Avadel Pharmaceuticals Earns FDA Orphan Drug Designation For LUMRYZ™ (Extended-Release Sodium Oxybate) To Treat Idiopathic Hypersomnia

Avadel Pharmaceuticals plc, a biopharmaceutical company focused on developing innovative treatments, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to LUMRYZ™ (sodium oxybate) for the treatment of idiopathic hypersomnia (IH). This rare neurological sleep disorder is marked by excessive daytime sleepiness that cannot be explained by other medical conditions and often requires long-term treatment.

The FDA granted this designation based on the possibility that LUMRYZ may offer significant clinical advantages over currently approved treatments for the same condition. LUMRYZ is unique in that it is a once-at-bedtime extended-release formulation, which may provide a meaningful improvement in patient care and convenience compared to immediate-release alternatives that require multiple nightly doses.

LUMRYZ is currently being studied in a Phase 3 clinical trial known as REVITALYZ™, a double-blind, placebo-controlled, randomized withdrawal study. The trial aims to evaluate the safety and effectiveness of once-nightly LUMRYZ in approximately 150 adult patients diagnosed with idiopathic hypersomnia. Participants include both individuals who are already taking immediate-release oxybate products and those who are not currently on any oxybate therapy. The study is being conducted across multiple centers and is expected to complete enrollment by the end of 2025.

Greg Divis, Chief Executive Officer of Avadel Pharmaceuticals, said in a statement, "We are pleased that LUMRYZ has been granted Orphan Drug Designation for the treatment of IH, and this recognition reinforces our strategy to develop differentiated therapies for patients with rare sleep disorders. Receipt of ODD highlights the unique value of our next-generation, extended-release oxybate and its potential for IH patients, if approved.”

He also added, “We continually hears from clinicians and patients of the substantial need for an extended-release oxybate for this patient population – a population who suffers from profound sleep inertia, making waking up in the middle of the night an even greater challenge than in narcolepsy. With continued progress in our pivotal Phase 3 REVITALYZ trial, an established commercial foundation in narcolepsy, and a relentlessly devoted team, we are well-positioned to advance LUMRYZ in IH with the goal of ultimately transforming the IH treatment landscape, if approved.”

Orphan Drug Designation is intended to encourage the development of treatments for rare diseases, defined in the U.S. as conditions affecting fewer than 200,000 people. The designation offers several incentives to the drug developer, including seven years of marketing exclusivity upon approval, assistance from the FDA in designing clinical trials, and exemption from certain FDA application fees.