The US Food and Drug Administration (FDA) has approved RIXUBIS, (Coagulation Factor IX [Recombinant]) manufactured by Baxter International, Inc., for routine prophylactic treatment, control and prevention of bleeding episodes, and perioperative management in children with hemophilia B (factor IX deficiency).
According to a Baxter press release, the approval is based on the results of a clinical trial investigating 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 twice weekly with RIXUBIS for a mean period of six months and a mean of 54 exposure days. The median annualized bleeding rate was 2.0. Nine patients in the study (39.1%) experienced no bleeds, while the remaining 14 patients experienced 23 bleeding episodes (88.5%), which were treated with 1-2 infusions.
Investigators made no reports of inhibitor development, severe allergic reactions, thrombotic or treatment-related adverse events in study participants. Common adverse reactions were observed in >1% of subjects, including distortion or decrease in sense of taste and pain in the extremities. The findings were previously presented during the 55th Annual Meeting of the American Society of Hematology in New Orleans in December 2013.
“In addition to the positive reception we’ve received from adult RIXUBIS patients, the approval for pediatric patients offers a valuable new option, particularly as our clinical data demonstrated a significant reduction in bleeding episodes for patients who were treated prophylactically, an important factor for this young patient population,” said John Orloff, MD, vice president of global research and development at Baxter BioScience.
Source: Baxter press release dated September 15, 2014
