DUBLIN, March 25, 2020 /PRNewswire/ -- Jazz
Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the U.S.
Food and Drug Administration (FDA) accepted for filing with
Priority Review the company's New Drug Application (NDA) seeking
marketing approval for JZP-258, an investigational medicine for the
treatment of cataplexy or excessive daytime sleepiness (EDS) in
patients 7 years of age and older 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). Xyrem is the only available product approved to
treat both cataplexy and EDS in patients with narcolepsy ages 7
years and older and is the standard of care for treatment of
cataplexy. The Prescription Drug User Fee Act (PDUFA) goal date for
an FDA decision is July 21, 2020.
"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 Robert Iannone, M.D., M.S.C.E., executive vice
president, research and development of Jazz Pharmaceuticals.
"Given the broad scientific consensus that reducing daily sodium
consumption is associated with clinically meaningful reductions in
blood pressure and cardiovascular disease risk, we believe that
JZP-258 has the potential to be an important treatment option for
patients living with the life-long condition of narcolepsy.
Narcolepsy patients are known to be at increased risk of
comorbidities, including obesity, hypertension, diabetes and
dyslipidemia.9,10,11,12"
About Narcolepsy
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
About Cataplexy
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
About JZP-258
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.
About Jazz Pharmaceuticals plc
Jazz
Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharmaceutical
company dedicated to developing life-changing medicines for people
with serious diseases — often with limited or no options. We have a
diverse portfolio of marketed medicines and novel product
candidates, from early- to late-stage development, in key
therapeutic areas. Our focus is in neuroscience, including sleep
medicine and movement disorders, and in oncology, including
hematologic and solid tumors. We actively explore new options for
patients including novel compounds, small molecule advancements,
biologics and innovative delivery technologies. Jazz is
headquartered in Dublin, Ireland
and has employees around the globe, serving patients in more than
90 countries. For more information, please visit
www.jazzpharmaceuticals.com and follow @JazzPharma on Twitter.
"Safe Harbor" Statement under the Private Securities
Litigation Reform Act of 1995
This press release contains forward-looking statements, including,
but not limited to, statements related to Jazz Pharmaceuticals'
belief in JZP-258's potential to be an important treatment option,
which was developed to be a safer and life-long treatment option
for patients and other statements that are not historical facts.
These forward-looking statements are based on the company's current
plans, objectives, estimates, expectations and intentions and
inherently involve significant risks and uncertainties. Actual
results and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of these
risks and uncertainties, which include, without limitation, the
risk that JZP-258 may not be approved by FDA by the PDUFA date, or
otherwise in a timely manner, or at all; effectively
commercializing JZP-258, if approved; and other risks and
uncertainties affecting the company, including those described from
time to time under the caption "Risk Factors" and elsewhere in Jazz
Pharmaceuticals plc's Securities and Exchange Commission filings
and reports (Commission File No. 001-33500), including the
company's Annual Report on Form 10-K for the year ended
December 31, 2019 and future filings
and reports. Other risks and uncertainties of which the company is
not currently aware may also affect the company's forward-looking
statements and may cause actual results and the timing of events to
differ materially from those anticipated.
Media Contact:
Jacqueline Kirby, Vice President,
Corporate Affairs & Government Relations
Ireland +353 1 697
2141 U.S. +1 215
867 4910
Investor Contact:
Kathee Littrell, Vice President,
Investor Relations
Ireland +353 1 634
7887 U.S. +1 650
496 2717
References:
- 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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SOURCE Jazz Pharmaceuticals plc