FDA Approves RIXUBIS for Hemophilia B Treatment in Children

The FDA has approved Baxter’s Rixubis [Coagulation Factor IX (Recombinant)], an intravenous prophylactic treatment intended to control and prevent bleeding episodes and assist with perioperative management for children 12 years and younger with hemophilia B.

Hemophilia B, often referred to as Christmas disease, is the second most common form of hemophilia. Affecting 26,000 individuals worldwide, the disease is identified by decreased amounts of a naturally present clotting protein called factor IX, according to the drug’s manufacturer.

In a clinical trial gauging Rixubis’ efficacy and safety, the drug was administered twice weekly (mean dose 56 IU/kg) for 6 months to 23 males under the age of 12 with moderate to severe hemophilia B.

Researchers noted no bleeding incidents for 9 patients (39.1%). The 21 subjects (88.5%) who experienced a bleeding episode were successfully treated with 1-2 infusions. Baxter did not report any inhibitor development, severe allergic reactions, and thrombotic or treatment-related adverse events (AE) among their subjects. However, in less than 1% of participants, common adverse reactions such as dysgeusia, extremity pain, and positive test for furin antibody were witnessed.

"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," commented John Orloff, MD, vice president of global research and development at Baxter BioScience.

Patients with a hamster allergy, disseminated intravascular coagulation (DIC), and exhibited signs of having fibrinolysis were recommended in Baxter’s full Prescribing Information to not be prescribed the drug.

The statement also warned since products containing factor IX have an established association with thromboembolic events and consumptive coagulopathy, special caution and supervision should be provided to patients undergoing treatment that have “liver disease, signs of fibrinolysis, [and] at risk for thromboembolic events or DIC.”

Recently, regulatory approval for Rixubis was passed in Australia. Approval for the drug is currently is under review by the European Union which a decision will be made this year.