Avadel and nference Announce Publication of Real-World Data on Sodium Oxybate Treatment Patterns in the Journal of Clinical Neuroscience

Rhea-AI Summary

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.

Positive

• Real-world data validates safety profile with no increased cardiovascular risks
• Large-scale study with 4,387 narcolepsy patients provides robust evidence base

Negative

• 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

-- 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 versus 33.0% of the matched cohort; as IH and narcolepsy are mutually exclusive disorders, these results reflect the frequent diagnostic change with repeat testing
• The rate of hypertension in the sodium oxybate treated group was 21.1% compared to the matched cohort group of 21.4% (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 43%), reflecting the frequent disruptive nighttime sleep inherent in narcolepsy,” said Melissa Lipford, M.D., a neurologist at the Center for Sleep Medicine at Mayo Clinic and lead author of the published paper. “The high rate of idiopathic hypersomnia (IH) diagnosis underscores the challenges with distinguishing between narcolepsy and IH based upon current testing. These findings also revealed insights into challenges with middle-of-the-night sodium oxybate dosing and lessened efficacy when the second dose was omitted.”

“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

What are the key findings of AVDL's sodium oxybate treatment study in 2025?

The study found no significant differences in comorbidity rates between treated and untreated groups, with about one-third of patients discontinuing treatment due to lack of efficacy and difficulty waking for the second dose.

How many narcolepsy patients were analyzed in AVDL's Mayo Clinic study?

The study analyzed 4,387 narcolepsy patients, with 351 receiving immediate-release sodium oxybate treatment and 351 matched controls without treatment.

What is the discontinuation rate for sodium oxybate treatment according to AVDL's research?

Approximately one-third of patients discontinued sodium oxybate treatment, with lack of efficacy being the most common reason.

What are the cardiovascular risks associated with sodium oxybate treatment based on AVDL's study?

The study showed no increased cardiovascular risks, with similar rates of hypertension (21.1% vs 21.4%) and coronary artery disease (7.1%) in both treated and untreated groups.