Understanding the Approval of Once-Nightly Sodium Oxybate for Pediatric Narcolepsy: Anne Marie Morse, DO, FAASM

WATCH TIME: 7 minutes

"This monumental day moves the needle forward, allowing us to treat every patient more effectively, not just some."

In May 2023, the FDA initially approved sodium oxybate (Lumryz; Avadel Pharamaceuticals) for cataplexy or excessive daytime sleepiness in adults with narcolepsy.¹ The approval was supported by data from the phase 3 REST-ON trial (NCT02720744) which showed a significantly greater increase in sleep latency with sodium oxybate treatment at week 3 for the 6-g dose group (8.1 vs 3.1 min, respectively; least-squares mean difference [LSMD], 4.98; 95% CI, 2.90-7.05; P <.001); at week 8 for the 7.5-g dose group (9.6 vs 3.3 min, respectively; LSMD, 6.21; 95% CI, 3.84-8.58; P <.001); and at week 13 for the 9-g dose group compared with placebo (10.8 vs 4.7 min, respectively; LSMD, 6.13; 95% CI, 3.52-8.75; P <.001).

Less than a week ago, the FDA approved the company's supplemental new drug application (sNDA) for sodium oxybate for the treatment of cataplexy or excessive daytime sleepiness in pediatric patients with narcolepsy aged 7 years and older.2 With this approval, the treatment could reduce the burden on families and caregivers of pediatric patients with narcolepsy who must wake up at night to administer a second dose. Following this news, Anne Marie Morse, DO, FAASM, the director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children's Hospital, sat down with NeurologyLive^® to share her reaction in an interview.

During the conversation, Morse, also the program director for Child Neurology Residency Program, talked about how the approval of once-nightly sodium oxybate impacts the treatment landscape for pediatric narcolepsy. She spoke about the key safety considerations and common adverse effects when initiating oxybate therapy in children. Moreover, Morse, who also serves as the host of the Sleeping Around the Podcast × NeurologyLive, emphasized the importance of ensuring that access to effective treatments for narcolepsy is not hindered by subjective assessments of severity.

REFERENCES
1. Avadel Pharmaceuticals announces final FDA approval of Lumryz (sodium oxybate) for extended-release oral suspension as the first and only once-at-bedtime oxybate for cataplexy or excessive daytime sleepiness in adults with narcolepsy. News release. Avadel Pharmaceuticals. May 1, 2023. Accessed October 18, 2023. https://www.globenewswire.com/news-release/2023/05/01/2658536/0/en/Avadel-Pharmaceuticals-Announces-Final-FDA-Approval-of-LUMRYZ-sodium-oxybate-for-Extended-Release-Oral-Suspension-as-the-First-and-Only-Once-at-Bedtime-Oxybate-for-Cataplexy-or-Exc.html
2. Avadel Pharmaceuticals Announces FDA Approval of LUMRYZ™ (sodium oxybate) Extended-Release Oral Suspension (CIII) for the Treatment of Cataplexy or Excessive Daytime Sleepiness in Patients 7 Years of Age and Older with Narcolepsy. News Release. Avadel Pharmaceuticals. Published October 17, 2024. Accessed October 18, 2024. https://investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-announces-fda-approval-lumryztm-sodium