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

Anne Marie Morse, DO, FAASM

(Credit: Society for Womens Health Research)

Pediatric narcolepsy, a chronic sleep-wake disorder, often begins in childhood with symptoms like excessive sleepiness, cataplexy, and REM-related phenomena such as sleep paralysis and hallucinations. Research shows that these symptoms can impair pediatric patients’ functioning, impacting their social interactions, academics, and quality of life, potentially leading to emotional and behavioral issues.¹ Pharmacological treatments, including new medications, can help to reduce cataplexy and daytime sleepiness, while psychosocial interventions—requiring collaboration among the child, family, school, and healthcare providers—can further support their adjustment and well-being.

In May 2023, the FDA initially approved sodium oxybate (Lumryz; Avadel Pharamaceuticals) for cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.² The approval was supported by data from the phase 3 REST-ON trial (NCT02720744) which showed a significantly greater increase in sleep latency with the treatment compared with placebo. More recently, the FDA approved the company's supplemental new drug application of the therapy for the treatment of cataplexy or EDS in pediatric patients with narcolepsy aged 7 years and older.³ With this approval, the medication could reduce the burden on families and caregivers of pediatric patients with narcolepsy who must wake up at night to administer a second dose.

As part of a new iteration of NeuroVoices, sleep disorder expert Anne Marie Morse, DO, FAASM, director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children's Hospital, shared her reaction following the news. She talked about how the introduction of once-nightly sodium oxybate in pediatric narcolepsy changes the treatment approach for this patient population. Morse, also the program director for Child Neurology Residency Program, spoke about the common adverse effects associated with the treatment, and how they can be managed. Moreover, Morse, also serves the host of the Sleeping Around the Podcast × NeurologyLive, explained why it is crucial to consider the individual treatment needs of each pediatric patient with narcolepsy without bias.

NeurologyLive: What impact does the approval of once-nightly sodium oxybate have on your approach to treating pediatric narcolepsy?

Anne Marie Morse, DO, FAASM: My specific area of focus, both clinically and from a research perspective, tends to be central disorders of hypersomnolence, including conditions like narcolepsy and idiopathic hypersomnia. However, don't let my title fool you; I see patients across all ages, from pediatric to adult. So, when I heard this news today, I was incredibly delighted, as it is essential for us to continue improving treatment options for children and adolescents with narcolepsy. This news resonated strongly with me because the approval of an oxybate medication was what initially sparked my interest in narcolepsy during my fellowship, back when the initial studies on twice-nightly sodium oxybate for pediatrics led to its first approval for use in children. Since that time, the treatment landscape has remained limited. Most recently, we saw the approval of an alerting agent, pitolisant, for pediatric excessive daytime sleepiness. But now, having a once-nightly sodium oxybate available allows me to treat more patients more effectively, making today truly monumental as we move closer to treating every patient—not just some.

What makes the availability of once-nightly sodium oxybate particularly meaningful for pediatric narcolepsy treatment?

I find the availability of once-nightly sodium oxybate for pediatric treatment to be especially meaningful. Although this once-nightly formulation is the newest available, we have over 20 years of experience using oxybate medications in various forms to treat this condition. Thus, we also have a strong understanding of its safety profile and common dose-related adverse effects. Most often, adverse effects appear when starting the medication, as the body adapts to it. The most common adverse effects include nausea—sometimes vomiting, though that’s less frequent—and drowsiness, which patients might describe as a “drug effect.” Typically, these adverse effects occur when first starting and can reemerge with dose adjustments, but over time, they usually diminish. In clinical trials, including those on adults, this trend is consistent even under a forced titration model, where dose changes occur without gradual adjustment periods. Despite this, only a minority of patients discontinued the trials due to adverse effects, as many issues, like nausea or headache, can be anticipated and managed with appropriate guidance.

Many people worry about needing to wake up for a second dose, or how they’ll handle taking it, and these are all factors that can be discussed to find the best approach. Introducing a once-nightly oxybate, particularly for pediatric use, may benefit not only the child but also the family, as it’s often a parent who wakes up for the second dose. This once-nightly option can be restorative not only for the child but for the entire family. Though the medication is taken at night, its FDA indication targets daytime symptoms—EDS and cataplexy—which makes it suitable for treating both narcolepsy types 1 and 2. As we see more treatment options emerging, it’s about determining which oxybate or strategy works best for each individual. The significant advantage of today’s approval is that it offers us a third oxybate formulation, which, by eliminating the need for redosing, may provide a solution for patients who previously weren’t successful with or open to other oxybate forms. Ultimately, it’s essential that we evaluate personal experiences and journeys to tailor treatment to each individual.

How can clinicians address the stigma associated with oxybate medications to ensure that all patients receive treatment based on their quality-of-life needs rather than perceived severity?

A key consideration with this medication’s approval is recognizing that oxybates, like most narcolepsy medications, carry a certain level of stigma as “big” or “potentially dangerous” medications. However, all medications in this area are controlled substances, each with unique risk and benefit profiles. The stigma surrounding oxybate family medications often stems from the notion that they should be reserved for only the most severe cases. I urge physicians, advanced practitioners, and clinicians to leave such assumptions aside. Deciding whether someone is “severe enough” is not our role. If a treatment can make a meaningful difference in quality of life, then that is “severe enough.” This is particularly critical when treating children and adolescents; the decisions we make as clinicians in partnership with young patients have lifelong impacts. We must not allow our biases on severity to deprive a child of academic success, social experiences, or the life they want to lead because we’ve deemed them “not sick enough” for effective treatment. This is not an endorsement of any one oxybate or medication, but a call to action to ensure that our own ignorance doesn’t prevent our patients from living their best lives.

Transcript edited for clarity. Click here to view more NeuroVoices.

REFERENCES
1. Chung IH, Chin WC, Huang YS, Wang CH. Pediatric Narcolepsy-A Practical Review. Children (Basel). 2022;9(7):974. Published 2022 Jun 29. doi:10.3390/children9070974
2. 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
3. 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