Baxter International Inc. announced on Monday, September 15 that the United States Food and Drug Administration (FDA) has approved RIXUBIS [Coagulation Factor IX (Recombinant)] for routine prophylactic treatment, control and prevention of bleeding episodes, and perioperative management in children with hemophilia B. RIXUBIS was the first recombinant factor IX (rFIX) approved for routine prophylaxis and control of bleeding episodes in the U.S. for adults living with this chronic condition.
“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, M.D., vice president of global research and development at Baxter BioScience.
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聽12 years of age with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly RIXUBIS prophylaxis regimen (mean dose聽56 IU/kg) for a mean treatment duration of six months and a mean of 54 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 bleeding episodes (88.5%) were treated with 1-2 infusions. There were no reports of inhibitor development, no severe allergic reactions, and no thrombotic or treatment-related adverse events among the study participants. Common adverse reactions observed in >1% of subjects in clinical studies were dysgeusia, pain in extremity, and positive test for furin antibody. These data were presented during the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA.
Baxter’s application for marketing approval for RIXUBIS for adults and pediatric patients is currently under review in the European Union, with a regulatory decision expected later this year. The treatment also recently gained regulatory approval in Australia.
RIXUBIS [Coagulation Factor IX (Recombinant)] is an antihemophilic factor indicated in adults and children with hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis. RIXUBIS is not indicated for induction of immune tolerance in patients with hemophilia B.
RIXUBIS was the first rFIX approved by the FDA for both routine prophylaxis and control of bleeding episodes in adults with this chronic condition.