FDA OKs supplemental NDA for Lumryz in patients aged 7 years and older with narcolepsy

The FDA has approved a supplemental new drug application for a once-daily, extended-release therapeutic to treat cataplexy or excessive daytime sleepiness in patients with narcolepsy aged 7 years and older, according to the manufacturer.

Lumryz (sodium oxybate, Avadel Pharmaceuticals) was initially approved for adults with narcolepsy in May 2023. In a press release, the company said the approval was based on results from the phase 3, randomized, double-blind, placebo-controlled REST-ON study.

According to data cited in the release, treatment with Lumryz led to significant improvements in a trio of primary endpoints: excessive daytime sleepiness (EDS) as measured by the Maintenance of Wakefulness Test, clinician-based assessment of overall function as determined by the Clinical Global Impressions – Improvement scale, as well as the number of cataplexy attacks for all three doses compared with placebo.

“Pediatric narcolepsy patients — and their caregivers — can finally access a once-at-bedtime medication with Lumryz, avoiding the chronic, disruptive middle-of-the-night dose required by other twice-nightly oxybates, which is known to be a burden on patients and their families,” Avadel CEO Greg Divis told Healio in an email.

A chronic condition that impedes regulation of the brain’s sleep-wake cycle, narcolepsy affects about one in 2,000 Americans, and approximately 5% of those are aged younger than 18 years, according to the release. Among the most debilitating symptoms of the condition are EDS as well as cataplexy, defined as a sudden loss of muscle tone usually triggered by heightened emotions.

The prescribing information for Lumryz includes a boxed warning as a central nervous system depressant, as well as for the possibility of abuse and misuse. The treatment is only available through a Risk Evaluation and Mitigation Strategy program. The most common adverse events associated with Lumryz in the study of adults included dizziness, enuresis, headache, nausea and vomiting. In a study of pediatric patients, the most common adverse events were decreased appetite or weight, dizziness, enuresis, headache, nausea, sleepwalking and vomiting.

In addition to the new indication of Lumryz in pediatric patients, the FDA also granted it orphan drug exclusivity through Oct. 16, 2031.