Sodium oxybate (Xyrem) has been approved to treat cataplexy or excessive daytime sleepiness (EDS) in patients with narcolepsy aged seven and older by USFDA, announced Jazz Pharmaceuticals.
Sodium oxybate, a central nervous system depressant, was previously only indicated for use in adults.
The drug won the approval for the treatment of cataplexy or EDS in paediatric patients with narcolepsy after the findings of multisite Phase 2/3 EXPRESS study, which enrolled patients seven to 17 years of age with narcolepsy with cataplexy.
Study patients who were sodium oxybate-naive at entry underwent open-label titration to reach a tolerable and effective dose. All patients then underwent a 2-week, double-blind, randomised-withdrawal period and were randomised to either remain on sodium oxybate or receive placebo.
The primary efficacy endpoint was the change in a weekly number of cataplexy attacks from baseline to the end of the double-blind period. After this period, patients entered an open-label safety period for up to 47 additional weeks.
The study found that compared with sodium oxybate-treated patients, those who received the placebo experienced a statistically significant increase in weekly cataplexy attacks. In addition, patients in the placebo group, during the double blind treatment period, experienced a statistically significant worsening of EDS vs patients who continued to receive sodium oxybate.