On June 5, 2025, Avadel Pharmaceuticals announced that the FDA granted orphan drug designation to Lumryz, its extended-release sodium oxybate medication, for the treatment of idiopathic hypersomnia (IH). The FDA’s orphan drug designation provides incentives for drugs and biologics for the treatment of rare diseases.
About Lumryz
Lumryz is a once-at-bedtime sodium oxybate medication that offers an alternative to twice-nightly dosing. Its reduced dosing frequency eliminates the need for patients to wake during the night.
The FDA approved Lumryz in 2023 for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. In 2024, the approval was expanded to include pediatric patients ages 7 and older with narcolepsy.
Lumryz is currently being studied for the treatment of IH in the REVITALYZ trial, a double-blind, placebo-controlled, randomized withdrawal, multicenter Phase 3 study evaluating the efficacy and safety of once-at-bedtime dosing. The trial is expected to conclude by the end of 2025.
According to the company’s press release, Lumryz received orphan drug designation based on the hypothesis that it may offer a major contribution to patient care through its once-nightly dosing, potentially making it clinically superior to existing treatments for IH.
Currently, Lumryz is approved only for the treatment of cataplexy or EDS in patients ages 7 and older with narcolepsy.
About idiopathic hypersomnia
IH is a rare sleep disorder characterized by excessive daytime sleepiness despite normal nighttime sleep duration and quality. Individuals with IH may experience difficulty waking up, exhibit confusion or a “sleep drunkenness” state upon awakening, and may find their prolonged daytime naps to be unrefreshing, posing potential risks in activities requiring alertness, such as driving or operating machinery.
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