FDA Approves Sodium Oxybate for Cataplexy, Excessive Daytime Sleepiness in Pediatric Narcolepsy

The US Food and Drug Administration (FDA) approved the supplemental new drug application (sNDA) for sodium oxybate (LUMRYZ) to treat cataplexy and excessive daytime sleepiness in children aged ≥ 7 years with narcolepsy.¹

Announced by Avadel Pharmaceuticals on October 17, 2024, this indication would expand the availability of oxybate to children. This demographic already comprises of 5% of the narcolepsy patient population who receive oxybate.

"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, a board-certified and fellowship-trained pediatric neurologist and sleep medicine specialist at Geisinger Health System, in a statement. “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."

The decision was based on a submission package of double-blind randomized withdrawal study, led by Michel Lecendreux, MD, from the Pediatric Sleep Center at Hospital Robert-Debré in Paris, which evaluated the long-term efficacy and safety of sodium oxybate in children and adolescents aged 7 – 16 years with narcolepsy and cataplexy.²

The FDA already approved sodium oxybate to treat cataplexy in adult patients with narcolepsy last year on May 1, 2023, based on robust data from the pivotal phase 3 REST-ON clinical trial.³ This trial demonstrated sodium oxybate provided statistically significant (P < .001) and clinically meaningful improvement compared to placebo across all 3 primary endpoints: Maintenance of Wakefulness Test, Clinical Global Impression-Improvement, and mean weekly cataplexy attacks. Sodium oxybate was the first FDA approved once-at-bedtime oxybate for people living with narcolepsy.

Not only did the FDA approve sodium oxybate, but the FDA granted the drug 7 years of Orphan Drug Exclusivity for the treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy due to findings of sodium oxybates’ superiority over currently available therapies, such as modafinil, armodafinil, mixed oxybate.

"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," said Greg Divis, Chief Executive Officer of Avadel Pharmaceuticals. :With this label expansion, pediatric patients 7 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."

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. https://investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-announces-fda-approval-lumryztm-sodium. Accessed October 17, 2024.}
2. ^{Lecendreux M, Plazzi G, Dauvilliers Y, et al. Long-term safety and maintenance of efficacy of sodium oxybate in the treatment of narcolepsy with cataplexy in pediatric patients. J Clin Sleep Med. 2022;18(9):2217-2227. doi:10.5664/jcsm.10090}
3. ^{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. Avadel. May 1, 2023. https://investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-announces-final-fda-approval-lumryztm#:~:text=Avadel's%20commercial%20product%2C%20LUMRYZ%2C%20was,EDS)%20in%20adults%20with%20narcolepsy. Accessed September 6, 2024.}