Pharma/Biotech Update: Rixubis approved for therapy for Children with Hemophilia B

As webleed.org reported back on December 9, 2013, Baxter had submitted to the FDA their hemophilia B product, Rixubis, for pediatric indication.

Baxter International Inc. announced 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.

According to Baxter, 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 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 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.

“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., VP of global research and development at Baxter BioScience.

For RIXUBIS prescribing information, click here.

We are thrilled that pediatric patients with hemophilia B now have RIXUBIS as an option for their treatment.

Photo Credit – Baxter International, Inc.

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