Biogen asks FDA for approval of first drug in potential $1B franchise

Biogen Idec ($BIIB) has shipped its application to the FDA for approval of an experimental therapy for the rare bleeding disorder hemophilia B. And the long-lasting clotting factor is the most advanced candidate in what could become the Weston, MA-based biotech giant's next blockbuster business.

Biogen, the world's leading provider of multiple sclerosis drugs, lacks a marketed product in the multibillion-dollar hemophilia business. Yet the company boasts a pair of candidates that succeeded in pivotal trials last year for treating both hemophilia B, a factor IX deficiency, and the more common hemophilia A. Its long-lasting factor IX therapy (rFIXFc)--partnered with the Swedish Orphan Biovitrum or Sobi--gave patients the option of getting dosed every one or two weeks, as opposed to two or three times per week with existing treatments such as Pfizer's ($PFE) Benefix.

The hemophilia business has the potential to become a significant moneymaker for Biogen, which was dealt a blow in its late-stage pipeline yesterday with news about the failure of a Phase III study for its experimental ALS treatment. Based on enthusiasm about Biogen's hemophilia pipeline, Cowen analysts in November projected that the franchise would bring in $1 billion by 2020. Biogen CEO George Scangos bet on hemophilia becoming a growth area after he decided to keep the program in a major pipeline review in 2010 that included an exit from oncology research.

Dr. Glenn Pierce, the medical chief of Biogen's hemophilia area, has told FierceBiotech that the company has made inroads with the tight-knit hemophilia community and believes that the company's lead recombinant therapies have the potential to significantly advance treatment for patients with the bleeding disorders.

"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 hemophilia B community in 15 years," Pierce said in a statement today.

Biogen aims to follow the submission of the hemophilia B candidate with its hemophilia A contender in the first half of 2013.

- here's the release

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