Jazz Pharmaceuticals Submits New Drug Application for JZP-258 for Cataplexy and Excessive Daytime Sleepiness Associated with Narcolepsy
JZP-258 is a novel oxybate product candidate with a unique composition of cations resulting in 92%, or approximately 1,000 to 1,500 milligrams, less sodium than 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
"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," said
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
Narcolepsy is a chronic, debilitating neurological disorder characterized by excessive daytime sleepiness (
Cataplexy, the most specific symptom of narcolepsy, is the sudden, generally brief (<2 minutes) loss of muscle tone with retained consciousness. It is usually triggered by strong emotions, such as laughter, surprise, or anger.8,9,14 Although many emotions can potentially lead to cataplexy, those associated with mirth are usually the most potent.8 Cataplexy occurs in about 70% of people with narcolepsy.15 Presentation differs widely among people with narcolepsy, ranging from sporadic partial attacks triggered by laughter to frequent complete collapse brought about by a variety of emotions.8,9 Complete collapse is less common.9 More commonly, episodes of cataplexy involve only certain muscle groups, such as arms and legs (e.g., knees buckling), the head and neck (e.g., head dropping), or the face and jaw (e.g., sagging, slurred speech, eyelid drooping).8,9,14,15
JZP-258 is an investigational medicine developed for the treatment of cataplexy and excessive daytime sleepiness in patients 7 years of age and older with narcolepsy, as well as for the treatment of idiopathic hypersomnia. JZP-258 is a novel oxybate product candidate with a unique composition of cations resulting in 92%, or approximately 1,000 to 1,500 milligrams, less sodium than Xyrem. JZP-258 has the same oxybate concentration as Xyrem and includes other cations, including calcium, magnesium and potassium. While the exact mechanism of action of JZP-258 is not fully understood, it is hypothesized that the therapeutic effects of JZP-258 on sleep/wake symptoms are mediated through modulation of GABAB during sleep.
About Jazz Pharmaceuticals plc
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