FDA Grants Orphan Drug Status to Avadel's LUMRYZ for Idiopathic Hypersomnia

Avadel Pharmaceuticals plc, a biopharmaceutical company dedicated to revolutionizing therapy for individuals with rare sleep disorders, has announced a significant milestone. Their drug, LUMRYZ, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Idiopathic Hypersomnia (IH). This designation is awarded to drugs that show potential for treating rare diseases affecting fewer than 200,000 individuals in the U.S.

LUMRYZ is being highlighted for its potential superiority over existing treatments for IH, primarily due to its convenient once-nightly dosing regimen. This feature is particularly beneficial for patients with IH, a chronic condition characterized by excessive daytime sleepiness. Unlike narcolepsy, IH patients experience severe sleep inertia, making waking up, especially in the middle of the night, a major challenge.

Greg Divis, Avadel's CEO, expressed his satisfaction with the FDA's recognition of LUMRYZ, emphasizing the drug's potential to enhance patient care. He noted the persistent demand from healthcare professionals and patients for an extended-release oxybate option, which LUMRYZ aims to fulfill. The drug's development is progressing through the pivotal Phase 3 REVITALYZ trial, which is designed to assess its efficacy and safety. This trial is a double-blind, placebo-controlled, randomized withdrawal study that involves around 150 adult participants diagnosed with IH. Enrollment is open to those currently using immediate-release oxybates and those who are not. The study is expected to conclude by the end of 2025.

The ODD provides multiple advantages for LUMRYZ's development and potential commercialization. These benefits include a seven-year market exclusivity period in the U.S. after approval, exemption from FDA user fees, and FDA assistance in designing clinical trials.

LUMRYZ is already approved by the FDA for the treatment of cataplexy and excessive daytime sleepiness (EDS) in adults and pediatric patients aged seven and older with narcolepsy. This approval came after the successful results of the REST-ON trial, a phase 3 study that showed significant improvements in EDS, overall patient functioning, and cataplexy attacks compared to a placebo. LUMRYZ stands out as it offers a once-nightly dosing schedule, which is a considerable improvement over existing twice-nightly oxybate products, allowing patients to avoid waking up during the night for a second dose.

The FDA’s approval in 2023 and 2024 for LUMRYZ included granting it seven years of Orphan Drug Exclusivity due to its clinical superiority over other oxybate treatments. This superiority is attributed to its unique dosing regimen that greatly enhances patient compliance and quality of life by eliminating the need for nocturnal arousal.

LUMRYZ remains focused on treating narcolepsy-related symptoms in individuals aged seven and older, with no sales for indications beyond narcolepsy. The drug's active ingredient, sodium oxybate, is a controlled substance, and its use is tightly regulated due to potential risks, including abuse, misuse, and serious side effects when combined with CNS depressants.

Overall, Avadel Pharmaceuticals continues to advance its mission of transforming the lives of patients with rare sleep disorders by pushing forward with the development and potential approval of LUMRYZ for IH. With the ongoing REVITALYZ trial and the support of the FDA’s ODD, the company is well-positioned to make significant strides in addressing the unmet needs of this unique patient population.