Baxter Seeks Pediatric Indication from FDA for RIXUBIS
Baxter International has submitted an application to the US Food and Drug Administration (FDA) for a pediatric indication for RIXUBIS, the company’s recombinant factor IX (rFIX) therapy for treatment in patients with hemophilia B. The submission was based on the results of a study presented during the 55th Annual Meeting of the American Society of Hematology on December 7-10, 2013, in New Orleans, LA.
In June 2013, the FDA approved RIXUBIS in hemophilia B patients who are 16 years old or older, making it the first new rFIX treatment in 15 years. It is currently the only rFIX product approved for the control and prevention of bleeding episodes, and for routine prophylaxis (twice weekly) to prevent or reduce the frequency of bleeding episodes. It is also approved for perioperative management.
Efficacy and safety of RIXUBIS were evaluated in phase 2/3 clinical trials among 23 previously treated male patients younger than 12 years old with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly RIXUBIS prophylaxis regimen over six months or for a minimum of 50 exposure days. The median annualized bleeding rate was 2.0. Nine patients in the study (39.1%) experienced no bleeds and 23 patients (88.5%) were treated with 1-2 infusions. RIXUBIS was rated “excellent or good” at resolving bleeds in 96% of all episodes. Of the 26 bleeds seen in the trial, only two (in two patients) were spontaneous. There were no reports of inhibitor development, no allergic reactions, and no thrombotic or treatment-related adverse events among the study participants.
“These positive results among a pediatric patient population are consistent with those observed in the RIXUBIS pivotal study among adult patients with hemophilia B,” noted Anders Ullman, MD, PhD, vice president of global research and development in Baxter BioScience. “We submitted these data as part of our application for a pediatric indication for RIXUBIS to advance effective therapeutic solutions for children with hemophilia B.”
Source: Baxter press release dated December 9, 2013