Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum (Sobi) (STO: SOBI) today announced positive results from A-LONG, a clinical study that evaluated a new long-lasting clotting factor candidate in people with hemophilia A. Hemophilia A is a rare inherited disorder that impairs blood coagulation.
Top-line results from A-LONG, a global, multi-center, Phase 3 clinical study of the companies’ long-lasting recombinant Factor VIII Fc fusion protein (rFVIIIFc), showed that rFVIIIFc was effective in the control and prevention of bleeding, routine prophylaxis and perioperative management. Recombinant FVIIIFc was generally well-tolerated. Additional analyses of the A-LONG study are ongoing, and the companies anticipate presenting detailed results at a future scientific meeting.
Biogen Idec plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2013. Consistent with guidelines published by the European Medicines Agency (EMA) that require a study in children less than 12 years of age prior to filing, Biogen Idec and Sobi expect to file a Marketing Authorization Application with the EMA upon completion of the ongoing Kids A-LONG study.
“These top-line 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,” said Glenn Pierce, M.D., Ph.D., Senior Vice President of Global Medical Affairs and Chief Medical Officer of Biogen Idec’s hemophilia therapeutic area. “We share the enthusiasm of the hemophilia community including the study participants and clinical investigators who supported the rapid enrollment of the A-LONG study. We are diligently working to prepare our regulatory submission with the goal of providing rFVIIIFc to people with hemophilia A as soon as possible.”“We are very encouraged by the positive A-LONG study results, which support the application of Fc fusion technology in hemophilia A to prolong factor activity and potentially offer extended protection from bleeding,” said Geoffrey McDonough, M.D., Chief Executive Officer of Sobi. “The A-LONG findings, coupled with recently announced results from the B-LONG study of our companies’ long-lasting recombinant Factor IX Fc fusion product candidate for hemophilia B, represent a major step forward for the hemophilia community.”