JZP 258 filed with FDA for cataplexy and excessive daytime sleepiness.- Jazz Pharma

Jazz Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA on January 21, 2020 seeking marketing approval for JZP 258 (sodium oxybate), an investigational medicine for the treatment of cataplexy and excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The company redeemed its priority review voucher for the NDA submission.The submission is based on a Phase III global, double-blind, placebo-controlled, randomized-withdrawal, multicenter study that demonstrated the efficacy and safety of JZP 258 in the treatment of cataplexy and EDS associated with narcolepsy. In the trial, there was a significant increase in median weekly number of cataplexy attacks in participants randomized to placebo compared with participants randomized to JZP 258 (median [Q1, Q3]: 2.35 [0.00, 11.61] vs 0.00 [-0.49, 1.75], respectively; treatment difference, P<0.0001). as expected, initial cataplexy rates differed based on prior therapy at study entry, with participants taking sodium oxybate only or sodium oxybate and an antidepressant anticataplectic reporting the least cataplexy at study entry. in participants taking sodium oxybate only at study entry, cataplexy was stable with jzp 258 treatment across the open-label treatment titration and optimization period and the stable dose period.>