Baxter International Inc. (NYSE:BAX) today announced pivotal Phase III study results evaluating the efficacy and safety of routine prophylaxis compared to on-demand treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex], Nanofiltered and Vapor Heated, in patients with hemophilia A or B and inhibitors. Top-line results from the study showed a reduced median annual bleed rate (ABR) from 28.7 during FEIBA NF on-demand treatment to 7.9 during FEIBA NF prophylactic treatment (a 72.5% reduction). The Phase III study will form the basis of a biologics license application (BLA) to be filed with the U.S. Food and Drug Administration (FDA) in the first quarter of 2013.
As many as one-third of people with hemophilia develop an inhibitor to a product 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.
''Treatment with FEIBA NF resulted in a significant reduction in annual bleed rate (ABR) of all types of bleeds in the prophylaxis arm as compared to the on-demand arm,'' said lead investigator, Dr. Sandra Antunes MD, UNIFESP, Sao Paulo, Brazil. ''Three of the 17 intent to treat patients (17.6 %) in the prophylaxis arm did not experience any bleeding episodes during the study, and this is very significant for hemophilia patients with inhibitors.''
The Phase III 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 a transitory increase in hepatitis B surface antibodies has been seen in certain plasma-derived products and could be attributed to the passive transfer of antibodies following FEIBA NF treatment. None of the subjects showed any signs or symptoms of hepatitis B infection.