Avadel and nference Announce Publication of Real-World Data on Sodium Oxybate Treatment Patterns in the Journal of Clinical Neuroscience
Avadel Pharmaceuticals (AVDL) and nference published real-world data analysis on sodium oxybate treatment patterns in narcolepsy patients in the Journal of Clinical Neuroscience. The study analyzed 4,387 Mayo Clinic patients with narcolepsy, including 351 treated with immediate-release sodium oxybate.
Key findings revealed:
- No significant differences in comorbidity rates between treated and untreated groups, including hypertension (21.1% vs 21.4%) and coronary artery disease (7.1% in both groups)
- Approximately one-third of patients discontinued treatment, primarily due to lack of efficacy
- Inability to wake up was the most common reason for missing the second nightly dose
- Both groups showed high rates of insomnia (approximately 43%)
The study highlights challenges with middle-of-the-night sodium oxybate dosing and reduced efficacy when second doses are missed, suggesting potential underutilization of the therapy due to dosing requirements.
Avadel Pharmaceuticals (AVDL) e nference hanno pubblicato un'analisi dei dati reali sui modelli di trattamento con ossibato di sodio in pazienti con narcolessia, sul Journal of Clinical Neuroscience. Lo studio ha analizzato 4.387 pazienti con narcolessia della Mayo Clinic, di cui 351 trattati con ossibato di sodio a rilascio immediato.
I risultati principali hanno evidenziato:
- Nessuna differenza significativa nei tassi di comorbidità tra i gruppi trattati e non trattati, inclusa l'ipertensione (21,1% vs 21,4%) e la malattia coronarica (7,1% in entrambi i gruppi)
- Circa un terzo dei pazienti ha interrotto il trattamento, principalmente per inefficacia
- L'incapacità di svegliarsi è stata la causa più comune per saltare la seconda dose notturna
- Entrambi i gruppi hanno mostrato alti tassi di insonnia (circa il 43%)
Lo studio evidenzia le difficoltà legate alla somministrazione notturna di ossibato di sodio e la ridotta efficacia quando si saltano le seconde dosi, suggerendo un possibile sotto-utilizzo della terapia a causa delle modalità di dosaggio.
Avadel Pharmaceuticals (AVDL) y nference publicaron un análisis de datos del mundo real sobre los patrones de tratamiento con oxibato de sodio en pacientes con narcolepsia, en el Journal of Clinical Neuroscience. El estudio analizó a 4,387 pacientes con narcolepsia de la Mayo Clinic, incluyendo 351 tratados con oxibato de sodio de liberación inmediata.
Los hallazgos clave revelaron:
- No hubo diferencias significativas en las tasas de comorbilidades entre los grupos tratados y no tratados, incluyendo hipertensión (21.1% vs 21.4%) y enfermedad coronaria (7.1% en ambos grupos)
- Aproximadamente un tercio de los pacientes interrumpió el tratamiento, principalmente por falta de eficacia
- La incapacidad para despertarse fue la razón más común para omitir la segunda dosis nocturna
- Ambos grupos mostraron altas tasas de insomnio (aproximadamente 43%)
El estudio destaca los desafíos relacionados con la dosificación de oxibato de sodio a mitad de la noche y la reducción de eficacia cuando se omiten las segundas dosis, sugiriendo un posible subuso de la terapia debido a los requisitos de dosificación.
Avadel Pharmaceuticals (AVDL)와 nference가 Journal of Clinical Neuroscience에 기면증 환자의 나트륨 옥시베이트 치료 패턴에 대한 실제 데이터 분석을 발표했습니다. 이 연구는 Mayo Clinic의 4,387명의 기면증 환자를 분석했으며, 그 중 351명은 즉시 방출형 나트륨 옥시베이트로 치료받았습니다.
주요 결과는 다음과 같습니다:
- 고혈압(21.1% 대 21.4%) 및 관상동맥질환(양 그룹 모두 7.1%)을 포함한 치료군과 비치료군 간 공존 질환 비율에 유의미한 차이 없음
- 약 3분의 1의 환자가 주로 효과 부족으로 치료를 중단함
- 두 번째 야간 복용을 놓친 가장 흔한 이유는 깨어나기 어려움
- 두 그룹 모두 약 43%의 높은 불면증 비율을 보임
이 연구는 한밤중 나트륨 옥시베이트 복용의 어려움과 두 번째 복용을 놓칠 경우 효능 감소 문제를 강조하며, 복용 방식 때문에 치료가 충분히 활용되지 못할 가능성을 시사합니다.
Avadel Pharmaceuticals (AVDL) et nference ont publié une analyse de données en conditions réelles sur les schémas de traitement au oxybate de sodium chez des patients atteints de narcolepsie dans le Journal of Clinical Neuroscience. L'étude a analysé 4 387 patients atteints de narcolepsie à la Mayo Clinic, dont 351 traités avec de l'oxybate de sodium à libération immédiate.
Les résultats clés ont révélé :
- Pas de différences significatives dans les taux de comorbidités entre les groupes traités et non traités, y compris l'hypertension (21,1 % vs 21,4 %) et la maladie coronarienne (7,1 % dans les deux groupes)
- Environ un tiers des patients ont arrêté le traitement, principalement en raison d'un manque d'efficacité
- L'incapacité à se réveiller était la raison la plus fréquente pour manquer la deuxième dose nocturne
- Les deux groupes présentaient des taux élevés d'insomnie (environ 43 %)
L'étude met en lumière les difficultés liées à la prise d'oxybate de sodium au milieu de la nuit et la réduction de l'efficacité lorsque la deuxième dose est manquée, suggérant une possible sous-utilisation de la thérapie en raison des contraintes posologiques.
Avadel Pharmaceuticals (AVDL) und nference veröffentlichten eine Analyse von Real-World-Daten zu Behandlungsmustern mit Natriumoxibat bei Patienten mit Narkolepsie im Journal of Clinical Neuroscience. Die Studie analysierte 4.387 Patienten der Mayo Clinic mit Narkolepsie, darunter 351, die mit sofort freisetzendem Natriumoxibat behandelt wurden.
Wesentliche Ergebnisse zeigten:
- Keine signifikanten Unterschiede bei Komorbiditätsraten zwischen behandelten und unbehandelten Gruppen, einschließlich Bluthochdruck (21,1 % vs. 21,4 %) und koronarer Herzkrankheit (jeweils 7,1 %)
- Etwa ein Drittel der Patienten brach die Behandlung hauptsächlich wegen mangelnder Wirksamkeit ab
- Unfähigkeit aufzuwachen war der häufigste Grund, die zweite nächtliche Dosis zu verpassen
- Beide Gruppen wiesen hohe Raten von Schlaflosigkeit auf (etwa 43 %)
Die Studie hebt die Herausforderungen der Mitternachtsdosierung von Natriumoxibat hervor und die verringerte Wirksamkeit bei Auslassen der zweiten Dosis, was auf eine mögliche Unterausnutzung der Therapie aufgrund der Dosierungsanforderungen hinweist.
- Real-world data validates safety profile with no increased cardiovascular risks
- Large-scale study with 4,387 narcolepsy patients provides robust evidence base
- One-third of patients discontinue treatment due to lack of efficacy
- Significant compliance issues with second nightly dose administration
- Current treatment shows underutilization due to dosing challenges
Insights
Publication validates challenges with twice-nightly sodium oxybate, potentially supporting Avadel's alternative formulation for narcolepsy treatment.
This real-world data publication offers modest positive implications for Avadel Pharmaceuticals' strategic positioning in the narcolepsy treatment landscape. The retrospective analysis examined 351 narcolepsy patients treated with immediate-release sodium oxybate compared to a matched untreated cohort using Mayo Clinic's electronic health records.
The findings address two critical aspects relevant to Avadel's business:
- Safety profile validation: No significant differences in comorbidity rates (including cardiovascular conditions) between sodium oxybate users and non-users after statistical adjustment
- Treatment adherence challenges: Approximately one-third of patients discontinued twice-nightly sodium oxybate therapy, with inability to wake up for the middle-of-the-night dose emerging as a significant barrier
The study effectively identifies a market opportunity for improved treatment options. Dr. Gudeman's statement about "first-generation oxybates requiring chronic, middle-of-the-night dosing" strongly suggests Avadel is positioned with a once-nightly alternative that could address these adherence challenges.
This publication provides scientific validation in a peer-reviewed journal that can support physician education and payer conversations about the real-world limitations of existing sodium oxybate therapy. While not announcing new product features or financial impacts, it establishes credibility for Avadel's presumed differentiation strategy in developing therapies that overcome the identified treatment barriers.
-- Retrospective analysis reveals no significant differences in comorbidities in narcolepsy patients treated vs. not treated with sodium oxybate --
-- One third of patients taking immediate-release, twice-nightly sodium oxybate discontinued treatment, most often due to lack of efficacy; inability to wake up was most common reason for missing second nightly dose --
DUBLIN, April 17, 2025 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, and nference, a health tech company dedicated to making biomedical data computable, today announced the publication of real-world data on comorbidities and treatment patterns of people with narcolepsy treated versus not treated with immediate-release sodium oxybate. The paper, titled “Characterization and Treatment Patterns of Patients Treated With Immediate-Release Sodium Oxybate for Narcolepsy: A Propensity-Matched Cohort Study,” was published online in the Journal of Clinical Neuroscience.
A review of de-identified electronic health records (EHR) from patients at Mayo Clinic identified 4,387 individuals living with narcolepsy, of whom 351 had received immediate-release sodium oxybate treatment. This cohort of 351 patients with narcolepsy treated with sodium oxybate was age/sex matched with a cohort of 351 patients with narcolepsy without sodium oxybate treatment. The retrospective analysis was conducted through Mayo Clinic’s Clinical Data Analytics Platform, which leverages nference’s AI software to provide access to curated, deidentified electronic health data in a secure, privacy-protected environment. These types of data provide a more comprehensive view of patient information than claims data alone.
Key findings included:
- No differences between the sodium oxybate treated group and the matched cohort in the rates of the top 10 overall comorbidities (unadjusted analysis), except for idiopathic hypersomnia and fatigue. After adjusting the analysis to reduce the risk of a false positive result, there were no significant differences in the rates of comorbidities between the sodium oxybate cohort and the matched cohort
- Idiopathic hypersomnia was reported in
22.8% of the sodium oxybate treated group versus33.0% of the matched cohort; as IH and narcolepsy are mutually exclusive disorders, these results reflect the frequent diagnostic change with repeat testing
- Idiopathic hypersomnia was reported in
- The rate of hypertension in the sodium oxybate treated group was
21.1% compared to the matched cohort group of21.4% (adjusted P>0.9) - The rate of coronary artery disease was
7.1% in both groups (adjusted P>0.9)
Additional findings from the cohort of 351 patients with narcolepsy treated with twice-nightly sodium oxybate revealed that:
- Approximately 1/3 had documented clinical notes for discontinuation, with lack of efficacy being most common
- Chart review confirmed instances of missing the second dose
- Inability to wake up was the most frequently cited reason
- Next-day consequences of the missed second dose (e.g., cataplexy, lower daytime alertness) were documented
“In this real-world study of de-identified Mayo Clinic EHR data comparing patients treated with sodium oxybate to a matched narcolepsy cohort without sodium oxybate, we see comparable rates of comorbidities, including hypertension and coronary artery disease. Other relevant findings included high rates of insomnia in both groups (approximately
“This is the second study for which we have collaborated with nference to generate real-world de-identified data from patients with narcolepsy,” said Jennifer Gudeman, Pharm.D., Senior Vice President, Medical and Clinical Affairs of Avadel. “Despite sodium oxybate being FDA approved for more than 20 years, these findings reflect the underutilization of sodium oxybate therapy, some of which may be due to the necessity of first-generation oxybates requiring chronic, middle-of-the-night dosing. These data provide further reassurance into the lack of a signal for sodium oxybate utilization and cardiovascular risk.”
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 patients 7 years of age and older with narcolepsy. For more information, please visit www.avadel.com.
About nference
nference is unlocking the potential of healthcare data with AI, delivering real-world evidence, insights, and solutions informed by the most comprehensive multimodal data across all therapeutic areas. Our extensive de-identified, longitudinal data includes decades of clinical notes along with rich patient histories, vitals, lab tests, radiology images, digital pathology, genomics, and electrophysiology waveforms. Our federated data platform, comprised of premier healthcare organizations, including Banner Health, Duke Health, Emory Healthcare, Mayo Clinic, and Vanderbilt University Medical Center, enables advanced research and custom AI model development. With unprecedented access to data from over 40 million patient journeys, our partners can accelerate target discovery, optimize clinical trial design, and enhance lifecycle management. Follow nference on LinkedIn. Visit us at www.nference.com.
Avadel
Investor Contact:
Austin Murtagh
Precision AQ
Austin.Murtagh@precisionaq.com
(212) 698-8696
Media Contact:
Lesley Stanley
Real Chemistry
lestanley@realchemistry.com
(609) 273-3162
nference
Media Contact:
Matt Hurchik
info@nference.net
FAQ
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