The US Food and Drug Administration (FDA) has accepted for priority review Jazz Pharmaceuticals plc’s supplemental new drug application (sNDA) seeking revised labeling for Xyrem (sodium oxybate) oral solution, CIII, to include an indication to treat cataplexy and excessive daytime sleepiness (EDS) in pediatric narcolepsy patients. The Prescription Drug User Fee Act (PDUFA) goal date for an FDA decision is October 27, 2018.
“There has been a great deal of interest from the narcolepsy community in understanding the safety and efficacy of Xyrem in pediatric patients. We look forward to the FDA review and the potential for a new Xyrem indication specific to cataplexy and EDS in children and adolescents,” says Jed Black, MD, senior vice president, Sleep and CNS Medicine at Jazz Pharmaceuticals and adjunct professor, Stanford Center for Sleep Sciences and Medicine, in a release. “Jazz continues to strive to address unmet needs within the sleep community.”
Xyrem oral solution, CIII, is indicated for the treatment of cataplexy in narcolepsy and for the treatment of EDS in narcolepsy. Xyrem may only be dispensed to patients enrolled in the Xyrem REMS Program. Xyrem was first approved in the United States in 2002, based on clinical trial data in adults. The current United States Package Insert (USPI) for Xyrem indicates that safety and effectiveness in pediatric patients have not been established. Jazz filed an sNDA on April 27, 2018 to expand the currently approved indication to include pediatric narcolepsy patients. With collaboration and a Pediatric Written Request from the FDA, Jazz initiated the global Phase 2/3 EXPRESS study in 2014 to assess the safety, efficacy, and pharmacokinetics of Xyrem in pediatric patients, seven to 17 years of age.