FDA Approves Sodium Oxybate to Treat Cataplexy or EDS in Pediatric Narcolepsy Patients

Updated on October 17, 2024 at 2:33 PM.

The FDA has approved a supplemental new drug application (sNDA) for sodium oxybate (Lumryz; Avadel Pharmaceuticals) for the treatment of epilepsy or excessive daytime sleepiness (EDS) in patients with narcolepsy aged 7 to the upper one. It is an extended-release oral suspension that is taken once at bedtime, which is important for patients who may adhere to second-nightly regimens that require waking up in the middle of the night to take second degree.¹

About the Test

Test Name: Once-Nightly Sodium Oxybate for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy.

ClinicalTrials.gov ID: NCT02720744

Sponsor: Avadel

Last Date: March 25, 2020

In May 2023, the treatment was approved by the FDA to treat EDS or cataplexy in adults with narcolepsy, making it the first and only once-in-a-lifetime treatment for this indication. at the time it is received. Its initial approval was supported by results from the phase 3 clinical trial REST-ON (NCT02720744).^1,2

Narcolepsy is a chronic neurological condition that interferes with the brain’s ability to regulate sleep and wakefulness. This condition affects around 1 in 2000 people in the US, of which about 5% are under the age of 18. Once the symptom of disability is EDS, and others can include loss sudden muscle spasms that are usually caused by cataplexy (or severe). emotions), disturbed sleep at night, sleep paralysis, and visions when falling asleep or waking up.¹

“This recognition represents an important step for the narcolepsy community, especially for young people [patients with narcolepsy] and their caregivers who face significant challenges associated with waking up in the middle of the night to complete treatments. With this label extension, pediatric patients age 7 and older living with narcolepsy now have the same choice that adult patients with epilepsy have—choosing the treatment option of immediately at night without disrupting sleep for the night dose,” said Greg. Divis, CEO of Avadel Pharmaceuticals, in a press release.¹

The REST-ON clinical trial (NCT02720744) was a phase 3 double-blind trial in which 190 patients with type 1 or type 2 16 years of age and older were assigned to receive sodium oxybate once every night of various doses (4.5 g, 6 g, 7.5 g, or 9 g; n = 97), or placebo (n = 93).³ The primary endpoints of the trial were maintaining alertness, the number of cataplexy attacks, and patients who had a “great” or “very” improvement in global perception of improvement from baseline, all of which were evaluated. at week 14.⁴ Secondary endpoints included polysomnographic measures of sleep stage changes and nocturnal awakenings and patient-reported assessments of sleep quality and restful nature of sleep at 6, 7.5, and 9 g; and changes in the time spent in each stage of sleep, delta power, and analyzes of motivational use subgroups for the specified endpoints.³

Studies have shown that sodium oxybate has been shown to be clinically and statistically significant in reducing the number of transitions from wakefulness/N1 to the N1, N2, and fast-paced phases. (REM), as well as the number of nocturnal awakenings. In addition, the quality of sleep and the relaxing nature of sleep were significantly improved with sodium oxybate compared to placebo. Furthermore, post hoc analyzes showed a significant reduction in time spent in N1 and REM as well as increased time spent in N3 with sodium oxybate compared to placebo.^3,4

“FDA’s expanded approval for [sodium oxybate] allows me to now share with my patients and their families that there is an FDA-approved treatment that provides a single dose of sleep medication, provided in a pre-filled packet. I am now able to offer more options to more patients which allows me to continue my role as a partner in my patients’ journeys,” said Anne Marie Morse, DO, a pediatric neurologist at certified and trained in conjunction with a sleep medicine specialist at Geisinger Health, in a news release.¹

ADDED

1. Avadel. 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 and Older with Narcolepsy. News release. October 17, 2024. Accessed October 17, 2024. https://investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-announces-fda-approval-lumryztm-sodium

2. American Academy of Sleep Medicine. Avadel receives FDA approval for Lumryz. News release. May 2, 2023. Accessed October 17, 2024. https://aasm.org/avadel-receives-fda-approval-for-lumryz/#:~:text=Avadel%20Pharmaceuticals%20announced%20on%20May, cataplexy%20in %20 adults%20and%20narcolepsy

3. Roth T, Dauvilliers Y, Thorpy MJ, et al. Effect of FT218, a Once-Nightly Form of Sodium Oxybate, on Disturbed Nighttime Sleep in Patients with Narcolepsy: Results from the Randomized Phase III REST-ON Trial. CNS drugs. 2022;36(4):377-387. doi:10.1007/s40263-022-00904-6

4. Once-Nightly Sodium Oxybate for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy. ClinicalTrials.gov reference: NCT02720744. Updated March 22, 2022. Accessed October 17, 2024. https://clinicaltrials.gov/study/NCT02720744