FDA Expands Approval of Avadel's LUMRYZ for the Treatment of Pediatric Patients With Narcolepsy

Avadel Pharmaceuticals announced today that the US Food and Drug Administration (FDA) has approved its supplemental new drug application (sNDA) for LUMRYZ for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy.

Narcolepsy, which affects the brain’s ability to regulate the sleep-wake cycle, impacts approximately 1 in 2,000 people in the United States, where roughly 5% of patients are under the age of 18. Common symptoms of narcolepsy, along with EDS and cataplexy, include disrupted nighttime sleep, sleep paralysis and hallucinations when falling asleep or waking up.

LUMRYZ is a once-at-bedtime formulation extended-release sodium oxybate. The treatment was first approved by the FDA on May 1, 2023, for the treatment of cataplexy or EDS in adult patients with narcolepsy. LUMRYZ is now the only FDA-approved treatment of cataplexy or EDS in the pediatric narcolepsy population 7 years and older. The approval, which took place on October 16, sees LUMRYZ granted Orphan Drug Exclusivity through October 16, 2031.

“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,” said Greg Divis, Chief Executive Officer of Avadel Pharmaceuticals via 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 3 pivotal trial evaluating LUMRYZ for the treatment of idiopathic hypersomnia, and now the expansion of LUMRYZ into the pediatric narcolepsy population.”

“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. “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.”