Partners Biogen Idec and Swedish Orphan Biovitrum (Sobi) reported positive data from a Phase III study evaluating the long-lasting recombinant Factor IX Fc fusion protein (rFIXFc) in hemophilia B patients. Biogen Idec says it now plans to submit a BLA application to FDA during the first half of 2013. A European filing will first require completion of the ongoing pediatric trial, as EMEA guidelines require a study to be completed in children aged under 12 years prior to filing.
The B-Long trial included 123 male patients aged 12 years and over, and was designed with four treatment arms: weekly prophylaxis, individualized interval prophylaxis, episodic treatment, and perioperative management. The results demonstrated that rFIXFc was effective for routine prophylaxis and perioperative management, as well as for the control and prevention of bleeding.
More specifically, the overall median annualized bleeding rates (including spontaneous and traumatic bleeds) were 2.95 in the weekly prophylaxis arm, 1.38 in the individualized interval prophylaxis arm, and 17.69 in the episodic treatment arm. In the individualized interval prophylaxis arm the median dosing interval during the last six months of the study was 14 days.
“Our companies are pioneering the application of Fc fusion technology to exend the half-life of clotting factors,” remarks Geoffrey McDonough, M.D., Sobi CEO. “This approach holds promise for combining more consistent protection with fewer injections. The B-Long study results are highly encouraging and support the potential use of this technology in hemophilia B.”
Biogen Idec and Sobi are jointly developing both the rFIXFc clotting factor and a long-lasting recombinant Factor VIII FC fusion protein (rFVIIIFc) for hemophilia A patients, which is also in Phase III development. Topline data from the A-Long rFVIIIFc study are expected during 2012. Under terms of the partnership Biogen Idec is heading product development and retains manufacturing rights. Sobi has the right to opt in and take over final development and commercialization of the products in Europe (including Russia), the Middle East and North Africa. Biogen Idec retains all commercialization rights in North America and all other regions outside of Sobi’s territories.
The firm acquired the Fc Fusion technology platform through its buyout of Syntonix back in 2007. The technology has already been applied to the development of seven FDA approved products for the long-term treatment of chronic diseases including rheumatoid arthritis, psoriasis, and platelet disorders.