FDA approves Biogen hemophilia drug
Two months after US regulators approved the first new hemophilia treatment in 17 years, its maker, Biogen Idec Inc., got the okay Friday to market a similarly long-acting drug that controls and prevents bleeding in adults and children with the most common form of the disease.
Food and Drug Administration approval of Eloctate opens a new market for Cambridge-based Biogen Idec among an estimated 16,000 people in the United States with hemophilia A. The inherited disorder prevents normal blood clotting in patients, leading to recurrent and extended bleeding that can cause pain and joint damage.
"The approval of this product provides an additional therapeutic option for use in the care of patients with hemophilia A," said Dr. Karen Midthun, director of the FDA's Center for Biologics Evaluation and Research.
About four times as many people suffer from hemophilia A than from hemophilia B. Biogen Idec won FDA approval on March 28 to sell another drug, called Alprolix, based on the same mechanism of action as Eloctate, to treat hemophilia B.
"The innovation here is very similar to the innovation we brought forward with Alprolix," said Doug Williams, executive vice president of research and development at Biogen Idec.
Most hemophilia A patients currently inject themselves at home with an earlier class of therapies once every two to three days. With the new longer-acting Biogen Idec treatment, doctors are recommending patient injections about once every four or more days. "Reducing the frequency of these infusions is a big benefit to patients," Williams said.
Tony Kingsley, executive vice president of global commercial operations at Biogen Idec, said the company is targeting two segments of patients: those who already use existing drugs to prevent bleeding and those who currently don't take preventive therapies but only treat their symptoms "on demand" when they are bleeding. The less frequent dosages required by Eloctate make it easier for patients to begin such "prophylactic" treatment, he said.
Before the hemophilia drug approvals, Biogen Idec's portfolio consisted entirely of drugs to treat the neurodegenerative disease multiple sclerosis, though it continues to receive revenue from Rituxan, a cancer drug it once co-marketed with Swiss drug maker Roche AG. The approval of Alprolix and Eloctate mark Biogen Idec's entry into a new disease area and the beginning of a diversification push for the largest biotech company based in Massachusetts.
The global market for both types of hemophilia has been estimated at $7 billion annually, but Biogen Idec would have to compete against drugs already on the market, including a Pfizer Inc. medicine originally developed by the former Cambridge biotech Genetics Institute Inc. and -- potentially -- other longer-acting drugs now being tested by competitors.