Jazz Pharmaceuticals plc submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) on January 21, 2020, seeking marketing approval for JZP-258, 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.
JZP-258 is a oxybate product candidate with a unique composition of cations resulting in 92%, or approximately 1,000 mg to 1,500 mg, less sodium than Jazz’s Xyrem (sodium oxybate). This substantial reduction could allow patients to achieve a daily sodium intake target of 2,300 mg, and ideally 1,500 mg, as set by the American Heart Association.
“Narcolepsy is a chronic disorder, for which lifelong therapy may be clinically indicated, and is associated with an increased risk of comorbid conditions, including hypertension and cardiovascular disease,” says Robert Iannone, MD, MSCE, executive vice president, research and development of Jazz Pharmaceuticals, in a release. “Jazz is committed to addressing unmet needs in sleep medicine, which includes working for nearly a decade to develop a novel oxybate formulation with a significant reduction in sodium.”
The submission is based on a Phase 3 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.