DUBLIN, Jan. 22, 2020 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration 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 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 American Heart Association.1
"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 Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "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.
Narcolepsy is a chronic, debilitating neurological disorder characterized by excessive daytime sleepiness (EDS), and the inability to regulate sleep-wake cycles normally.2 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.3 Studies have shown it may take 10 years or more for people with narcolepsy to receive a diagnosis.4 EDS is the primary symptom of narcolepsy and is present in all people with the disorder.5 EDS is characterized by the inability to stay awake and alert during the day resulting in drowsiness and unplanned lapses into sleep.3,5,6 There are five primary symptoms of narcolepsy, including EDS, cataplexy, disrupted nighttime sleep, sleep-related hallucinations, and sleep paralysis.7 While all people with narcolepsy experience EDS, not all individuals with narcolepsy experience all five symptoms.8,9 There is also an increased prevalence of cardiometabolic comorbidities, including obesity, hypertension, diabetes and dyslipidemia, in patients with narcolepsy.10,11,12,13
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
Jazz Pharmaceuticals plc (Nasdaq: JAZZ), a global biopharmaceutical company, is dedicated to developing life-changing medicines for people with limited or no options. As a leader in sleep medicine, with an R&D expansion into neuroscience, and a growing hematology/oncology portfolio, Jazz has a diverse portfolio of products and product candidates in development, and is focused on transforming biopharmaceutical discoveries into novel medicines. Jazz Pharmaceuticals markets Sunosi® (solriamfetol), Xyrem® (sodium oxybate) oral solution, Defitelio® (defibrotide sodium), Erwinaze® (asparaginase Erwinia chrysanthemi) and Vyxeos® (daunorubicin and cytarabine) liposome for injection in the U.S. and markets Sunosi®, Defitelio® (defibrotide), Erwinase® and Vyxeos® liposomal 44 mg/100 mg powder for concentrate for solution for infusion in countries outside the U.S. For country-specific product information, please visit https://www.jazzpharmaceuticals.com/medicines. For more information, please visit www.jazzpharmaceuticals.com and follow us on Twitter at @JazzPharma.
Jacqueline Kirby, Vice President, Corporate Affairs & Government Relations
Ireland +353 1 697 2141 U.S. +1 215 867 4910
Kathee Littrell, Vice President, Investor Relations
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- Amer. Heart Assoc, How much sodium should I eat per day (2018), https://www.heart.org/en/healthy-living/healthy-eating/eat-smart/sodium/how-much-sodium-should-i-eat-per-day.
- Thorpy M, Krieger A. Delayed diagnosis of narcolepsy: characterization and impact. Sleep Medicine. 2014;15(5):502–507.
- Ahmed I, Thorpy, M. Clinical Features, Diagnosis and Treatment of Narcolepsy. Clin Chest Med. 2010;31(2):371-381.
- Morrish E, King M, et al. Factors associated with a delay in the diagnosis of narcolepsy. Sleep Medicine. 2004;5(1):37-41.
- American Academy of Sleep Medicine. The International Classification of Sleep Disorders. Third Edition (ICSD-3). 2014.
- Ahmed I, Thorpy, M. Sleepiness: Causes, Consequences and Treatment, ed. Cambridge University Press. 2011:36-49.
- Pelayo R, Lopes MC. Narcolepsy. In: Lee-Chiong TL, ed. Sleep: A Comprehensive Handbook. Hoboken, NJ: Wiley and Sons, Inc.; 2006:145-149.
- American Academy of Sleep Medicine. Central disorders of hypersomnolence. In: The International Classification of Sleep Disorders – Third Edition (ICSD-3). Darien, IL: American Academy of Sleep Medicine; 2014.
- Ahmed I, Thorpy M. Clinical features, diagnosis and treatment of narcolepsy. Clin Chest Med. 2010;31(2):371-381.
- Cohen A, et al. Sleep Med. 2018;43:14-18.
- Black J, et al. Sleep Med. 2017;33:13-18.
- Jennum P, et al. Sleep. 2013;36(6):835-840.
- Ohayon MM. Sleep Med. 2013;14(6):488-492.
- Overeem S, van Nues SJ, van der Zande WL, et al. The clinical features of cataplexy: a questionnaire study in narcolepsy patients with and without hypocretin-1 deficiency.Sleep Med. 2011;12(1):12-18.
- Overeem S. The clinical features of cataplexy. In: Baumann CR, Bassetti CL, Scammell TE, eds. Narcolepsy: Pathophysiology, Diagnosis, and Treatment. New York, NY: Springer Science+Business Media; 2011:283-290.
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