Final FDA decision anticipated by August 12
Phase 3 study results to be presented during AAN Annual
Meeting Clinical Trials Plenary Session on April 20
Company to host investor webcast to review Phase 3 data on
April 20 at 12:30 PM ET
DUBLIN, April 12, 2021 /PRNewswire/ -- Jazz
Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the U.S.
Food and Drug Administration (FDA) has granted Priority Review
designation and confirmed the acceptance for substantive review of
the supplemental New Drug Application (sNDA) seeking approval for
XywavTM (calcium, magnesium, potassium, and sodium
oxybates) oral solution in adult patients with idiopathic
hypersomnia. The sNDA will be filed by the FDA on April 13, 2021 and a PDUFA goal date for an FDA
decision has been set for August 12,
2021.
Jazz submitted to FDA the clinical study report for the Phase 3
study of Xywav in a double-blind, multicenter,
placebo-controlled, randomized withdrawal study evaluating the
efficacy and safety of Xywav in adult patients with
idiopathic hypersomnia in December
2020 under rolling review, and completed the rolling
submission of the sNDA in February
2021. The trial met its primary endpoint of clinically
meaningful improvements in the Epworth Sleepiness Scale (ESS)
p-value <0.0001 and the key secondary endpoints of Patient
Global Impression of change (PGIc) p-value <0.0001 and
Idiopathic Hypersomnia Severity Scale (IHSS) p-value
<0.0001.
The Phase 3 study results will be presented during the Clinical
Trials Plenary Session of the 2021 American Academy of Neurology
(AAN) Annual Meeting on April 20, 2021, with an investor webcast to
follow.
Webcast/conference call details
The company will host
an audio webcast on April 20, 2021 at 12:30 PM ET to review the
Phase 3 idiopathic hypersomnia data being presented at the 2021 AAN
Annual Meeting. The live webcast may be accessed from the Investors
section of the company's website at www.jazzpharmaceuticals.com.
Investors may participate in the conference call by dialing (855)
353-7924 in the U.S. or (503) 343-6056 outside the U.S., and
entering passcode 8075156.
An archived version of the webcast will be available for at
least one week on the Investors section of the company's website.
An audio replay will be available through April 27, 2021 by dialing
(855) 859-2056 in the U.S. or (404) 537-3406 outside the U.S., and
entering passcode 8075156.
About Idiopathic Hypersomnia
Idiopathic hypersomnia
is an often debilitating, neurologic sleep disorder characterized
by chronic excessive daytime sleepiness (the inability to stay
awake and alert during the day resulting in the irrepressible need
to sleep or unplanned lapses into sleep or drowsiness) that is not
caused by other medical, behavioral or psychiatric
conditions.1,2,3,4 Symptoms may also include a prolonged
main sleep episode of more than 9 hours or a sleep duration of 11
hours or longer over a 24-hour period, prolonged, non-restorative
nighttime sleep and long and unrefreshing naps, and severe sleep
inertia (prolonged difficulty waking, with frequent reentries into
sleep, confusion, and irritability).1,2,3,4 Idiopathic
hypersomnia is a condition with its own specific diagnostic
criteria.1 Idiopathic hypersomnia is a debilitating
illness that can significantly affect social, school and
occupational functioning.4,5 Insurance claims data
suggest the number of people diagnosed with idiopathic hypersomnia
and actively seeking healthcare is 37,000 patients in the U.S.;
however, given that idiopathic hypersomnia is often misdiagnosed,
in addition to the lack of an FDA-approved treatment, many people
with idiopathic hypersomnia remain undiagnosed indicating that the
unmet need may be significantly greater.
More information about Xywav, including Full Prescribing
Information and Medication Guide, is available here.
<http://pp.jazzpharma.com/pi/xywav.en.USPI.pdf>
About Xywav™ (calcium, magnesium, potassium, and sodium
oxybates) oral solution
Xywav, also known as
JZP-258, is a lower-sodium oxybate approved by the U.S. Food and
Drug Administration (FDA) for the treatment of cataplexy or
excessive daytime sleepiness in patients 7 years of age and older
with narcolepsy. It is an investigational product being evaluated
for the treatment of idiopathic hypersomnia in adult patients.
While the exact mechanism of action of Xywav is unknown, it
is hypothesized that the therapeutic effects of Xywav on
cataplexy and excessive daytime sleepiness are mediated through
GABAB actions during sleep at noradrenergic and
dopaminergic neurons, as well as at thalamocortical neurons.
Xywav received Fast Track designation by the FDA in
September 2020 for the treatment of
idiopathic hypersomnia.6
Important Safety Information
WARNING: Taking XYWAV
with other central nervous system (CNS) depressants such as
medicines used to make you or your child fall asleep, including
opioid analgesics, benzodiazepines, sedating antidepressants,
antipsychotics, sedating anti-epileptic medicines, general
anesthetics, muscle relaxants, alcohol, or street drugs, may cause
serious medical problems, including trouble breathing (respiratory
depression), low blood pressure (hypotension), changes in alertness
(drowsiness), fainting (syncope), and death.
The active ingredient of XYWAV is a form of gamma
hydroxybutyrate (GHB). Abuse or misuse of illegal GHB alone or with
other drugs that cause changes in alertness (or consciousness) has
caused serious side effects. These effects include seizures,
trouble breathing (respiratory depression), changes in alertness
(drowsiness), coma, and death. Call your doctor right away if you
or your child has any of these serious side effects. Because
of these risks, you have to go through the XYWAV and XYREM REMS
Program to have your or your child's prescription for XYWAV
filled.
Do not take XYWAV if you take or your child takes other
sleep medicines or sedatives (medicines that cause sleepiness),
drinks alcohol, or has a rare problem called succinic semialdehyde
dehydrogenase deficiency.
Keep XYWAV in a safe place to prevent abuse and misuse. Selling
or giving away XYWAV may harm others, and is against the law. Tell
your doctor if you have ever abused or been dependent on alcohol,
prescription medicines, or street drugs.
Anyone who takes XYWAV should not do anything that requires them
to be fully awake or is dangerous, including driving a car, using
heavy machinery, or flying an airplane, for at least 6 hours after
taking XYWAV. Those activities should not be done until you know
how XYWAV affects you or your child.
XYWAV can cause serious side effects, including the
following:
- Breathing problems, including slower breathing, trouble
breathing, and/or short periods of not breathing while sleeping
(sleep apnea). People who already have breathing or lung problems
have a higher chance of having breathing problems when they use
XYWAV.
- Mental health problems, including confusion, seeing or
hearing things that are not real (hallucinations), unusual or
disturbing thoughts (abnormal thinking), feeling anxious or upset,
depression, thoughts of killing yourself or trying to kill
yourself, increased tiredness, feelings of guilt or worthlessness,
or difficulty concentrating. Tell your doctor if you or your child
have or had depression or have tried to harm yourself or
themselves. Call your doctor right away if you have or your
child has symptoms of mental health problems or a change in weight
or appetite.
- Sleepwalking. Sleepwalking can cause injuries. Call your
doctor if you or your child starts sleepwalking. Your doctor should
check you or your child.
The most common side effects of XYWAV in adults include
headache, nausea, dizziness, decreased appetite, parasomnia (a
sleep disorder that can include abnormal dreams, abnormal rapid eye
movement (REM) sleep, sleep paralysis, sleep talking, sleep terror,
sleep-related eating disorder, sleep walking, and other abnormal
sleep-related events), diarrhea, excessive sweating
(hyperhidrosis), anxiety and vomiting.
The most common side effects of XYWAV in children include
bedwetting, nausea, headache, vomiting, weight decrease, decreased
appetite, and dizziness.
XYWAV can cause physical dependence and craving for the medicine
when it is not taken as directed. These are not all the possible
side effects of XYWAV.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call
1-800-FDA-1088.
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 malignancies 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 the goal date for an FDA decision on the sNDA
submission and the potential timing of the availability of
Xywav for people with idiopathic hypersomnia; and other
statements that are not historical facts. These forward-looking
statements are based on Jazz Pharmaceuticals' 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, risks and
uncertainties associated with: pharmaceutical product development;
the regulatory approval process, including the risk that the
company may be unable to obtain approval by the FDA of its sNDA for
Xywav in a timely manner or at all; effectively
commercializing Xywav; and other risks and uncertainties
affecting the company and its development programs, 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 Jazz Pharmaceuticals' Annual Report on Form
10-K for the year ended December 31,
2020 and future filings and reports by Jazz Pharmaceuticals.
Other risks and uncertainties of which Jazz Pharmaceuticals is not
currently aware may also affect Jazz Pharmaceuticals'
forward-looking statements and may cause actual results and the
timing of events to differ materially from those anticipated. The
forward-looking statements herein are made only as of the date
hereof or as of the dates indicated in the forward-looking
statements, even if they are subsequently made available by Jazz
Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals
undertakes no obligation to update or supplement any
forward-looking statements to reflect actual results, new
information, future events, changes in its expectations or other
circumstances that exist after the date as of which the
forward-looking statements were made.
- 2015 Review article in Chest, by Khan/Trotti et al, "Central
Disorders of Hypersomnolence: Focus on the Narcolepsies and
Idiopathic Hypersomnia"
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4694150/
- 2016 Sleep Medicine Review article, by Billiard/Sonka et al,
"Idiopathic Hypersomnia"
https://www.ncbi.nlm.nih.gov/pubmed/26599679
- International Classification of Sleep Disorders, Third Edition
(ICSD 3):
http://www.aasmnet.org/store/product.aspx?pid=849
- Diagnostic and Statistical Manual of Mental Disorders (DSM-V)
p. 368-372 Hypersomnolence Disorder:
https://www.psychiatry.org/psychiatrists/practice/dsm
- G Hess, R Mehra, G Carls, J Profant, J Altenburger, O
Pasenchenko, J F Acquavella, 0625 US Prevalence of Narcolepsy and
Other Sleep Disorders From 2013–2016: A Retrospective,
Epidemiological Study Utilizing Nationwide Claims, Sleep, Volume
41, Issue suppl_1, April 2018, Page
A232, https://doi.org/10.1093/sleep/zsy061.624
- Xywav (calcium, magnesium, potassium and sodium oxybates) oral
solution Prescribing Information. Palo
Alto, CA: Jazz Pharmaceuticals, Inc.
Media Contact:
Jacqueline
Kirby, Vice President, Corporate Affairs & Government
Relations
Ireland +353 1 697
2141 U.S. +1 215 867 4910
Investor Contact:
Andrea N.
Flynn, Ph.D., Vice President, Head, Investor Relations
Ireland +353 1 634
7887 U.S. +1 650 496 2717
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