US approves Biogen Idec treatment for one type of hemophilia

Associate scientist Kate Henry at work in the immunology lab at Biogen.
Associate scientist Kate Henry at work in the immunology lab at Biogen.

Federal regulators Friday approved a long-acting Biogen Idec drug as the first new hemophilia treatment in 17 years, launching a new class of therapies to control and prevent bleeding in people with hemophilia and opening a potentially lucrative new market for Biogen Idec, the largest biotechnology company in Massachusetts.

The drug, called Alprolix, was okayed by the US Food and Drug Administration specifically for adults and children with hemophilia B, one type of the disease. Biogen Idec, based in Cambridge, is expecting an FDA decision by mid-year on an experimental drug based on the same mechanism of action to treat the more common hemophilia A. An estimated 4,000 people, mostly men, suffer from hemophila B in the United States, while about 16,000 are affected by hemophilia A.

“There’s a big need here,” said Tony Kingsley, executive vice president of global commercial operations at Biogen Idec. “Patients need a life time of treatment. And the ability to reduce the frequency of treatments is the biggest unmet need in this area short of a cure.”

Hemophilia B is a rare inherited disorder that prevents normal blood clotting in patients, leading to recurrent and extended bleeding that can cause pain and joint damage. The newly approved Alprolix can be given through infusions at home once a week or once every two weeks, compared to current medicines that patients have to inject two or three times a week.

Up until now, Biogen Idec’s drug portfolio has consisted entirely of treatments for the neurodegenerative disease multiple sclerosis, though it continues to receive a revenue stream from Rituxan, a cancer drug it once co-marketed with Swiss drug maker Roche AG. The approval of Alprolix marks Biogen Idec’s entry into a new disease area and the beginning of a diversification push for the company that is now the largest Massachusett-based biotech.

The global market for treatment of both types of hemophila has been estimated at $7 billion annually, but Biogen Idec would have to compete against drugs already on the market, including a Pfizer Inc. treatment originally developed by the former Cambridge biotech Genetics Institute Inc. and potentially against other longer-acting drug candidates being tested by competitors.

Approval of Aprolix in the United States comes a week after Health Canada, that nation’s drug regulatory agency, became the first in the world to give the new hemophila drug the green light. Biogen Idec has also filed applications for approval with regulators in Japan and Australia.

Rob Weisman covers life sciences and healthcare for the Boston Globe. Email him at
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