Avadel Pharmaceuticals Announces Publication of RESTORE Data Highlighting Challenges with Twice-Nightly Oxybates and Strong Patient Preference for Once-Nightly LUMRYZ™ Dosing (sodium oxybate) Extended-Release Oral Suspension (CIII)
Avadel Pharmaceuticals (Nasdaq: AVDL) announced the publication of final data from the RESTORE study, highlighting the advantages of LUMRYZ, their once-nightly oxybate treatment for narcolepsy, over twice-nightly alternatives. Key findings include:
- 94% of participants preferred LUMRYZ's once-nightly regimen
- 91% reported better sleep through the night with LUMRYZ
- 93% would recommend LUMRYZ to others with narcolepsy
- 79% were very satisfied with LUMRYZ compared to previous treatments
The study also revealed challenges with twice-nightly oxybates, including missed doses, grogginess, and the need for assistance in taking the second dose. LUMRYZ, approved by the FDA in 2023, addresses these issues as the first and only once-nightly oxybate treatment for narcolepsy.
Avadel Pharmaceuticals (Nasdaq: AVDL) ha annunciato la pubblicazione dei dati finali dello studio RESTORE, evidenziando i vantaggi di LUMRYZ, il loro trattamento di ossibato una volta per notte per la narcolessia, rispetto alle alternative di ossibato a dose doppia per notte. I risultati chiave includono:
- Il 94% dei partecipanti ha preferito il regime notturno di LUMRYZ
- Il 91% ha riportato un sonno migliore durante la notte con LUMRYZ
- Il 93% raccomanderebbe LUMRYZ ad altri con narcolessia
- Il 79% era molto soddisfatto di LUMRYZ rispetto ai trattamenti precedenti
Lo studio ha anche rivelato le difficoltà associate agli ossibati a dose doppia per notte, tra cui dosi mancate, sonnolenza e necessità di assistenza per assumere la seconda dose. LUMRYZ, approvato dalla FDA nel 2023, affronta questi problemi come il primo e unico trattamento di ossibato per narcolessia da assumere una volta per notte.
Avadel Pharmaceuticals (Nasdaq: AVDL) anunció la publicación de los datos finales del estudio RESTORE, destacando las ventajas de LUMRYZ, su tratamiento de oxibato de una sola vez por noche para la narcolepsia, frente a las alternativas de dos veces por noche. Los hallazgos clave incluyen:
- El 94% de los participantes prefirió el régimen de LUMRYZ de una sola vez por noche
- El 91% informó de un mejor sueño durante la noche con LUMRYZ
- El 93% lo recomendaría a otros con narcolepsia
- El 79% estaba muy satisfecho con LUMRYZ en comparación con tratamientos anteriores
El estudio también reveló los desafíos de los oxibatos tomados dos veces por noche, incluyendo dosis omitidas, somnolencia y la necesidad de asistencia para tomar la segunda dosis. LUMRYZ, aprobado por la FDA en 2023, aborda estos problemas como el primer y único tratamiento de oxibato para la narcolepsia administrado una vez por noche.
Avadel Pharmaceuticals (Nasdaq: AVDL)는 RESTORE 연구의 최종 데이터를 발표하며, 나르콜렙시를 위한 일회 복용제인 LUMRYZ의 장점을 밤에 두 번 복용하는 대안에 비해 강조했습니다. 주요 발견 사항은 다음과 같습니다:
- 참가자의 94%가 LUMRYZ의 일회 복용 요법을 선호했습니다.
- 91%가 LUMRYZ로 밤새 더 나은 수면을 경험했습니다.
- 93%가 나르콜렙시가 있는 다른 사람에게 LUMRYZ를 추천할 것이라고 답했습니다.
- 79%는 이전 치료와 비교하여 LUMRYZ에 매우 만족했습니다.
이 연구는 또한 복용이 두 번인 옥시베이트의 문제점, 즉 복용 누락, 졸린 느낌, 두 번째 복용을 위한 도움 필요성을 밝혔습니다. 2023년 FDA에 의해 승인된 LUMRYZ는 이러한 문제를 해결하며 나르콜렙시를 위한 유일한 일회 복용 옥시베이트 치료제입니다.
Avadel Pharmaceuticals (Nasdaq: AVDL) a annoncé la publication des données finales de l'étude RESTORE, mettant en lumière les avantages de LUMRYZ, leur traitement d'oxybate administré une fois par nuit pour la narcolepsie, par rapport aux alternatives administrées deux fois par nuit. Les principaux résultats comprennent :
- 94 % des participants ont préféré le régime nocturne de LUMRYZ
- 91 % ont signalé un meilleur sommeil pendant la nuit avec LUMRYZ
- 93 % recommanderaient LUMRYZ à d'autres personnes atteintes de narcolepsie
- 79 % étaient très satisfaits de LUMRYZ par rapport aux traitements précédents
L'étude a également révélé des défis liés aux oxybates administrés deux fois par nuit, notamment des doses manquées, de la somnolence et le besoin d'assistance pour prendre la deuxième dose. LUMRYZ, approuvé par la FDA en 2023, traite ces problèmes en tant que premier et unique traitement d'oxybate administré une fois par nuit pour la narcolepsie.
Avadel Pharmaceuticals (Nasdaq: AVDL) gab die Veröffentlichung der endgültigen Daten aus der RESTORE-Studie bekannt, in der die Vorteile von LUMRYZ, ihrer einmal nächtlichen Oxybatbehandlung für Narkolepsie, gegenüber zweimal nächtlichen Alternativen hervorgehoben werden. Zu den wichtigsten Ergebnissen gehören:
- 94 % der Teilnehmer bevorzugten das einmalige nächtliche Regime von LUMRYZ
- 91 % berichteten von einem besseren Schlaf in der Nacht mit LUMRYZ
- 93 % würden LUMRYZ anderen mit Narkolepsie empfehlen
- 79 % waren mit LUMRYZ im Vergleich zu vorherigen Behandlungen sehr zufrieden
Die Studie zeigte auch Herausforderungen bei doppelt nächtlichen Oxybaten, darunter versäumte Dosen, Schläfrigkeit und die Notwendigkeit einer Unterstützung bei der Einnahme der zweiten Dosis. LUMRYZ, das 2023 von der FDA genehmigt wurde, adressiert diese Probleme als die erste und einzige einmal nächtliche Oxybatbehandlung für Narkolepsie.
- FDA approval of LUMRYZ as the first and only once-nightly oxybate treatment for narcolepsy
- 94% of study participants preferred LUMRYZ's once-nightly regimen over twice-nightly alternatives
- 91% of participants reported better sleep through the night with LUMRYZ
- 93% of participants would recommend LUMRYZ to others with narcolepsy
- 79% were very satisfied with LUMRYZ compared to previous narcolepsy treatments
- 91% reported better adherence to LUMRYZ's recommended medication schedule
- None.
Insights
The RESTORE study results for LUMRYZ are highly positive for Avadel Pharmaceuticals. Key findings include:
94% of participants preferred LUMRYZ's once-nightly dosing over twice-nightly oxybates91% reported better sleep through the night93% would recommend LUMRYZ to others with narcolepsy
These outcomes suggest strong patient satisfaction and potential for market penetration. The data highlighting challenges with twice-nightly oxybates (e.g.,
This publication bolsters Avadel's market position in the narcolepsy treatment sector. With LUMRYZ as the first and only FDA-approved once-nightly oxybate, Avadel has a significant competitive edge. The strong preference data (
The RESTORE study results reveal a clear market opportunity for LUMRYZ. The high preference rates and reported improvements in daily functioning indicate strong product-market fit. The data highlighting challenges with existing twice-nightly oxybates (e.g.,
– Survey data demonstrate
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DUBLIN, Sept. 03, 2024 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today announced the publication of final data from the RESTORE open-label/switch study, demonstrating that people with narcolepsy prefer once-at-bedtime LUMRYZ versus a twice-nightly immediate-release oxybate treatment option. The U.S. Food and Drug Administration approved LUMRYZ in 2023 as the first and only once-nightly oxybate, which marked a significant milestone for people with narcolepsy, as twice-nightly oxybates were the only other oxybate options available for more than 20 years.
The paper, titled “RESTORE: Once-Nightly Oxybate Dosing Preference and Nocturnal Experience With Twice-Nightly Oxybates,” was published online in Sleep Medicine: X. Initial and end-of-study survey data from RESTORE were previously presented in April 2022 at the American Academy of Neurology Annual Meeting and in June 2024 at SLEEP 2024.
“Twice-nightly oxybates can present significant treatment burdens and adherence concerns given the required middle-of-the-night awakening for a second dose. RESTORE study participants overwhelmingly preferred the once-nightly dosing regimen of LUMRYZ over twice-nightly oxybates. RESTORE further highlighted the inconvenience with the middle-of-the-night dose, as well as the associated grogginess and unsteadiness the following morning if the dose was taken late, and the worsening of symptoms when the dose was missed,” said Asim Roy, M.D., co-author of the paper and RESTORE investigator, and Medical Director of the Ohio Sleep Medicine Institute. “With LUMRYZ, the majority of switch participants who participated in the survey reported improvements in their ability to get through the day without falling asleep, being better able to sleep through the night, and accomplish more in professional and social settings.”
As part of the multicenter, open-label Phase 3 RESTORE extension/switch study, participants with narcolepsy type 1 or 2 who switched from a twice-nightly oxybate to once-nightly LUMRYZ completed a nocturnal adverse events questionnaire at baseline, a patient preference questionnaire after three months of taking LUMRYZ, and an end-of-study questionnaire.
- Of the 129 switch participants who completed the nocturnal adverse events questionnaire at baseline:
69% reported missing their second oxybate dose, and of those participants,80% felt that control of their symptoms was worse the next day compared to days after which they had taken both doses as prescribed- More than half (
51% ) of the 51 participants who took their second nightly oxybate dose more than four hours after the first dose reported feeling somewhat to extremely groggy or unsteady the next morning 92% reported getting out of bed after taking their second dose of oxybate, with7.5% of those reporting falling after waking up for the second dose, and4.2% reporting injuries23% of participants stated they required another person to wake with them in the middle of the night to ensure they took the second dose of their twice-nightly oxybate
- Of the 98 switch participants who completed the patient preference questionnaire,
94% preferred once-at-bedtime LUMRYZ to twice-nightly oxybate dosing - Of the 68 switch participants who completed the end-of-study questionnaire:
79% were very satisfied with LUMRYZ compared to other narcolepsy treatments they had previously taken93% would recommend LUMRYZ to a family member or friend with narcolepsy91% said they were better able to sleep through the night since starting treatment with LUMRYZ91% said they were better able to follow the recommended medication schedule of LUMRYZ than their previous oxybate
“The RESTORE study, which was conducted for more than three years, allowed investigators switching participants from twice-nightly, first-generation oxybates to understand the myriad challenges associated with chronically taking a middle-of-the-night dose of medication. Nearly 1 out of 4 switch participants reported needing someone else to wake up with them to take the middle-of-the-night dose. Once-at-bedtime LUMRYZ avoids the dosing burden of a twice-nightly oxybate, which as RESTORE affirms, is experienced in multiple negative aspects for patients, which can be avoided with LUMRYZ. Not surprisingly, among those switching, more than nine in 10 would recommend LUMRYZ to a family member or friend with narcolepsy,” said Jennifer Gudeman, Pharm.D., Senior Vice President, Medical and Clinical Affairs of Avadel. “Data from RESTORE demonstrate LUMRYZ was well tolerated, with a low rate of discontinuation due to adverse events, and further confirmed the long-term safety and tolerability of this innovative treatment option.”
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.
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. |
INDICATIONS
LUMRYZ (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 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 excessive daytime sleepiness (EDS) in adults with narcolepsy. For more information, please visit www.avadel.com.
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 potential therapeutic benefit of LUMRYZ in treating narcolepsy-related EDS or cataplexy, including potential impact on patient weight; and the results and analysis of the Phase 3 REST-ON trial, including the details and content thereof. 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, 2023, which was filed with the Securities and Exchange Commission (SEC) on February 29, 2024, 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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FAQ
What are the key findings of the RESTORE study for Avadel's LUMRYZ (AVDL)?
How does LUMRYZ (AVDL) differ from other oxybate treatments for narcolepsy?
What challenges did the RESTORE study reveal about twice-nightly oxybate treatments for narcolepsy (AVDL)?
When was LUMRYZ (AVDL) approved by the FDA for narcolepsy treatment?
