Avadel Pharmaceuticals plc (Nasdaq: AVDL), a company focused
on transforming medicines to transform lives, today announced new
and encore data supporting the clinical profile for LUMRYZ™ (sodium
oxybate) for extended-release oral suspension (CIII) and patient
preference for a once-nightly oxybate in 15 poster presentations
and two oral presentations, at World Sleep 2023, being held
from October 20-25, 2023 in Rio de Janeiro, Brazil.
LUMRYZ (previously known as FT218), is a U.S. Food and Drug
Administration (FDA) approved extended-release formulation of
sodium oxybate indicated to be taken once at bedtime for the
treatment of cataplexy or excessive daytime sleepiness (EDS) in
adults with narcolepsy.
“We are excited to be at this year’s World Sleep meeting with
another robust display of data that supports both the established
clinical benefit of LUMRYZ,” said Jennifer Gudeman, PharmD,
Senior Vice President, Medical and Clinical Affairs of Avadel.
“Quality sleep is a foundational pillar of health for people with
narcolepsy, yet ~65% of patients with narcolepsy experience poor,
fragmented sleep in addition to their daytime symptoms.* LUMRYZ is
the only FDA-approved oxybate treatment that provides clinically
proven symptom improvement for excessive daytime sleepiness (EDS)
or cataplexy while its novel once-at-bedtime formulation removes
the need for forced awakenings to take a middle of the night
oxybate dose.”
LUMRYZ has a boxed warning as a central nervous system
depressant, and for its potential for abuse and misuse. LUMRYZ is
available only through a restricted program under a Risk Evaluation
and Mitigation Strategy called the LUMRYZ REMS. Most common adverse
reactions (incidence > 5% and greater than placebo) reported for
all doses of LUMRYZ combined were nausea, dizziness, enuresis,
headache, and vomiting.
Highlights from the presentations include:
- Post-hoc analyses reinforcing data from the completed pivotal
Phase 3 REST-ON trial, demonstrating that treatment with LUMRYZ
resulted in statistically significant and clinically meaningful
improvement in EDS as measured by the Epworth Sleepiness Scale, a
patient reported outcome, with the median score in the range
considered normal at the end of the study.
- A poster from the open-label RESTORE study demonstrating
long-term tolerability and clinically significant improvement in
symptoms, including EDS, cataplexy, nighttime awakenings and
severity of narcolepsy, with LUMRYZ, providing insight into its
real-world use.
- Two posters including new post-hoc analyses that demonstrate
the robust clinical efficacy of LUMRYZ and provide further insight
into the improvements on measures of EDS and cataplexy compared
with placebo in different demographic and clinical subgroups based
on age, sex, race, body mass index, narcolepsy subtype and alerting
agent use.
- A poster presenting demographic differences between patients
with narcolepsy subtypes NT1 (with cataplexy) and NT2 (without
cataplexy), with data from objective measures of EDS showing
identical sleep latency, comparable subjective assessments of sleep
quality and refreshing nature of sleep, with some other baseline
parameters suggestive of more severe disease in NT1.
- A poster with results from a survey of people living with
narcolepsy describing their significant daytime and nighttime
symptoms and primary focus on treatment of daytime symptoms in
discussions between clinicians and patients, highlighting the need
for comprehensive discussion and understanding of the full range of
symptoms experienced by people with narcolepsy.
Presentation details are as follows:
Title |
Session |
Presenter |
Date & Time (BRT) |
Oral Presentations |
Improvement in Sleep Latency With Once-Nightly Sodium Oxybate:
Analysis From the Phase 3 REST-ON Clinical Trial |
O02 |
Michael J. Thorpy |
October 23; 09:52 - 10:05 a.m. |
Sodium Oxybate Treatment Patterns in Narcolepsy Patients: A
Propensity Score–Matched Cohort Study Subanalysis |
O02 |
Lois Krahn |
October 23; 10:05 - 10:18 a.m. |
Poster Presentations |
Long-Term Safety and Efficacy of Extended-Release Once-Nightly
Sodium Oxybate for Narcolepsy |
Poster #105 |
Clete A. Kushida |
October 22; 5:00 - 6:00 p.m. |
Characterization of Patients With Narcolepsy Treated vs Not Treated
With Sodium Oxybate: A Propensity Score–Matched Cohort Study |
Poster #108 |
Lois Krahn |
October 22; 5:00 - 6:00 p.m. |
Understanding Daytime and Nighttime Treatment Needs From the
Patient’s Perspective: A Survey of People Living With
Narcolepsy |
Poster #110 |
Luis E. Ortiz |
October 22; 5:00 - 6:00 p.m. |
Patient Preferences and Nocturnal Experiences With Oxybate Therapy
for Narcolepsy: RESTORE Study Interim Analysis |
Poster #112 |
Akinyemi O. Ajayi |
October 22; 5:00 - 6:00 p.m. |
Cataplexy Response With Once-Nightly Sodium Oxybate: Post Hoc
Responder Analysis From the Phase 3 REST-ON Clinical Trial |
Poster #114 |
Michael J. Thorpy |
October 22; 5:00 - 6:00 p.m. |
Magnitude of Improvement in Excessive Daytime Sleepiness With the
Once-at-Bedtime Oxybate for Narcolepsy |
Poster #109 |
Clete Kushida |
October 23; 6:00 - 7:00 p.m. |
Path to Diagnosis and Impact of Narcolepsy on Quality of Life: A
Survey of People Living With Narcolepsy |
Poster #116 |
Anne Marie Morse |
October 23; 6:00 - 7:00 p.m. |
Understanding Narcolepsy Treatments From the Patient’s Perspective:
A Survey of People Living With Narcolepsy |
Poster #117 |
Luis E. Ortiz |
October 23; 6:00 - 7:00 p.m. |
Application of AASM Clinical Significance Thresholds to
Once-Nightly Sodium Oxybate for Improvement in Narcolepsy
Symptoms |
Poster #119 |
Yves Dauvilliers |
October 23; 6:00 - 7:00 p.m. |
Dose Titration of Once-Nightly Sodium Oxybate:Analysis of Interim
Data From RESTORE |
Poster #120 |
Jennifer Gudeman |
October 23; 6:00 - 7:00 p.m. |
Consistent Efficacy of Once-Nightly Sodium Oxybate Regardless of
Patient Demographic and Baseline Disease Characteristics |
Poster #123 |
Michael J. Thorpy |
October 23; 6:00 - 7:00 p.m. |
Comparison of Demographics and Baseline Narcolepsy SymptomsBetween
Participants With NT1 and NT2 From the Phase 3 REST-ON Clinical
Trial |
Poster #096 |
Yves Dauvilliers |
October 24; 6:00 - 7:00 p.m. |
Composite Response With Once-Nightly Sodium Oxybate: Symptom
Improvement in Participants With Narcolepsy Type 1 in REST-ON |
Poster #103 |
Luis E. Ortiz |
October 24; 6:00 - 7:00 p.m. |
Characterization of Patients Who Had ≥5% Weight Loss With Once-
Nightly Sodium Oxybate: Post Hoc Analysis From REST-ON |
Poster #105 |
Anne Marie Morse |
October 24; 6:00 - 7:00 p.m. |
Long-Term Safety of Once-Nightly Sodium Oxybate for Narcolepsy:
RESTORE Study Interim Analysis of Data |
Poster #106 |
Akinyemi O. Ajayi |
October 24; 6:00 - 7:00 p.m. |
In addition to Avadel’s oral and poster presentations, the
Company will support a symposium for U.S. healthcare professionals
titled “Evolving the Therapeutic Landscape for Narcolepsy”
on Tuesday, October 24, 12:30 p.m. – 2:00 p.m. BRT
at the Windsor Convention & Expo Center.
The symposium will feature Anne Marie Morse, D.O., Director of
Child Neurology and Pediatric Sleep Medicine at Geisinger
Medical Center at Janet Weis Children’s Hospital; Yves
Dauvilliers, M.D., Ph.D., Director of the Sleep and Wake Disorders
Centre in the Department of Neurology at the Gui
de Chauliac Hospital in
Montpellier, France; Michael J. Thorpy, M.D., Director of
the Sleep-Wake Disorders Center at Montefiore and Professor of
Neurology at Albert Einstein College of Medicine; Clete
Kushida, M.D., Ph.D., Division Chief and Medical Director of
Stanford Sleep Medicine, neurologist and Professor in
the Department of Psychiatry and Behavioral
Sciences at Stanford University Medical Center and
Director of the Stanford Center for Human Sleep
Research at Stanford University.
About LUMRYZ™ (sodium oxybate) for extended-release oral
suspensionLUMRYZ, is an extended-release sodium oxybate
medication approved by the FDA on May 1, 2023, as the first
and only once-at-bedtime oxybate treatment for cataplexy or
excessive daytime sleepiness (EDS) in adults with narcolepsy.
The FDA approval of LUMRYZ was supported by results from
REST-ON, a randomized, double-blind, placebo-controlled, pivotal
Phase 3 trial in adults with narcolepsy. LUMRYZ demonstrated
statistically significant and clinically meaningful improvements in
the three co-primary endpoints: EDS, clinicians’ overall assessment
of patients’ functioning (CGI-I) and cataplexy attacks, for all
three evaluated doses when compared to placebo.
With its approval, the FDA also granted seven years of Orphan
Drug Exclusivity to LUMRYZ for the treatment of cataplexy or EDS in
adults with narcolepsy due to a finding of clinical superiority of
LUMRYZ relative to currently available oxybate treatments. In
particular, the FDA found that LUMRYZ makes a major contribution to
patient care over currently available, twice-nightly oxybate
products by providing a once-nightly dosing regimen that avoids
nocturnal arousal to take a second dose.
IMPORTANT SAFETY INFORMATION
WARNING: Taking LUMRYZ™ (sodium oxybate) with other central
nervous system (CNS) depressants, such as medicines used to make
you 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 LUMRYZ (sodium oxybate) is a form
of gamma hydroxybutyrate (GHB), a controlled substance. Abuse or
misuse of illegal GHB alone or with other CNS depressants (drugs
that cause changes in alertness or consciousness) have 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
have any of these serious side effects. |
|
Because of these risks, LUMRYZ is available only by
prescription and filled through certified pharmacies in the LUMRYZ
REMS. You must be enrolled in the LUMRYZ REMS to receive LUMRYZ.
Further information is available
at www.LUMRYZREMS.com or
by calling 1-877-453-1029. |
INDICATIONSLUMRYZ (sodium oxybate) for
extended-release oral suspension is a prescription medicine used to
treat the following symptoms in adults with narcolepsy:
- sudden onset of weak or paralyzed muscles (cataplexy)
- excessive daytime sleepiness (EDS)
It is not known if LUMRYZ is safe and effective in people less
than 18 years of age.
Do not take LUMRYZ if you take other sleep
medicines or sedatives (medicines that cause sleepiness), drink
alcohol or have a rare problem called succinic semialdehyde
dehydrogenase deficiency.
Keep LUMRYZ in a safe place to prevent abuse and misuse. Selling
or giving away LUMRYZ 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 LUMRYZ 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 six (6)
hours after taking LUMRYZ. Those activities should not be done
until you know how LUMRYZ affects you.
Falling asleep quickly, including while standing or while
getting up from the bed, has led to falls with injuries that have
required some people to be hospitalized.
LUMRYZ can cause serious side effects, including the
following:
- Breathing problems, including slower
breathing, trouble breathing and/or short periods of not breathing
while sleeping (e.g., sleep apnea). People who already have
breathing or lung problems have a higher chance of having breathing
problems when they take LUMRYZ.
- 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 and difficulty concentrating.
Tell your doctor if you have or had depression or have tried to
harm yourself. Call your doctor right away if you have
symptoms of mental health problems or a change in weight or
appetite.
- Sleepwalking. Sleepwalking can cause
injuries. Call your doctor if you start sleepwalking.
Tell your doctor if you are on a salt-restricted diet or if you
have high blood pressure, heart failure or kidney problems. LUMRYZ
contains a lot of sodium (salt) and may not be right for you.
The most common side effects of LUMRYZ in adults include nausea,
dizziness, bedwetting, headache and vomiting. Your side effects may
increase when you take higher doses of LUMRYZ. LUMRYZ 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
LUMRYZ.
For more information, ask your doctor or pharmacist.
Call your doctor for medical advice about side
effects.
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.
Please see full Prescribing
Information, including BOXED Warning.
About Avadel Pharmaceuticals plcAvadel
Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical
company focused on transforming medicines to transform lives. Our
approach includes applying innovative solutions to the development
of medications that address the challenges patients face with
current treatment options. Avadel’s commercial product, LUMRYZ, was
approved by the U.S. Food & Drug Administration (FDA)
as the first and only once-at-bedtime oxybate for the treatment of
cataplexy or excessive daytime sleepiness (EDS) in adults with
narcolepsy. For more information, please
visit www.avadel.com.
Cautionary Disclosure Regarding Forward-Looking
StatementsThis press release includes “forward-looking
statements” within the meaning of Section 27A of the Securities Act
of 1933 and Section 21E of the Securities Exchange Act of 1934.
These forward-looking statements relate to our future expectations,
beliefs, plans, strategies, objectives, results, conditions,
financial performance, prospects or other events. Such
forward-looking statements include, but are not limited to,
expectations regarding the potential therapeutic benefit of LUMRYZ
and the potential market preference for LUMRYZ. In some cases,
forward-looking statements can be identified by the use of words
such as “will,” “may,” “could,” “believe,” “expect,” “look
forward,” “on track,” “guidance,” “anticipate,” “estimate,”
“project,” “next steps” and similar expressions and the negatives
thereof (if applicable).
The Company’s forward-looking statements are based on estimates
and assumptions that are made within the bounds of our knowledge of
our business and operations and that we consider reasonable.
However, the Company’s business and operations are subject to
significant risks, and, as a result, there can be no assurance that
actual results and the results of the company’s business and
operations will not differ materially from the results contemplated
in such forward-looking statements. Factors that could cause actual
results to differ from expectations in the Company’s
forward-looking statements include the risks and uncertainties
described in the “Risk Factors” section of Part I, Item 1A of the
Company’s Annual Report on Form 10-K for the year
ended December 31, 2022, which was filed with
the Securities and Exchange Commission (SEC)
on March 29, 2023, and subsequent SEC filings.
Forward-looking statements speak only as of the date they are
made and are not guarantees of future performance. Accordingly, you
should not place undue reliance on forward-looking statements. The
Company does not undertake any obligation to publicly update or
revise our forward-looking statements, except as required by
law.
Investor Contact:Courtney TurianoStern Investor
Relations, Inc.Courtney.Turiano@sternir.com(212)
698-8687
Media Contact:Lesley StanleyReal
Chemistrylestanley@realchemistry.com(609)
273-3162
References*Basseti CLA, Kallweit U,
Vignatelli L, et al. European guideline and expert statements on
the management of narcolepsy in adults and children. Euro J
Neurol. 2012;28(9):2815-2830.
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