Despite having a highly differentiated clinical value proposition compared to branded, twice-nightly oxybates, Avadel is pricing Lumryz at $64.67 per gram or around $177,034 annually, which is on par with twice-nightly oxybate products that require chronic, middle-of-the-night awakening.
FDA granted final approval to Avadel Pharmaceutical’s Lumryz (sodium oxybate), the first and only once-at-bedtime oxybate for people with narcolepsy.
The extended-release formulation treats cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. Additionally, Lumryz was granted Orphan Drug Exclusivity for seven years, according to Avadel.
“Today’s landmark approval and receipt of Orphan Drug Exclusivity represents a major milestone for both Avadel and people living with narcolepsy,” said Greg Divis, CEO of Avadel. “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. 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.”
Avadel originally received Orphan Drug Designation for Lumryz in 2018 and received tentative approval from the FDA in July 2022. Final approval was pending resolution of a regulatory exclusivity, which was resolved in February 2023.
The FDA granted Lumryz seven years of Orphan Drug Exclusivity after it determined the treatment is “clinically superior to currently marketed twice-nightly oxybate products,” according to 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 pharma maker said.
Despite having a highly differentiated clinical value proposition compared to branded, twice-nightly oxybates, Divis told Formulary Watch, Avadel is pricing Lumryz at $64.67 per gram or around $177,034 annually, which is on par with twice-nightly oxybate products that require chronic, middle-of-the-night awakening.
“Our focus with the payers has always been to ensure parity access for Lumryz with other branded oxybates,” Divis said.
Avadel will be rolling out patient support services through RYZUP, which will aim to help patients with narcolepsy with assistance and affordability programs, Divis added.
Narcolepsy, a chronic neurological condition, affects approximately one in 2,000 people in the U.S. with the cardinal symptom of EDS, according to Avadel. Additional symptoms can vary by person but may include disrupted nighttime sleep, a sudden loss of muscle tone usually triggered by strong emotion (cataplexy), sleep paralysis and hallucinations.
“Our current research suggests the target patient population who could benefit from Lumryz could be greater than 30,000, approximately double that of the current twice-nightly oxybate market,” Divis said.
“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,” said Michael J. Thorpy, M.D., an investigator from the REST-ON Phase 3 trial and director at the Sleep-Wake Disorders Center at Montefiore Medical Center and professor of Neurology at the Albert Einstein College of Medicine.
The FDA’s final approval of Lumryz was based on results from the 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.
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 (REMS).
The most common adverse reactions (incidence > 5% and greater than placebo) reported for all doses of Lumryz combined were nausea, dizziness, enuresis, headache, and vomiting.