Understanding Treatment Benefit of Once-Nightly Sodium Oxybate in Narcolepsy Types 1 and 2: Jennifer Gudeman, PharmD

WATCH TIME: 5 minutes

"Ensuring that clinicians are having conversations with patients with narcolepsy routinely, and asking about the more subtle presentation of cataplexy, is important. Many patients have their diagnosis changed from NT2 to NT1."

Earlier this year, the FDA approved Avadel Pharmaceuticals oral, extended-release formulation of sodium oxybate for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. With the approval, it became the first and only marketed once-nightly oxybate for patients with the condition. Like other oxybates, its label came with a boxed warning as a central nervous depressant, and for its potential abuse and misuse.

The therapy was approved based on data from the phase 3 REST-ON trial (NCT02720744), a pivotal study featuring 222 patients with narcolepsy type 1 or 2, all aged 16 years or older. All told, the study met all 3 of its primary end points of change from baseline in mean sleep latency on the Maintenance of Wakefulness test, Clinical Global Impression Improvement, and weekly cataplexy attacks within the 6-, 7.5-, and 9-g groups.¹

Months following the approval, a group of investigators conducted a post-hoc analysis of REST-ON, specifically focusing on the effects of once-nightly sodium oxybate on narcolepsy subtypes 1 and 2.² At the conclusion of the analysis, senior investigator Jennifer Gudeman, PharmD, and colleagues observed that the therapy maintained its efficacy and safety, regardless of the narcolepsy subtype. Gudeman, senior vice president of medical and clinical affairs for Avadel Pharmaceuticals, sat down with NeurologyLive^® to discuss the reasons behind the evaluation, the clinical significance behind the data, and the differences between narcolepsy type 1 and 2.

REFERENCES
1. Kushida CA, Shapiro CM, Roth T, et al. Once-nightly sodium oxybate (FT218) demonstrated improvements of symptoms in a phase 3 randomized clinical trial in patients with narcolepsy. SLEEP. 2021; zsab200. doi:10.1093/sleep/zsab200
2. Dauvilliers Y, Roth T, Thorpy MJ, et al. Efficacy of once-nightly sodium oxybate (FT218) in narcolepsy type 1 and type 2: post hoc analysis from the phase 3 REST-ON trial. Sleep. 2023;11(46). doi:10.1093/sleep/zsad152