Biogen Idec and Swedish Orphan Biovitrum announced Phase 1/2a trial data showing that the companies’ long-lasting fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) was well tolerated and demonstrated an approximately 1.7-fold increase in half-life compared with Advate (antihemophilic factor recombinant, plasma/albumin-free method, rFVIII), a commercially-available factor VIII product, in 16 previously-treated patients with severe hemophilia A. The findings, which were seen consistently across all patients and dose levels, are being presented today at the XXIIIrd Congress of the International Society on Thrombosis and Haemostasis in Kyoto, Japan.
“This trial is a step toward addressing the significant unmet need for a long-lasting recombinant factor VIII product,” said Neil Josephson, M.D., Co-Principal Investigator of the Phase 3 rFVIIIFc A-LONG trial and associate professor of Medicine in the Division of Hematology at the University of Washington School of Medicine, Seattle, Wash. “Results from the Phase 1/2a study show that rFVIIIFc has an extended half-life, which may have the potential to provide extended protection from bleeding and reduce the number of infusions necessary for prophylactic treatment of severe hemophilia A.”
Currently, prophylactic treatment of severe hemophilia A requires intravenous infusions three times a week or every other day. rFVIIIFc is a fully-recombinant and fully-active clotting factor designed to replace the protein that hemophilia A patients lack and to last longer in the body than commercially-available factor VIII products. Developed using Biogen Idec’s proprietary Fc fusion technology, rFVIIIFc utilizes a natural pathway that recycles rFVIIIFc in the circulation to extend its half-life.
“Biogen Idec is driven to deliver innovative treatments that can make much-needed progress for people with hemophilia,” said Glenn Pierce, M.D., Ph.D., Senior Vice President of Hemophilia at Biogen Idec. “These study results demonstrate the potential of our Fc fusion technology to develop long-lasting clotting factors that may significantly reduce the burden of treatment and improve quality of life for people with hemophilia.”
“These results are promising and supported the advancement of rFVIIIFc into a Phase 3 trial last year,” said Peter Edman, Ph.D., Chief Scientific Officer of Swedish Orphan Biovitrum. “We are excited about the potential of rFVIIIFc to make a positive impact on the health and quality of life of hemophilia A patients by providing extended protection from bleeding.”
rFVIIIFc is currently being studied in a registrational, open-label, multicenter trial (A-LONG), which is evaluating its safety, pharmacokinetics and efficacy in the prevention and treatment of bleeding in previously-treated patients with severe hemophilia A.
Additionally, the European Medicines Agency’s (EMA) Pediatric Committee recently adopted an opinion agreeing to the pediatric investigational plan for rFVIIIFc. In accordance with the opinion, Biogen Idec and Swedish Orphan Biovitrum plan to initiate a global pediatric trial in previously-treated patients under 12 years of age as soon as sufficient data are available from a study of older patients. Under draft guidelines published by the EMA for the development of factor VIII products, pediatric data from this trial will be required in the initial submission of a Marketing Authorization Application to the European regulatory agency.
Date: July 26, 2011
Source: Biogen Idec
Filed Under: Drug Discovery