FDA grants orphan drug status to Avadel’s IH treatment

Published 05/06/2025, 12:42
FDA grants orphan drug status to Avadel’s IH treatment

DUBLIN - Avadel Pharmaceuticals plc (NASDAQ: AVDL), a company specializing in biopharmaceuticals, has received Orphan Drug Designation (ODD) from the U.S. Food & Drug Administration (FDA) for its drug LUMRYZ™, aimed at treating Idiopathic Hypersomnia (IH), a chronic sleep disorder. The designation, granted today, is based on the potential of LUMRYZ to be clinically superior to existing treatments due to its once-nightly dosing regimen.

IH is a condition characterized by excessive daytime sleepiness not caused by other medical issues. LUMRYZ, which is an extended-release sodium oxybate, is currently under evaluation in a Phase 3 trial named REVITALYZ. This double-blind, placebo-controlled study is designed to assess the drug’s efficacy and safety, with an enrollment target of approximately 150 adults diagnosed with IH. The trial is expected to complete enrollment by the end of 2025.

The FDA’s Orphan Drug Designation is aimed at facilitating drug development for rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. This status provides several development and commercial benefits, including seven years of market exclusivity post-approval, FDA guidance on clinical trial design, and exemption from FDA user fees. The company’s strong financial position is evident in its healthy current ratio of 2.73, indicating solid ability to meet short-term obligations. InvestingPro data reveals 8 additional key insights about Avadel’s financial health and growth prospects.

LUMRYZ was previously approved by the FDA on May 1, 2023, as a once-at-bedtime treatment for cataplexy or excessive daytime sleepiness in adults with narcolepsy, making it the first of its kind for this indication. Following this, on October 16, 2024, it was also approved for use in pediatric narcolepsy patients aged 7 and older. These approvals were based on the REST-ON trial, which showed significant improvements in narcolepsy symptoms compared to placebo.

Greg Divis, CEO of Avadel Pharmaceuticals, stated, "Receipt of ODD highlights the unique value of our next-generation, extended-release oxybate and its potential for IH patients, if approved." He also emphasized the company’s commitment to developing differentiated therapies for patients with rare sleep disorders. The company’s revenue has grown significantly, showing a 252% increase in the last twelve months, and analysts maintain a strong buy consensus with a price target suggesting substantial upside potential. For detailed analysis and comprehensive insights, access the full Pro Research Report available on InvestingPro.

The information about LUMRYZ’s Orphan Drug Designation and its development status is based on a press release statement from Avadel Pharmaceuticals.

In other recent news, Avadel Pharmaceuticals has reported steady demand for its narcolepsy medication, LUMRYZ, with Q1 2025 patient demand metrics showing improvement over the last quarter of 2024. The company is advancing its Phase 3 pivotal trial, REVITALYZ, for Idiopathic Hypersomnia, with full enrollment expected in the latter half of 2025. Avadel has also initiated four patent infringement lawsuits against Jazz Pharmaceuticals, claiming infringement on multiple Avadel patents. Additionally, Avadel received a favorable court ruling allowing it to pursue FDA approval for LUMRYZ in conditions beyond narcolepsy, including Idiopathic Hypersomnia. The company announced the appointment of Susan Rodriguez as Chief Operating Officer to enhance the commercial strategy for LUMRYZ. Avadel also disclosed equity grants to key executives, including options and restricted share awards, to align their interests with those of shareholders. The company highlighted its diversified supply chain for LUMRYZ, ensuring domestic manufacturing capabilities amid recent U.S. tariff policy changes. Lastly, Avadel provided an update at the Needham Virtual Healthcare Conference, although specific details of the update were not disclosed.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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