Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for approval of RIXUBIS, recombinant factor IX (nonacog gamma) for the treatment and prophylaxis of bleeding in patients of all ages 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 RIXUBIS was approved for adult patients and launched in the United States and Puerto Rico earlier in 2013. The filing is based on results of research with hemophilia B patients previously treated with other therapies. A global Phase I/III study of adult patients with severe or moderately severe hemophilia B demonstrated that twice-weekly prophylactic treatment with RIXUBIS for six months achieved a median annualized bleed rate (ABR) of 2.0, with 43 percent (24/56) of patients experiencing no bleeds. In the study, no patients developed an inhibitory antibody to FIX and no cases of anaphylaxis were reported. The most common adverse reactions observed in >1% of subjects of this study were dysgeusia, and pain in extremity. The application is further supported by data from a study of 23 hemophilia B patients less than twelve years old. 2 “Our focus at Baxter is to strengthen and expand the portfolio of treatments for patients with bleeding disorders, particularly when there are limited treatment options, such as for patients with hemophilia B,” said Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter’s BioScience business. “We are actively working to extend access to these treatments and look forward to introducing RIXUBIS in Europe and other countries around the world.” Current Indications for RIXUBIS In the United States, RIXUBIS [Coagulation Factor IX (Recombinant)] is an antihemophilic factor indicated for:
- Control and prevention of bleeding episodes in adults with hemophilia B.
- Perioperative management in adults with hemophilia B.
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia B.
Important Risk Information for RIXUBISRIXUBIS is contraindicated in patients who have:
- Known hypersensitivity to RIXUBIS or its excipients including hamster protein
- Disseminated intravascular coagulation (DIC)
- Signs of fibrinolysis
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 RIXUBIS, including expectations with regard to 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: satisfaction of regulatory approval and other requirements; actions of regulatory bodies and other governmental authorities; product quality or patient safety issues; changes in law and regulations; and other risks identified in Baxter's most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter's website. Baxter does not undertake to update its forward-looking statements. 1 Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on April 20, 2012 Available at: http://www.wfh.org/index.asp?lang=EN 2 Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level ≤2%) haemophilia B. Haemophilia. 2013 Jul 9.; pediatric study not yet published 3 Types of Bleeding Disorders: Hemophilia B. National Hemophilia Foundation. Accessed on May 10, 2013. Available at: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=181&contentid=46&rptname=bleeding 4 2011 Annual Global Survey. World Federation of Hemophilia. Accessed on May 10, 2013. Available at: http://www1.wfh.org/publications/files/pdf-1488.pdf 5 Lee-Rodríguez-Merchán, E.-C. and Valentino, L. A. (2011) New Developments in Hemophilic Arthropathy, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford, UK. doi: 10.1002/9781119979401.ch29