FDA Extends Rixubis Indication For Hemophilia B To Pediatric Patients

Baxter International announced in a press release that the Food and Drug Administration (FDA) has approved Rixubis [Coagulation Factor IX (Recombinant)] for pediatric patients. The recombinant factor IX (rFIX), which was originally approved by the FDA in June 2013 for patients aged 16 years and older, is now approved for children with hemophilia B, for prophylactic treatment, control, and prevention of bleeding episodes. John Orloff, M.D., VP of global research and development at Baxter BioScience, said, "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.”

Rixubis

Rixubis is an antihemophilic factor, a purified protein (not containing human or animal proteins) produced with recombinant DNA technology. The product is packaged in single-use vials of freeze-dried powder. After reconstitution with sterile water, the agent is administered by intravenous injection twice weekly.

The FDA based its approval in part on clinical trial data showing that 39 percent of patients 12 years of age showed no bleeds while using Rixubis as a prophylactic treatment. In the trial, 23 previously treated male patients with severe or moderately severe hemophilia B were treated with a twice-weekly dose of 56 IU/kg for an average of six months. Nine patients (39.1%) experienced no bleeds and there were no reports of severe allergic reactions.

Current Hemophilia B Treatments

Hemophilia B is a hereditary disease, which occurs in approximately 1 in 5,000 births in the U.S., in all races and ethnic groups. Hemophilia B is much less common than hemophilia A. Concentrated FIX products, also known as clotting factor, are the main treatment. The treatments are administered intravenously. People with more severe forms of the disease are treated routinely with prophylactic care that administers clotting factor regularly to prevent bleeding episodes.

In March 2014, the FDA approved Biogen Idec’s Alprolix for Hemophilia B. The approval marked the federal agency’s first approval for hemophilia B in 17 years. The product, Alprolix, requires fewer injections than other products to control and prevent bleeding in adults and children. Alprolix allows patients to receive prophylactic infusions on at least a weekly basis.