Summary: The FDA has approved Avadel Pharmaceuticals’ LUMRYZ for pediatric patients aged 7 and older with narcolepsy, offering a once-nightly treatment for cataplexy and excessive daytime sleepiness.

Key Takeaways: 

  • FDA Approval for Pediatric Use: LUMRYZ, previously approved for adults, is now FDA-approved for children aged 7 and older with narcolepsy, offering a once-nightly treatment for cataplexy and excessive daytime sleepiness.
  • Orphan Drug Exclusivity: LUMRYZ has been granted Orphan Drug Exclusivity through 2031, providing market protection for its use in treating pediatric narcolepsy.
  • Positive Impact on Families: Neurologist-sleep specialist Anne Marie Morse, DO, highlights the expanded approval as a significant advancement, allowing for greater flexibility and convenience in treatment options for children with narcolepsy.

The US Food and Drug Administration (FDA) has approved Avadel Pharmaceuticals plc’s supplemental new drug application for LUMRYZ for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy.

“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 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,” says Greg Divis, CEO of Avadel Pharmaceuticals, in a 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 3 pivotal trial evaluating LUMRYZ for the treatment of idiopathic hypersomnia, and now the expansion of LUMRYZ into the pediatric narcolepsy population.”

Anne Marie Morse, DO, a board-certified and fellowship-trained pediatric neurologist and sleep medicine specialist at Geisinger Health System., says in a release, “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. 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.”

LUMRYZ is a once-at-bedtime formulation extended-release sodium oxybate and was first approved by the FDA on May 1, 2023, for the treatment of cataplexy or excessive daytime sleepiness in adult patients with narcolepsy.

LUMRYZ was approved by the FDA for use in the treatment of cataplexy or excessive daytime sleepiness in the pediatric narcolepsy population 7 years and older on October 16, 2024, and was granted Orphan Drug Exclusivity through October 16, 2031.

LUMRYZ has a boxed warning as a central nervous system depressant, and for its potential for abuse and misuse. LUMRYZ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the LUMRYZ REMS. Most common adverse reactions (incidence > 5% and greater than placebo) reported for all doses of LUMRYZ combined in a trial of adults with narcolepsy were nausea, dizziness, enuresis, headache, and vomiting. Similarly, in a trial of pediatric narcolepsy patients receiving immediate-release sodium oxybate, the most commonly observed adverse reactions (incidence ≥5%) were nausea, enuresis, vomiting, headache, decreased weight, decreased appetite, dizziness, and sleepwalking.