($BIIB) long-acting hemophilia A drug performed well in a late-stage study on children, the biotech said, giving it another Phase III victory as it races with rivals Bayer and Novo Nordisk ($NVO) to launch a more convenient treatment for the bleeding disorder.

In top-line results from a study on 71 boys with severe cases of hemophilia A, the most common form of the disease, Biogen's Eloctate led to low instances of bleeding on twice-a-week dosing, the company said. Patients posted a median annualized bleed rate of 2.0, and 93% of bleeding episodes were controlled by one to two doses of the drug, according to Biogen. On the safety side, the trial met its primary endpoint as no patients developed inhibitors to Eloctate, and Biogen said the injected treatment was well-tolerated.

The drug, developed alongside Swedish Orphan Biovitrum (Sobi), is a factor VIII replacement therapy that works by attaching the missing coagulation link to a common bodily protein, allowing for a longer-lasting effect and less-frequent dosing. That's especially important for children who need regular prophylactic therapy, Biogen Hematology Vice President Glenn Pierce said, as daily injections can present a serious challenge.

The pediatric trial is a European Medicines Agency condition for approval, and results from the Phase III study aren't included in Biogen's pending application with the FDA, though it'll likely come in handy when the biotech seeks a broader indication for Eloctate.

Meanwhile, Biogen is working to get its long-lasting treatment on the market before competing offerings from Bayer and Novo Nordisk. The Cambridge, MA, biotech is widely considered to lead the pack despite a November FDA delay, and Biogen has said it expects to win approval and launch Eloctate in the second half of this year.

Lagging behind is Bayer and its BAY94-9027, which has performed well in weekly and twice-a-week dosing, followed by Novo's N8-GP, for which Phase III results should be available in the next 12 months. The whole class of extended-half-life treatments is expected to unseat reigning offerings from Baxter ($BAX) and Pfizer ($PFE), and Morningstar figures the market for A and B drugs to reach $11.4 billion by 2016.

Eloctate is part of Biogen's two-pronged pipeline of long-acting hemophilia drugs, and the B-treating Alprolix won FDA approval late last month.

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