FDA Approves Treatment for Children with Hemophilia B

Baxter International announced on Sept. 15, 2014 that FDA 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 United States for adults living with this chronic condition.

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. According to Baxter, the patients were treated with a twice-weekly RIXUBIS prophylaxis regimen (mean dose
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 one to two infusions. There were no reports of inhibitor development, no severe allergic reactions, and no thrombotic or treatment-related adverse events among the study participants.