Biogen Idec Inc. and partner Swedish Orphan Biovitrum AB said their hemophilia A therapy controlled bleeding with fewer injections in a late-stage study. The companies plan to apply for U.S. approval early next year.
The therapy, long-lasting recombinant Factor VIII Fc fusion protein, or rFVIIIFc, yielded annual rates of 1.6 bleeds in a preventive treatment study arm in which patients received the drug about once every three and a half days. That compared with 3.6 bleeds for patients treated once a week, and 33.6 for patients that received the medicine as needed, the companies said in a statement today.
None of the patients in the study developed inhibitors, antibodies that stop the drug from working, and the treatment was generally well tolerated, the companies said. They plan to apply for Food and Drug Administration approval in the first half of 2013, they said.
The drug “has the potential to enhance the care of people living with hemophilia A by offering protection from bleeding with reduced treatment burden,” Glenn Pierce, chief medical officer for Biogen’s hemophilia therapeutic area, said in the statement.
Swedish Orphan rose 11 percent to 37.50 kronor at 5:30 p.m. in Stockholm, the biggest advance since Sept. 26. Biogen declined 3.3 percent to $138.21 at 4 p.m. New York time. The shares have gained 26 percent this year.
Hemophilia is an inherited blood disorder caused by a low levels or absence of a protein essential for blood-clotting. In the first study arm, which treated patients on an individualized basis to prevent bleeds, 30 percent of people began being treated about once every five days in the last three months, from once every three and a half previously, Biogen said.
“This is perhaps a bit below our expectations of about 40 percent to 70 percent plus that earlier data had predicted,” Mark Schoenebaum, an analyst with ISI Group in New York, wrote in a note to clients today. “The annualized bleeding rate in this arm was stellar at 1.6 (well below historical averages). In addition, the weekly dosing data look very good.”
The half-life of Biogen and Swedish Orphan’s drug was 19 hours, compared with 12.4 hours for Baxter International Inc.’s Advate, a therapy that leads the $5 billion market for hemophilia A recombinant treatments, according to research from Barclays Capital.
“We would expect Biogen to pound the message that ‘whatever Baxter can do, we can do 1.5x better,’” Schoenebaum wrote.
Biogen and Swedish Orphan’s drug may draw $306 million in annual revenue by 2016, according to Brian Abrahams, an analyst with Wells Fargo.
The companies reported data last month on their therapy for hemophilia B, a bleeding disorder that affects a smaller number of patients. The hemophilia A market is about four times the size of that for hemophilia B, according to Eric Schmidt, an analyst with Cowen & Co.
Those data showed Biogen and Swedish Orphan’s drug was well-tolerated and enabled patients to be treated less frequently.