Jazz's Xyrem meets in Phase II/III for pediatric narcolepsy with cataplexy

Jazz Pharmaceuticals plc (NASDAQ:JAZZ) reported top-line data from the double-blind, international Phase II/III EXPRESS trial in 106 patients ages 7-17 with narcolepsy with cataplexy showing that nightly oral Xyrem sodium oxybate (JZP-6) met the primary endpoint of reducing the weekly number of cataplexy attacks at 2 weeks vs. placebo. Xyrem also met the secondary endpoints of improving Clinical Global Impression of Change (CGI-C) for cataplexy severity and Epworth Sleepiness Scale (ESS) for children and adolescents scores vs. placebo. Data will be presented at the Associated Professional Sleep Societies meeting in Boston in June.

Jazz plans to submit an sNDA to FDA to include the EXPRESS data in the product's label in 4Q17. Xyrem is approved to treat cataplexy in narcolepsy and for excessive daytime sleepiness in narcolepsy. Xyrem was first approved in the U.S. in 2002 based on clinical trial data in adults; Jazz said the current U.S. label for the drug indicates that safety and effectiveness in pediatric patients have not been established...