DEERFIELD, Ill., September 4, 2012 - Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a biologics license application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for approval of BAX 326, a recombinant factor IX (rFIX) protein being investigated for the treatment and prophylaxis of bleeding episodes for patients over 12 years of age with hemophilia B.
Hemophilia B, also known as Christmas disease, is the second most common type of hemophilia and results from insufficient amounts of clotting factor IX, a naturally occurring protein in blood that helps to control bleeding.1 Approximately 25,000 people worldwide, including more than 4,000 in the U.S., have been diagnosed with hemophilia B.2
The BLA filing is based on results from a global Phase III study conducted in 10 countries around the world. The prospective, controlled, multicenter study evaluated the pharmacokinetics, efficacy, safety and immunogenicity of BAX 326 in 73 patients with severe or moderately severe hemophilia B previously treated with other factor IX therapy. The study met its primary objectives and the company plans to present the complete data from the study in late 2012. Baxter expects to file its application for BAX 326 in Europe in 2013.
"Hemophilia B patients have relatively limited options for their treatment today, with only one commercially available recombinant (genetically engineered) protein. As part of our long-standing commitment to the hemophilia community, we continue to pursue new potential treatment options like BAX 326 to support patients with this debilitating disease," said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter's BioScience business.
In select countries, Baxter currently offers a plasma-derived factor IX treatment, Immunine [Factor IX Concentrate (Human)], for patients with hemophilia B, which has more than 16 years of patient experience in Europe and Latin America. In addition, Baxter recently announced a partnership with Chatham Therapeutics, LLC to develop a gene therapy based treatment for hemophilia B. Gene therapy could represent another important first for the community as an innovative potential therapy for hemophilia B treatment.
About Baxter in Hemophilia
With more than 60 years experience in hemophilia, Baxter has introduced a number of therapeutic firsts for people with hemophilia. With the broadest portfolio of hemophilia treatments in the industry, Baxter is able to meet individual therapy choices, providing a range of options at each treatment stage. The company is dedicated to optimizing hemophilia care and improving the lives of people living with hemophilia A and B worldwide.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.
This release includes forward-looking statements concerning potential treatments for hemophilia B, including anticipated Baxter regulatory filings. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: actions of regulatory bodies and other governmental authorities; satisfaction of regulatory approval and other requirements; and other risks identified in Baxter's most recent filing on Form 10-K and other SEC filings, all of which are available on the company's website. Baxter does not undertake to update its forward-looking statements.
1Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on April 20, 2012. Available at: http://www.wfh.org/en/page.aspx?pid=637
2World Federation of Hemophilia Report on the Annual Global Survey 2010. World Federation of Hemophilia. Accessed on April 20, 2012. Available at: http://www1.wfh.org/publications/files/pdf-1427.pdf