The US Food and Drug Administration (FDA) has granted final approval to Avadel Pharmaceuticals’ Lumryz, an extended-release formulation of sodium oxybate indicated to be taken once at bedtime for the treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy.
“Today’s landmark approval and receipt of Orphan Drug Exclusivity represents a major milestone for both Avadel and people living with narcolepsy. As we have heard from key stakeholders, previously approved narcolepsy therapies have the potential to disrupt sleep by either causing insomnia or through forced awakening during the middle of the night for their crucial second dose,” says Greg Divis, CEO of Avadel, in a release. “Lumryz can now offer people with narcolepsy the opportunity for an uninterrupted night sleep while receiving the full benefit of their prescribed treatment in one single bedtime dose that addresses their symptoms of narcolepsy. We would like to thank the patients, caregivers, clinical trial investigators, healthcare providers, and advocates who have tirelessly partnered with us throughout the drug development process and look forward to providing the narcolepsy community access to now approved Lumryz.”
The FDA’s final approval of Lumryz was based on positive results from the pivotal phase 3 REST-ON clinical study completed in March 2020. In the trial, once-at-bedtime Lumryz demonstrated highly statistically significant and clinically meaningful improvement compared to placebo across all three co-primary endpoints (maintenance of wakefulness test, clinical global impression-improvement, and mean weekly cataplexy attacks) for all three doses evaluated, 6, 7.5, and 9 grams.
“This long-awaited therapy for people living with narcolepsy fills a critical unmet need by avoiding the burden of a second middle-of-the-night dose that immediate-release oxybate products require. The once-at-bedtime dosing regimen of Lumryz may help restore a more natural sleep-wake cycle,” says Michael J. Thorpy, MD, an investigator from the REST-ON phase 3 trial, director at the Sleep-Wake Disorders Center at Montefiore Medical Center, and professor of neurology at the Albert Einstein College of Medicine, in a release.
With this approval, the FDA has also found Lumryz to be clinically superior to currently marketed twice-nightly oxybate products and granted Lumryz seven years of Orphan Drug Exclusivity, according to a release from Avadel. In particular, FDA found that Lumryz makes a major contribution to patient care over currently available twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose.
The FDA’s Orphan Drug program is designed to support the development of drugs that treat a condition affecting fewer than 200,000 US patients. The seven-year market exclusivity for Lumryz began on the date of FDA approval, May 1, 2023.
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. The most common adverse reactions reported for all doses of Lumryz combined were nausea, dizziness, enuresis, headache, and vomiting.