FDA Approves Expanded Indication for Lumryz in Pediatric Patients with Narcolepsy

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The FDA has approved an expanded indication for Avadel Pharmaceuticals’ Lumryz (sodium oxybate extended-release oral suspension) for treating cataplexy and excessive daytime sleepiness in pediatric patients over seven years of age with narcolepsy. According to the company, this approval marks a significant milestone in treating narcolepsy, as Lumryz is a once-nightly treatment that eliminates the need for multiple doses throughout the night. Lumryz was first approved for treating narcolepsy in adults based on results from the Phase III REST-ON trial.¹

“This approval represents an important milestone for the narcolepsy community, specifically for younger narcolepsy patients and their caregivers who face significant challenges associated with waking up in the middle of the night to complete treatment regimens. With this label expansion, pediatric patients seven years and older living with narcolepsy now have the same option that adult patients with narcolepsy have—to choose a once-nightly treatment option that does not disrupt sleep for a middle of the night dose,” said Greg Divis, CEO, Avadel Pharmaceuticals, in a press release. “In less than two years, Avadel has made great strides in establishing our commitment to the development of transformative medicines for sleep disorders. This includes successfully launching Lumryz for the adult narcolepsy population, initiating a Phase III pivotal trial evaluating Lumryz for the treatment of idiopathic hypersomnia, and now the expansion of Lumryz into the pediatric narcolepsy population.”

REST-ON was a randomized, double-blind, placebo-controlled trial that enrolled 212 adult patients with narcolepsy. Consisting of 212 patients, results of the trial found that a 9 g dose of once-nightly Lumryz demonstrated a highly significant and clinically meaningful improvement compared to placebo. The three primary endpoints of the study were efficacy at doses of 9 g, 7.5 g, and 6 g.¹

Results found that all three doses demonstrated clinically meaningful improvements on the Maintenance of Wakefulness Test (MWT), Clinical Global Impression-Improvement (CGI-I), and mean weekly cataplexy attacks. Specifically, patients treated with a 9 g dose of Lumryz demonstrated a 10.82-point change from baseline at week 13 in MWT score compared to a 4.89 score for placebo. Additionally, Lumryz demonstrated a CGI-I of 72% and a reduction of 11.51 points in mean weekly cataplexy attacks.

Avadel stated that Lumryz was well tolerated, with common adverse events (AEs) occurring at low frequencies, which included decreased appetite, dizziness, somnolence, tremor, and enuresis. A total of 3.9% of patients discontinued treatment due to AEs.²

According to the National Organization for Rare Disorders, the incidence of narcolepsy is approximately one in 2,000, with a majority of researchers believing that the disorder remains undiagnosed or misdiagnosed in a significant number of people.³

"I have been prescribing sodium oxybate for children and adolescents with narcolepsy for years as I have seen how effective this medication is and can safely be used," said Anne Marie Morse, DO, board-certified and fellowship-trained pediatric neurologist, sleep medicine specialist, Geisinger Health System, in the press release. “And before this, although numerous families have also witnessed the transformation in their children's lives, I have also had many families turn down the medication, or discontinue after starting, because of the challenge experienced or feared to experience with a forced awakening causing a purposeful nightly disruption, many times met with an exhausting fight, to take the second dose of first-generation oxybates. The expanded FDA approval for Lumryz allows me to now share with my patients and their families that there is an FDA-approved treatment that offers a single bedtime dose of medication, provided in a pre-filled packet. I can now offer more options to more patients which allows me to continue my role as a partner in my patients' journeys."

References

1. 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. Avadel. October 17, 2024. Accessed October 18, 2024. https://investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-announces-fda-approval-lumryztm-sodium

2. Avadel Pharmaceuticals Announces Positive Topline Results from its Pivotal Phase 3 REST-ON Trial of Once-Nightly FT218 for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Patients with Narcolepsy. Avadel. April 27, 2020. Accessed October 18, 2024. https://investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-announces-positive-topline-results-its

3. Narcolepsy. NORD. Accessed October 18, 2024. https://rarediseases.org/rare-diseases/narcolepsy/