BAX 326 Granted Orphan-drug Designation by U.S. Food and Drug Administration
Baxter Presents Phase I/III Data Evaluating Safety and Efficacy of BAX 326, an Investigational Recombinant Factor IX for Hemophilia B, Showing More Than 40 Percent of Patients Experienced No Bleeds
<0> Baxter International Inc.Brian Kyhos, (224) 948-4210Deborah Spak, (224) 948-2349orMary Kay Ladone, (224) 948-3371Clare Trachtman, (224) 948-3085 </0>
Baxter International Inc. (NYSE:BAX) today announced pivotal Phase I/III study results evaluating the safety and efficacy of BAX 326, an investigational recombinant factor IX (rFIX) protein, for the treatment and prophylaxis of bleeding episodes for patients with hemophilia B over 12 years of age. The data were presented at the 54 Annual Meeting and Exposition of the American Society of Hematology (ASH) in Atlanta. BAX 326 was recently granted orphan-drug designation by the U.S. Food and Drug Administration (FDA), a special status given to a product that when approved, will address an unmet need for people with a rare disease or condition. Data supporting the prophylaxis indication, which is the basis for the orphan drug designation, was included in the biologics license application (BLA).
''With only one recombinant protein currently available to treat hemophilia B, it was important to focus research efforts on finding another option for patients with this debilitating disease,'' said lead investigator Jerzy Windyga, MD, PhD, Institute of Hematology and Blood Transfusion, Warsaw, Poland. ''In this study, more than 40 percent of patients using BAX 326 as a prophylactic treatment experienced no bleeds, an important finding given the potentially harmful impact of bleeding episodes for patients.''
The Phase I/III prospective, controlled, multicenter study investigated the pharmacokinetics, efficacy and safety of BAX 326 in 73 previously-treated patients with severe or moderately severe hemophilia B. Results from the study showed that twice-weekly prophylactic treatment with BAX 326 achieved a median annualized bleed rate of 1.99 with 43 percent of patients experiencing no bleeds. The impact of prophylaxis with BAX 326 also translated into statistically significant improvements in physical health-related quality of life (HRQoL), as measured by improvements in the Bodily Pain and Role-Physical domains in the Short Form Health Survey (SF-36v2) tool. Statistically significant changes were not seen in the other physical health-related domains or the mental health domains.
No inhibitors were detected and no cases of anaphylaxis were reported in any patients. Three treatment-related adverse events were reported in 2 of the 73 (2.7%) patients, all of which were mild and transient: dysgeusia (distortion of the sense of taste) occurred twice in one patient and pain in extremity occurred once in another patient. More than 70 percent of subjects (56 of 73) had 50 or more exposure days to BAX 326 during the study.
''As a company committed to advancing care for people with hemophilia for more than 60 years, Baxter continues to deliver significant innovation in the field of hemophilia. If it is approved, BAX 326 will expand treatment options for patients,'' said Prof. Harmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business.
To continue on its path of innovation, earlier this year Baxter announced a partnership with Chatham Therapeutics, LLC to develop a gene therapy-based treatment for hemophilia B. Gene therapy represents another important area of research that may become the future of hemophilia B treatment.
Hemophilia B is the second most common type of hemophilia (also known as Christmas disease) and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding. Approximately 25,000 people worldwide, including more than 4,000 in the U.S., have been diagnosed with hemophilia B. Hemophilia B is often a debilitating, chronic disease with complications that include bleeding episodes, hemophilic arthropathy (bleeding into a joint) and hospitalization.
Baxter has more than 60 years experience in hemophilia and has introduced a number of therapeutic firsts for hemophilia patients. Baxter has the broadest portfolio of hemophilia treatments in the industry and is able to meet individual therapy choices, providing a range of options at each treatment stage. The company’s work is focused on optimizing hemophilia care and improving the lives of people living with hemophilia A and B worldwide.
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.
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World Federation of Hemophilia Report on the Annual Global Survey 2010. World Federation of Hemophilia. Accessed on November 28, 2012. Available at:
Lee, C. A. (2011) Hemophilia Care in the Modern World, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford, UK. Chapter 29. Accessed on November 28, 2012.