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UPDATED: Biogen races to FDA with positive PhIII data for hemophilia B drug

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Seeking to break into the market for bleeding-disorder drugs, Biogen Idec ($BIIB) and partner Swedish Orphan Biovitrum (Sobi) reported positive top-line data from a late-stage study of a long-lasting clotting factor treatment for patients with hemophilia B. With the key data in hand, Biogen said this morning that it plans to move forward with an application for approval of the treatment in the first half of 2013.

The pivotal B-LONG study demonstrated that median annualized bleeding rates of patients with the rare disorder who took the long-lasting recombinant Factor IX Fc fusion protein (rFIXFc) as protective treatment were in the low-single digits, with a 2.95 occurrence rate in patients in the weekly prophylaxis arm and 1.38 in an arm in which the median dosing interval during the last 6 months of the study was 14 days, the companies reported. The rate was much higher, 17.69, in patients in the episodic treatment arm. More than 90% of bleeding incidents in patients were reined in with a single shot of rFIXFc, and none of the patients in the study developed antibodies against the treatment.

There was a single serious adverse event in the trial that could be related to the experimental treatment, Biogen and Sobi said, with one patient experiencing problems associated with blood found in his urine. But he continued treatment with medical attention. Other common adverse events in the study included nasopharyngitis, influenza, joint pain, upper respiratory infection, hypertension and headache.

The long-lasting clotting factor offers the possibility of reducing injections of factor IX from two to three times a week with available treatments to every two weeks for some patients, which could bring a major change in the treatment of hemophilia B, Dr. Glenn Pierce, the medical chief of Biogen's hemophilia unit, told FierceBiotech in an interview.

"The idea that one may be able to have a much longer interval between treatments and have very good protection from bleeding really changes the potential for how hemophilia B can be treated," Pierce said. "In that sense, the results are really encouraging. It changes the paradigm for hemophilia B treatment."

Biogen, one of the largest makers of multiple sclerosis drugs, also has a long-acting hemophilia A treatment in late-stage development that the company expects will yield data later this year. The two treatments provide the company with a shot to break into the market for the bleeding disorders, for which the company has no existing products on the market. Weston, MA-based Biogen has been beefing up its hemophilia business in recent years under CEO George Scangos, who spared the bleeding-disorder programs in a series of R&D cutbacks in 2010 and 2011 that involved the company's exit from cancer drug research. Pierce says that he came to Biogen from Bayer's U.S. hemophilia research group, and others have joined the biotech with deep experience in hemophilia.

- here's the release
- see Bloomberg's article

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