Baxter International Inc. has submitted a biologics licence application (BLA) to the United States 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 haemophilia B.
Haemophilia B, also known as Christmas disease, is the second most common type of haemophilia and results from insufficient amounts of clotting factor IX, a naturally occurring protein in blood that helps to control bleeding.
The BLA filing is based on results from a global phase III study conducted in 10 countries around the world. The prospective, controlled, multi-centre study evaluated the pharmacokinetics, efficacy, safety and immunogenicity of BAX 326 in 73 patients with severe or moderately severe haemophilia 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.
"Haemophilia 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 haemophilia community, we continue to pursue new potential treatment options like BAX 326 to support patients with this debilitating disease," said Prof. Hartmut J Ehrlich, MD, 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 haemophilia 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 haemophilia B. Gene therapy could represent another important first for the community as an innovative potential therapy for haemophilia B treatment.
With more than 60 years experience in haemophilia, Baxter has introduced a number of therapeutic firsts for people with haemophilia. With the broadest portfolio of haemophilia 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 haemophilia care and improving the lives of people living with haemophilia A and B worldwide.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with haemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions.