Jazz Pharmaceuticals Announces FDA Acceptance of New Drug Application for JZP-258 for Cataplexy and Excessive Daytime Sleepiness Associated with Narcolepsy
"We developed JZP-258 to be a safer and long-term treatment option for patients. JZP-258 represents between 1,000 and 1,500 milligrams daily reduction of sodium for patients currently treated with Xyrem, depending on the dose," said
Narcolepsy is a chronic, debilitating neurological disorder characterized by EDS and the inability to regulate sleep-wake cycles normally.1 It affects an estimated one in 2,000 people in the United States, with symptoms typically appearing in childhood or adolescence. It is estimated that more than 50% of people with narcolepsy have not been diagnosed.2 Studies have shown it may take 10 years or more for people with narcolepsy to receive a diagnosis.3 There are five primary symptoms of narcolepsy, including EDS, cataplexy, disrupted nighttime sleep, sleep-related hallucinations, and sleep paralysis.6 While all people with narcolepsy experience EDS, not all individuals with narcolepsy experience all five symptoms.7,8 EDS is the primary symptom of narcolepsy and is present in all people with the disorder.4 EDS is characterized by the inability to stay awake and alert during the day resulting in drowsiness and unplanned lapses into sleep.2,4,5 There is also an increased prevalence of cardiometabolic comorbidities, including obesity, hypertension, diabetes and dyslipidemia, in patients with narcolepsy.9,10,11,12
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.7,8,13 Although many emotions can potentially lead to cataplexy, those associated with mirth are usually the most potent.7 Cataplexy occurs in about 70% of people with narcolepsy.14 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.7,8 Complete collapse is less common.8 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).7,8,13,14
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 in adult patients. 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 with each nightly dose. 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.
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