FDA Approves Avadel's LUMRYZ™ for Narcolepsy Treatment in Patients 7+

Avadel Pharmaceuticals plc, a biopharmaceutical company dedicated to developing transformative medications, has announced the approval by the U.S. Food and Drug Administration (FDA) of its supplemental new drug application (sNDA) for LUMRYZ. This approval extends the use of LUMRYZ to treat cataplexy or excessive daytime sleepiness (EDS) in narcolepsy patients aged 7 years and older. Previously, LUMRYZ had been approved solely for adult patients. This recent FDA authorization is significant for the narcolepsy community, particularly for younger patients who previously faced challenges with middle-of-the-night medication doses.

Greg Divis, CEO of Avadel Pharmaceuticals, highlighted the importance of this approval. He noted that pediatric narcolepsy patients now have the option of a once-nightly treatment, similar to what is available for adults. This advancement simplifies treatment regimens, eliminating the need for nighttime awakenings to take a second dose. Divis also emphasized the company's commitment to developing innovative treatments for sleep disorders, evidenced by the successful launch of LUMRYZ for adults and the initiation of a Phase 3 trial for idiopathic hypersomnia treatment.

Dr. Anne Marie Morse, a pediatric neurologist specializing in sleep medicine, expressed her support for the approval. She has long prescribed sodium oxybate to young narcolepsy patients, recognizing its effectiveness. However, she acknowledged that the necessity for a second nighttime dose had deterred some families from continuing the treatment. The new approval, providing a single dose at bedtime, offers a less disruptive option. Dr. Morse believes this will enable her to offer better treatment choices, ultimately enhancing her patients' quality of life.

Narcolepsy is a chronic neurological disorder affecting the brain's ability to regulate sleep-wake cycles, impacting approximately 1 in 2,000 people in the U.S., with 5% of those affected being under 18. Symptoms include excessive daytime sleepiness, cataplexy, disrupted nighttime sleep, sleep paralysis, and hallucinations during sleep transitions.

LUMRYZ, an extended-release sodium oxybate, was initially approved by the FDA on May 1, 2023, for treating cataplexy or EDS in adults with narcolepsy. This formulation allows for once-at-bedtime dosing, avoiding disruptions caused by a second nightly dose, a significant advancement over first-generation oxybate treatments. With the new approval on October 16, 2024, LUMRYZ is now sanctioned for use in pediatric patients aged 7 and older, with Orphan Drug Exclusivity granted through October 16, 2031.

LUMRYZ carries a boxed warning due to its potential for abuse and central nervous system depression. It is only available through a restricted program known as the LUMRYZ REMS. Common adverse reactions in adults include nausea, dizziness, enuresis, headache, and vomiting, while pediatric patients also report decreased weight, appetite, and sleepwalking.

This approval was supported by results from the REST-ON trial, a Phase 3 study involving adult narcolepsy patients. The trial demonstrated significant improvements in EDS, patient functioning, and frequency of cataplexy attacks with LUMRYZ when compared to a placebo. The FDA granted seven years of Orphan Drug Exclusivity to LUMRYZ for adult patients due to its clinical superiority in providing a once-nightly dosing regimen.

Avadel Pharmaceuticals focuses on developing innovative solutions to improve existing treatments. LUMRYZ stands out as the first and only once-at-bedtime oxybate treatment approved by the FDA for both adults and children with narcolepsy, addressing the challenge of nighttime dosing with a single pre-filled packet. This advancement is a testament to Avadel's dedication to enhancing patient care and quality of life.