Avadel Pharmaceuticals plc (Nasdaq: AVDL), a company focused on
transforming medicines to transform lives, today announced new data
supporting the clinical profile for LUMRYZ™ (sodium oxybate) for
extended-release oral suspension (CIII) and patient preference for
a once-nightly oxybate in 12 abstracts, including six oral
presentations, at SLEEP 2023, the 37th annual joint meeting of the
American Academy of Sleep Medicine and the Sleep Research Society,
being held from June 3-7, 2023 in Indianapolis.
LUMRYZ (previously known as FT218), is an 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. LUMRYZ was granted U.S. Food and
Drug Administration (FDA) approval in May 2023, and is the first
and only FDA approved once-at-bedtime oxybate for people living
with narcolepsy. LUMRYZ was granted Orphan Drug Exclusivity, as the
Office of Orphan Product Development identified LUMRYZ to be
clinically superior to immediate release oxybates based upon the
major contribution to patient care that LUMRYZ provides by way of
its once-nightly dosing.
“We are thrilled to attend SLEEP 2023 and present a broad range
of data further supporting the clinical value proposition of LUMRYZ
as a once-nightly option to manage EDS and cataplexy in narcolepsy,
including data from our RESTORE and REST-ON trials,” said Jennifer
Gudeman, PharmD, Senior Vice President, Medical and Clinical
Affairs of Avadel. “Additionally, presentations at this year’s
conference also include insights gleaned from MyNarcolepsyTeam,
where people with narcolepsy reported the need for relief from both
daytime and nighttime symptoms. With final approval of LUMRYZ,
we’re proud to offer the narcolepsy community a once-at-bedtime
treatment option for cataplexy or excessive daytime sleepiness
(EDS) that removes the burden of twice-nightly dosing and provides
the opportunity for an uninterrupted night’s sleep.”
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:
- An oral presentation demonstrating that treatment with LUMRYZ
at all tested doses in the completed pivotal Phase 3 REST-ON
clinical resulted in clinically significant improvement in EDS and
cataplexy according to American Academy of Sleep Medicine
(AASM)-established criteria.
- Two posters with results from an online survey given to U.S.
members of the MyNarcolepsyTeam, a social network of ~10,000
members living with narcolepsy, where patients reported
experiencing an initial under-diagnosis or misdiagnosis with a
disorder other than narcolepsy and an extreme or very severe impact
of narcolepsy on daily life, as well as experiencing and seeking
relief from both daytime and nighttime symptoms and needing
multiple medications to address both symptoms.
- Two oral presentations and one poster with interim analyses
from the ongoing RESTORE open-label extension/switch study of
LUMRYZ, showing: patient preference for once-nightly dosing of
LUMRYZ and high treatment burden with twice-nightly
immediate-release oxybate; that LUMRYZ is generally well-tolerated
with few patients discontinuing due to adverse reactions; and
successful titration of LUMRYZ to a therapeutic and tolerable dose
within one month.
- Post-hoc analyses reinforcing positive data from the REST-ON in
one oral presentation and two posters, demonstrating greater weight
loss and improvements in cataplexy and EDS in patients who received
LUMRYZ compared with placebo.
All abstracts have been published in an online supplement in the
journal Sleep. Presentation details are as follows:
Title |
Session |
Presenter |
Date and Time |
Oral Presentations (also available as
posters) |
Patient Preferences and Nocturnal Experiences With Oxybate Therapy
for Narcolepsy: RESTORE Study Interim Analysis |
O-04P-35, poster #316 |
John Harsh |
June 5th 10:30 – 10:45 a.m. ET |
Characterization of Patients with Narcolepsy Treated vs Not Treated
with Sodium Oxybate: A Propensity Score-Matched Cohort Study |
O-04P-35, poster #318 |
Melissa Lipford |
June 5th 10:45 – 11:00 a.m. ET |
Long-Term Safety of Once-Nightly Oxybate for Narcolepsy: RESTORE
Study Interim Analysis of Data |
O-31P-35, poster #315 |
John Harsh |
June 7th 3:15 – 3:30 p.m. ET |
Sodium Oxybate Treatment Patterns in Narcolepsy Patients: A
Propensity Score–Matched Cohort Study Subanalysis |
O-31P-35, poster #317 |
Lois Krahn |
June 7th 3:30 – 3:45 p.m. ET |
Cataplexy Response With FT218 (Once-Nightly Sodium Oxybate): Post
Hoc Responder Analysis From the Phase 3 Rest-ON Clinical Trial |
O-31P-35, poster #319 |
Michael Thorpy |
June 7th 3:45 – 4:00 p.m. ET |
Application of AASM Clinical Significance Thresholds to
Once-Nightly Sodium Oxybate for Improvement in Narcolepsy
Symptoms |
O-31P-35, poster #320 |
Thomas Roth |
June 7th 4:00 – 4:15 p.m. ET |
Poster Presentations |
Path to Diagnosis and Impact of Narcolepsy on Quality of Life: A
Survey of People Living With Narcolepsy |
P-13, poster #233 |
Anne Marie Morse |
June 5th 12:00 – 1:15 p.m. ET |
Demographic Characteristics and Comorbidities of Patients with
Narcolepsy: A Propensity Score-Matched Cohort Study |
P-13, poster #232 |
Melissa Lipford |
June 5th 5:00 – 6:00 p.m. ET |
Understanding Narcolepsy Treatments From the Patient’s Perspective:
A Survey of People Living With Narcolepsy |
P-13, poster #244 |
Matthew Horsnell |
June 5th 5:00 – 6:00 p.m. ET |
Characterization of Patients Who Had ≥5% Weight Loss With FT218
(Once-Nightly Sodium Oxybate): Post Hoc Analysis From REST-ON |
P-35, poster #229 |
Thomas Roth |
June 6th 5:00 – 6:00 p.m. ET |
Dose Titration of Once-Nightly Sodium Oxybate: Analysis of Interim
Data From RESTORE |
P-35, poster #283 |
Jennifer Gudeman |
June 6th 5:00 – 6:00 p.m. ET |
Improvement in Sleep Latency With FT218 (Once-Nightly Sodium
Oxybate): Analysis From the Phase 3 REST-ON Clinical Trial |
P-35, poster #300 |
Maurice Ohayon |
June 6th 5:00 – 6:00 p.m. ET |
In addition to Avadel’s oral and poster presentations, the
Company will support a symposium titled “Addressing Unmet Medical
Needs and Introduction of LUMRYZ, a New Narcolepsy Treatment” on
Tuesday, June 6, 11:45 a.m. – 12:45 p.m. ET.
The symposium will feature panel presentations from 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. In addition, the panel
will include a person with narcolepsy, enrolled in the open-label
RESTORE study since September 2020, who will speak about her
clinical trial experience.
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.
About LUMRYZ™ (sodium oxybate) for extended-release oral
suspension LUMRYZ, 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.
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:Natalie RubinoReal
Chemistrynrubino@realchemistry.com
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