Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a biologics license application (BLA) supplement to the U.S. Food and Drug Administration (FDA) for the approval of prophylaxis treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex], Nanofiltered and Vapor Heated, in patients with hemophilia A or B and inhibitors.
As many as one third of people with hemophilia develop an inhibitor to factor replacement used to treat or prevent bleeding episodes. The presence of an inhibitor makes response to treatment more challenging, and patients with inhibitors have an increased risk of developing complications such as joint damage. It is estimated that approximately 17,000 people in the U.S. have been diagnosed with hemophilia A or B.
''This regulatory submission helps fulfill Baxter’s commitment to advancing care for patients who have developed inhibitors, a serious and sometimes life-threatening complication for those with hemophilia A or B,'' said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business. ''Baxter has been supporting innovations in care for the hemophilia community for more than 60 years, and our work continues not only with FEIBA, but also with other treatments we are actively developing.''
Baxter recently shared top-line results from the pivotal Phase III study that formed the basis for the BLA submission. Results from the study demonstrated that routine prophylactic treatment with FEIBA NF reduced median annual bleed rate (ABR) from 28.7 during FEIBA NF on-demand treatment compared to 7.9 during FEIBA NF prophylactic treatment (a 72.5% reduction), with 17 percent (3 of 17) of patients in the intent-to-treat group experiencing zero joint bleeds. The complete results of the Phase III study will be presented at a scientific meeting later this year.
The prospective, open label, randomized, multi-center, parallel study investigated the efficacy, safety and health-related quality of life benefits of FEIBA NF prophylactic treatment compared to on-demand treatment in 36 patients with hemophilia A or B and inhibitors over a 12-month period. The most commonly reported adverse reactions in the study were hypersensitivity, dizziness, headache, rash, hypotension and hepatitis B surface antibody positive laboratory test result. The occurrence of hepatitis B surface antibodies has been seen in other plasma-derived products and could be due to the passive transfer of antibodies following FEIBA NF treatment. None of the subjects showed any signs or symptoms of hepatitis B infection.