WESTON, Mass.– Today,Â Biogen IdecÂ announced the company recently submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B. rFIXFc is the first product candidate in a new class of long-lasting clotting factor therapies that are being developed with the goals of reducing the burden of treatment for this condition and enhancing protection from bleeding. The regulatory submission was based on results from B-LONG, the largest registrational phase 3 clinical study in hemophilia B to date.
“The submission to FDA of rFIXFc is an important milestone towards the introduction of a long-lasting factor IX therapy which is the first major treatment advance for the hemophilia B community in 15 years,” 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. “Our Fc fusion technology uses a natural pathway in the body to keep factor in circulation longer. The clinical efficacy and safety data for rFIXFc were robust and showed that dosing every one to two weeks provided significant protection from bleeding. Based on these data, we believe rFIXFc has the potential to have a major impact on the global adoption of prophylactic treatment in hemophilia B, helping patients prevent bleeding episodes and potentially reduce their long-term consequences.”
Biogen Idec’s BLA submission to the U.S. FDA for rFVIIIFc for use in patients with hemophilia A is on track for filing in 1H 2013.
About the Fc Fusion Technology Platform
rFIXFc is a clotting factor developed using Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which makes use of a naturally occurring pathway that delays the destruction of factor and cycles it back into the bloodstream, resulting in a longer circulating half-life.
With this technology, rFIXFc is designed to provide long-lasting protection from bleeding and reduce the treatment burden associated with hemophilia B, which currently can require more than 100 injections annually for prophylaxis with commercially-available factor IX products. Fc fusion technology is used in seven FDA-approved products for the long-term treatment of chronic diseases including rheumatoid arthritis, psoriasis and platelet disorders.
About Hemophilia B
Hemophilia B is a rare, inherited disorder in which the ability of a person’s blood to clot is impaired. Hemophilia B occurs in about one in 25,000 male births annually and is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting. People with hemophilia B therefore need injections of factor IX to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages. The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia B. Currently, prophylaxis in hemophilia B typically requires injections up to three times per week to maintain a sufficient circulating level of clotting factor.
About the Biogen Idec and Sobi Collaboration
Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of rFIXFc in hemophilia B and rFVIIIFc in hemophilia A. Biogen Idec leads development, has manufacturing rights, and has commercialization rights in North America and all other regions excluding the Sobi territory. Sobi has the right to opt in to assume final development and commercialization in Europe including Russia, the Middle East and Northern Africa.
AboutÂ Biogen Idec
Through cutting-edge science and medicine,Â Biogen IdecÂ discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hemophilia and autoimmune disorders. Founded in 1978,Â Biogen IdecÂ is the world’s oldest independent biotechnology company. Patients worldwide benefit from its leading multiple sclerosis therapies, and the company generates more thanÂ $5 billionÂ in annual revenues. For product labeling, press releases and additional information about the company, please visit www.biogenidec.com.
This press release contains forward-looking statements, including statements about the commercialization and impact of long-lasting hemophilia therapies and regulatory filings. These statements may be identified by words such as “believe,” “expect,” “may,” “plan,” “potential,” “will” and similar expressions, and are based on our current beliefs and expectations. Drug development and commercialization involve a high degree of risk. Factors which could cause actual results to differ materially from our current expectations include the risk that unexpected concerns may arise from additional data or analysis, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates, or we may encounter other unexpected hurdles. For more detailed information on the risks and uncertainties associated with our drug development and commercialization activities, please review the Risk Factors section of our most recent annual or quarterly report filed with the Securities and Exchange Commission. Any forward-looking statements speak only as of the date of this press release and we assume no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.