Baxter International Inc. (NYSE:BAX) today announced that it has submitted an application to the United States Food and Drug Administration (FDA) for a pediatric indication for RIXUBIS [Coagulation Factor IX (Recombinant)] to treat hemophilia B. The submission was based on the results of a study presented during the 55 th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA. RIXUBIS was approved in the United States for adults with hemophilia B earlier this year and the company filed for marketing approval in Europe in November.
''Routine prophylactic infusions can be particularly beneficial for children with hemophilia, particularly if they are started early to help prevent joint bleeding,'' explained lead investigator Tomasz Urasinski, Pomeranian Medical University, Department of Paediatrics, Haematology and Oncology, Szczecin, Poland. ''The clinical evidence suggests that RIXUBIS prophylaxis can offer hemophilia B patients a reduction in bleeding episodes while also being tolerable, an important factor for this young population.''
The Phase II/III clinical trial investigated the efficacy and safety of RIXUBIS among 23 previously-treated male patients less than 12 years of age with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly RIXUBIS prophylaxis regimen (median dose 56 IU/kg) over six months or for a minimum of 50 exposure days (EDs). The median annualized bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds). Nine patients in the study (39.1%) experienced no bleeds and 23 patients (88.5%) were treated with 1-2 infusions. In hemostatic efficacy, RIXUBIS was rated excellent or good at resolving bleeds in 96 percent of all episodes. Of the 26 bleeds seen in the trial, only two (in two patients) were spontaneous. There were no reports of inhibitor development, no allergic reactions, and no thrombotic or treatment-related adverse events among the study participants.
''These positive results among a pediatric patient population are consistent with those observed in the RIXUBIS pivotal study among adult patients with hemophilia B,'' noted Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter BioScience. ''We submitted these data as part of our application for a pediatric indication for RIXUBIS to advance effective therapeutic solutions for children with hemophilia B.''