Avadel Pharmaceuticals plc (Nasdaq: AVDL) announced a favorable ruling from the U.S. District Court for the District of Columbia, which upheld the FDA's approval of LUMRYZ™ (sodium oxybate) for extended-release oral suspension. This decision comes after Jazz Pharmaceuticals Inc. filed a suit under the Administrative Procedure Act, challenging the FDA's approval of LUMRYZ as a clinically superior treatment for cataplexy and excessive daytime sleepiness (EDS) in adults and pediatric patients (7 years and older) with narcolepsy.
The court's decision affirms the FDA's grant of Orphan Drug Exclusivity (ODE) to LUMRYZ, which was initially approved in May 2023. Jazz Pharmaceuticals argued that the approval was not in alignment with the Orphan Drug Act. Avadel CNS Pharmaceuticals, LLC, intervened to defend the FDA's actions, successfully maintaining the approval of LUMRYZ.
Clinical Significance of LUMRYZ
LUMRYZ is the first and only once-at-bedtime oxybate treatment for narcolepsy. The FDA's approval was based on the Phase 3 REST-ON trial, a randomized, double-blind, placebo-controlled study in adults with narcolepsy. The trial demonstrated statistically significant and clinically meaningful improvements in EDS (measured by the Maintenance of Wakefulness Test, MWT), clinician-assessed overall functioning (CGI-I), and cataplexy attacks across all three evaluated doses compared to placebo.
Greg Divis, Chief Executive Officer at Avadel Pharmaceuticals, stated, "We are pleased with the Court’s ruling in favor of the FDA’s clinical superiority determination for LUMRYZ in conjunction with final approval for use in adults with narcolepsy. With the Court’s decision, LUMRYZ will continue to be available to the narcolepsy community and retain its Orphan Drug Exclusivity."
Orphan Drug Exclusivity
The FDA granted LUMRYZ seven years of Orphan Drug Exclusivity for treating cataplexy or EDS in narcolepsy patients. This exclusivity is based on the finding that LUMRYZ provides a major contribution to patient care over existing twice-nightly oxybate products by offering a once-nightly dosing regimen. This reduces the need for nocturnal arousal to take a second dose, minimizing sleep fragmentation and disruption of sleep architecture.
About Narcolepsy
Narcolepsy is a chronic neurological disorder affecting the brain's ability to regulate the sleep-wake cycle. It affects an estimated 1 in 2,000 individuals. Common symptoms include excessive daytime sleepiness, cataplexy (sudden muscle weakness), sleep paralysis, and hypnagogic hallucinations. Current treatments aim to manage symptoms, but the once-at-bedtime administration of LUMRYZ offers a significant advantage in patient compliance and sleep maintenance.
Safety Information
LUMRYZ carries a boxed warning regarding the risk of central nervous system (CNS) depressant effects. Taking LUMRYZ with other CNS depressants, such as alcohol, opioids, or sedatives, can cause serious medical problems, including respiratory depression, hypotension, syncope, and death. The active ingredient, sodium oxybate, is a form of gamma hydroxybutyrate (GHB), a controlled substance with potential for abuse and misuse. LUMRYZ is available only through a certified pharmacy under the LUMRYZ REMS program.
