The FDA approved Xyrem (sodium oxybate) for the treatment of cataplexy and excessive daytime sleepiness (EDS) in pediatric patients (seven to 17 years old) with narcolepsy. Xyrem is a central nervous system (CNS) depressant that was approved in 2002 for the treatment of cataplexy in adult patients with narcolepsy. Cataplexy is a sudden and transient episode of muscle weakness accompanied by full conscious awareness, typically triggered by emotions such as laughing, crying, or terror.
“Xyrem is the only FDA-approved treatment available for excessive daytime sleepiness and cataplexy in narcolepsy for adult patients,” said Jed Black, M.D., senior vice president, Sleep and CNS Medicine at Jazz Pharmaceuticals and adjunct professor, Stanford Center for Sleep Sciences and Medicine. “In a pivotal study we demonstrated both safety and efficacy of Xyrem in pediatric patients with narcolepsy. We are pleased to lead the sleep community in advancing the science and identifying meaningful treatment options for children and adolescents.”
The efficacy of Xyrem for the treatment of cataplexy or EDS in pediatric patients with narcolepsy was established in the multisite Phase 2/3 EXPRESS study, which enrolled patients seven to 17 years of age with narcolepsy with cataplexy.
Because of the risk of serious outcomes resulting from inappropriate prescribing, misuse, abuse, and diversion, Xyrem is only available through a risk evaluation mitigation strategy (REMS) programs.
The FDA granted this application Priority Review designation. Xyrem also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.