Biogen Idec and Swedish Orphan Biovitrum (SOBI) say they expect to file a BLA with FDA for their long-acting hemophilia A therapy during the first half of 2013, on the back of positive results from a Phase III study, which showed the recombinant protein significantly reduced bleeding episodes. The A-Long trial evaluated the recombinant Factor VIII Fc fusion protein (rFVIIIFc) in 165 male hemophilia patients aged 12 years and over. Patients were divided into three treatment arms; individualized prophylaxis, weekly prophylaxis, and episodic (on demand) treatment.
The results showed that overall, 98% of bleeding episodes were controlled by one or two injections of rFVIIIFc. Median annualized bleeding rates (including spontaneous and traumatic bleeds), were 1.6 in the individualized prophylaxis arm, 3.6 in the weekly prophylaxis arm, and 33.6 in the episodic treatment arm. The median dosing interval in the individualized prophylaxis arm was 3.5 days.
“These topline results demonstrated that rFVIIIFc has the potential to enhance the care of people living with hemophilia A by offering protection from bleeding with reduced treatment burden,” comments Glen Pierce, M.D., svp for global medical affairs and CMO at Biogen Idec’s hemophilia therapeutic area. “We are diligently working to prepare our regulatory submission with the goal of providing rFVIIIFc to people with hemophilia A as soon as possible.”
rFVIIIFc is also being evaluated in the open-label Phase III Kids A-Long study in previously treated hemophilia A patients under 12 years of age. Completion of this study will be required before Biogen Idec and SOBI can file for approval of the long-acting Factor VIII in the EU. An open-label, long-term study, Aspire, is also ongoing in patients who have either completed A-Long or Kids A-Long.
Biogen Idec and SOBI are partnering the development of rFVIIIFc and an rFIXFc product for treating hemophilia B. Biogen Idec is leading the development programs and retains manufacturing rights to the products, and also retains commercialization rights in North America and globally, excluding SOBI’s licensed territories of Europe, Russia, the Middle East, and North Africa. Both rFVIIIFc and rFIXFc have been developed using Biogen Idec’s monomeric Fc fusion technology. Positive data from the Phase III B-Long study evaluating rFIXFc in adult hemophilia patients was reported in September.