Avadel Announces Publications in CNS Drugs Supplement Highlighting Key Clinical Issues for People with Narcolepsy and Their Clinicians
Avadel Pharmaceuticals (Nasdaq: AVDL) has announced the publication of six manuscripts in a CNS Drugs supplement titled 'A New Dawn in the Management of Narcolepsy'. The publications focus on clinical management, shared decision-making, and goal setting for narcolepsy patients and their healthcare providers.
Dr. Michael J. Thorpy, Director at the Sleep-Wake Disorders Center at Montefiore Medical Center and a Phase 3 REST-ON trial investigator for LUMRYZ™, served as guest editor. The supplement emphasizes sodium oxybate therapy and includes new analyses of efficacy onset and practical considerations for once-nightly treatment.
Dr. Jennifer Gudeman, SVP of Medical and Clinical Affairs at Avadel, highlighted that while sodium oxybate has been FDA-approved for over 20 years, it remains underutilized due to burdensome first-generation dosing. The publications detail LUMRYZ's data, addressing nighttime sleep disturbances and treatment expectations, noting that patients may experience initial effects within 1-2 weeks, with increasing improvements from week three onward.
Avadel Pharmaceuticals (Nasdaq: AVDL) ha annunciato la pubblicazione di sei manoscritti in un supplemento di CNS Drugs intitolato 'Una Nuova Alba nella Gestione della Narcolessia'. Le pubblicazioni si concentrano sulla gestione clinica, sulla decisione condivisa e sulla definizione degli obiettivi per i pazienti affetti da narcolessia e i loro fornitori di assistenza sanitaria.
Il Dr. Michael J. Thorpy, Direttore del Centro Disturbi del Sonno e della Veglia presso il Montefiore Medical Center e investigatore del trial di Fase 3 REST-ON per LUMRYZ™, ha svolto il ruolo di editor ospite. Il supplemento enfatizza la terapia con sodio ossibato e include nuove analisi sull'insorgenza dell'efficacia e considerazioni pratiche per un trattamento notturno una volta al giorno.
La Dr.ssa Jennifer Gudeman, SVP di Affari Medici e Clinici presso Avadel, ha sottolineato che, sebbene il sodio ossibato sia stato approvato dalla FDA per oltre 20 anni, rimane sottoutilizzato a causa di un dosaggio di prima generazione gravoso. Le pubblicazioni dettagliano i dati di LUMRYZ, affrontando le interruzioni del sonno notturno e le aspettative di trattamento, notando che i pazienti possono sperimentare effetti iniziali entro 1-2 settimane, con miglioramenti crescenti a partire dalla terza settimana.
Avadel Pharmaceuticals (Nasdaq: AVDL) ha anunciado la publicación de seis manuscritos en un suplemento de CNS Drugs titulado 'Un Nuevo Amanecer en el Manejo de la Narcolepsia'. Las publicaciones se centran en la gestión clínica, la toma de decisiones compartidas y el establecimiento de objetivos para los pacientes con narcolepsia y sus proveedores de atención médica.
El Dr. Michael J. Thorpy, Director del Centro de Trastornos del Sueño y la Vigilia en el Montefiore Medical Center y un investigador del ensayo de Fase 3 REST-ON para LUMRYZ™, actuó como editor invitado. El suplemento enfatiza la terapia con oxibato de sodio e incluye nuevos análisis sobre el inicio de la eficacia y consideraciones prácticas para un tratamiento nocturno una vez al día.
La Dra. Jennifer Gudeman, SVP de Asuntos Médicos y Clínicos en Avadel, destacó que, aunque el oxibato de sodio ha sido aprobado por la FDA durante más de 20 años, sigue siendo subutilizado debido a la carga del dosaje de primera generación. Las publicaciones detallan los datos de LUMRYZ, abordando las interrupciones del sueño nocturno y las expectativas de tratamiento, señalando que los pacientes pueden experimentar efectos iniciales dentro de 1-2 semanas, con mejoras crecientes a partir de la tercera semana.
Avadel Pharmaceuticals (Nasdaq: AVDL)는 '기면증 관리의 새로운 새벽'이라는 제목의 CNS Drugs 보충판에 여섯 개의 원고가 발표되었다고 발표했습니다. 이 출판물은 기면증 환자와 그들의 의료 제공자를 위한 임상 관리, 공유 의사 결정 및 목표 설정에 중점을 두고 있습니다.
몬테피오르 메디컬 센터의 수면-각성 장애 센터 소장인 마이클 J. 토르피 박사는 LUMRYZ™의 3상 REST-ON 시험 조사관으로서 객원 편집자로 활동했습니다. 이 보충판은 나트륨 옥시베이트 요법을 강조하며, 하루에 한 번 밤에 치료를 위한 효능 발현과 실제 고려 사항에 대한 새로운 분석을 포함합니다.
Avadel의 의학 및 임상 업무 수석 부사장인 제니퍼 구데만 박사는 나트륨 옥시베이트가 FDA 승인을 받은 지 20년이 넘었지만, 첫 번째 세대의 복잡한 용량으로 인해 여전히 충분히 활용되지 않고 있다고 강조했습니다. 이 출판물은 LUMRYZ의 데이터를 자세히 설명하며, 야간 수면 방해 및 치료 기대를 다루고 있으며, 환자들이 1-2주 이내에 초기 효과를 경험할 수 있으며, 3주째부터 점차적인 개선이 이루어질 수 있음을 언급하고 있습니다.
Avadel Pharmaceuticals (Nasdaq: AVDL) a annoncé la publication de six manuscrits dans un supplément de CNS Drugs intitulé 'Une Nouvelle Aube dans la Gestion de la Narcolepsie'. Les publications se concentrent sur la gestion clinique, la prise de décision partagée et la définition des objectifs pour les patients atteints de narcolepsie et leurs prestataires de soins de santé.
Le Dr Michael J. Thorpy, directeur du Centre des troubles du sommeil et de l'éveil au Montefiore Medical Center et investigateur de l'essai de Phase 3 REST-ON pour LUMRYZ™, a agi en tant qu'éditeur invité. Le supplément met l'accent sur la thérapie au sodium oxybate et inclut de nouvelles analyses sur le début de l'efficacité et des considérations pratiques pour un traitement nocturne une fois par jour.
La Dr Jennifer Gudeman, SVP des affaires médicales et cliniques chez Avadel, a souligné que bien que le sodium oxybate soit approuvé par la FDA depuis plus de 20 ans, il reste sous-utilisé en raison d'un dosage de première génération lourd. Les publications détaillent les données de LUMRYZ, abordant les perturbations du sommeil nocturne et les attentes de traitement, notant que les patients peuvent ressentir des effets initiaux dans les 1 à 2 semaines, avec des améliorations croissantes à partir de la troisième semaine.
Avadel Pharmaceuticals (Nasdaq: AVDL) hat die Veröffentlichung von sechs Manuskripten in einem CNS Drugs Supplement mit dem Titel 'Ein Neuer Morgen im Management der Narkolepsie' angekündigt. Die Veröffentlichungen konzentrieren sich auf die klinische Betreuung, gemeinsame Entscheidungsfindung und Zielsetzung für Narkolepsie-Patienten und deren Gesundheitsdienstleister.
Dr. Michael J. Thorpy, Direktor des Schlaf-Wach-Störungen Zentrums am Montefiore Medical Center und Prüfer der Phase-3-Studie REST-ON für LUMRYZ™, fungierte als Gastredakteur. Das Supplement betont die Therapie mit Natrium-Oxybat und enthält neue Analysen zur Wirksamkeit und praktische Überlegungen für die einmal tägliche Behandlung.
Dr. Jennifer Gudeman, SVP für Medizinische und Klinische Angelegenheiten bei Avadel, hob hervor, dass Natrium-Oxybat seit über 20 Jahren von der FDA zugelassen ist, jedoch aufgrund der belastenden Dosierung der ersten Generation unterutilisiert bleibt. Die Veröffentlichungen erläutern die Daten zu LUMRYZ, behandeln nächtliche Schlafstörungen und Behandlungserwartungen und weisen darauf hin, dass Patienten innerhalb von 1-2 Wochen erste Effekte erleben können, mit zunehmenden Verbesserungen ab der dritten Woche.
- Publication of clinical data supporting LUMRYZ's efficacy and treatment protocol
- Potential market opportunity due to current underutilization of sodium oxybate therapy
- Demonstrated treatment effects beginning within 1-2 weeks
- Patients may experience transient tolerability effects during initial treatment
DUBLIN, March 25, 2025 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today announced the publication of six manuscripts covering clinical management, shared decision-making and goal setting for people with narcolepsy and their clinicians in a CNS Drugs supplement titled “A New Dawn in the Management of Narcolepsy”.
“I am delighted to serve as guest editor for the recently published CNS Drugs supplement, focused on key clinical issues for people with narcolepsy and their clinicians with a particular emphasis on sodium oxybate therapy, an underutilized treatment option,” said Michael J. Thorpy, M.D., an investigator for the Phase 3 REST-ON trial of LUMRYZ™, Director at the Sleep-Wake Disorders Center at Montefiore Medical Center and Professor of Neurology at the Albert Einstein College of Medicine. “This supplement provides clinicians with important information about how to optimize therapy to help address narcolepsy symptoms, including new analyses of onset of efficacy and practical considerations for use of a once-nightly sodium oxybate therapy, to enable clinicians to set proper expectations when initiating LUMRYZ therapy.”
Summary of Papers in CNS Drugs Supplement
| Paper Title | Authors/Synopsis |
| Narcolepsy: Beyond the Classic Pentad | Authored by Anne Marie Morse, D.O., Director of Pediatric Neurology at Geisinger Medical Center and others, this paper addresses the full 24-hour impact and symptom burden that patients experience, as well as the need for an individualized treatment approach and care considerations that extend beyond the hallmark features of the disease. |
| A Survey of People Living With Narcolepsy in the USA: Path to Diagnosis, Quality of Life, and Treatment Landscape From the Patient’s Perspective | Authored by Luis E. Ortiz, M.D., Assistant Professor of Pediatrics at Johns Hopkins Medicine and others, the survey reports data from 110 people living with narcolepsy. Among the key findings: nearly three-fourths ( |
| Therapeutic Use of γ-Hydroxybutyrate: History and Clinical Utility of Oxybates and Considerations of Once and Twice-Nightly Dosing in Narcolepsy | Authored by Thomas Roth, Ph.D., founder of the Sleep Disorders and Research Center at Henry Ford Health, this paper provides a 50-year historical overview of γ-Hydroxybutyrate (GHB) use in narcolepsy, including more than two decades of safe and appropriate use as a treatment. The paper also includes data on changes to sleep architecture that occur during treatment with LUMRYZ, aligning with a more “normal” sleep pattern in people with narcolepsy vs. chronic middle-of-the-night awakening. |
| Assessing Early Efficacy After Initiation of Once-Nightly Sodium Oxybate (ON-SXB; FT218) in Participants With Narcolepsy Type 1 or 2: A Post Hoc Analysis From the Phase 3 REST-ON Trial | Authored by Lois E. Krahn, M.D., Professor of Psychiatry at the Mayo Clinic College of Medicine and others, this analysis demonstrates significant improvements in certain narcolepsy symptoms (including sleep quality and refreshing nature of sleep) with LUMRYZ versus placebo one to two weeks after treatment initiation, with increasing improvement from week 3 onward. |
| Efficacy of Once-Nightly Sodium Oxybate in Patients With Narcolepsy: Post Hoc Analyses of Sensitivity, Effect Size, and Numbers Needed to Treat From the Phase 3 REST-ON Trial | Also authored by Dr. Roth and others, this paper provides further validation of the robust efficacy once-nightly LUMRYZ demonstrated in the REST-ON trial across primary and secondary endpoints. |
| Sodium Oxybate: Practical Considerations and Patient Perspectives | Authored by Maggie Lavender, M.S.N., of Comprehensive Sleep Medicine Associates in Houston, Texas and others, this paper includes illustrative clinical case scenarios of two people with narcolepsy, providing clinicians with a proposed framework to optimize discussions with patients and demystify sodium oxybate treatment, providing practical tips and support for initiating and maintaining therapy. |
“Despite sodium oxybate being FDA-approved for more than two decades and strongly recommended by the American Academy of Sleep Medicine for nearly 20 years, this therapy remains underutilized – in part due to the historical burdensome dosing for first-generation oxybates. The title of this supplement, A New Dawn in the Management of Narcolepsy, reflects that the patient community no longer needs to accept the status quo,” said Jennifer Gudeman, Pharm.D., Senior Vice President, Medical and Clinical Affairs of Avadel.
“These publications illustrate the impact of nighttime sleep disturbances on narcolepsy patients, highlight the robust data supporting LUMRYZ, and perhaps most importantly, demystify oxybate therapy, with a particular focus on LUMRYZ initiation and maintenance of therapy,” continued Dr. Gudeman. “Patients taking once-nightly LUMRYZ may experience transient tolerability effects, coupled with some degree of symptom relief after one to two weeks and increasing improvement from week three onward, which may help clinicians set expectations with patients to improve medication adherence.”
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 treatment for cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. On October 16, 2024, LUMRYZ was additionally approved as a once-at-bedtime treatment for cataplexy or EDS in patients 7 years of age and older 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. A second 7-year period of Orphan Drug Exclusivity was granted with the approval of the expanded indication in October 2024.
INDICATIONS
LUMRYZ (sodium oxybate) for extended-release oral suspension is a prescription medicine used to treat the following symptoms in patients 7 years of age and older with narcolepsy:
- sudden onset of weak or paralyzed muscles (cataplexy)
- excessive daytime sleepiness (EDS)
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. |
Do not take LUMRYZ if you take or your child takes 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 or your child 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 or your child have or had depression or have tried to harm yourself. Call your doctor right away if you or your child have 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 start sleepwalking.
Tell your doctor if you or your child are on a salt-restricted diet or 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. The most common side effects in children include nausea, bedwetting, vomiting, headache, decreased weight, decreased appetite, dizziness, and sleepwalking. 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 plc
Avadel 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 EDS in both adults and pediatrics with narcolepsy. For more information, please visit www.avadel.com.
Avadel intends to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company’s Investor Relations website, in addition to following the Company’s press releases, SEC filings, public conference calls, presentations, and webcast.
Cautionary Disclosure Regarding Forward-Looking Statements
This 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 safety, tolerability and potential therapeutic benefit of LUMRYZ; and expectations regarding potential benefits of the publications. 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, 2024, which was filed with the Securities and Exchange Commission (SEC) on March 3, 2025, 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:
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Precision AQ
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(212) 698-8696
Media Contact:
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FAQ
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