Summary: The FDA granted Orphan Drug Designation to Avadel Pharmaceuticals’ once-at-bedtime LUMRYZ for idiopathic hypersomnia, recognizing its potential clinical superiority over existing oxybates due to improved patient care. 

Key Takeaways:

  • LUMRYZ is already FDA-approved for cataplexy or excessive daytime sleepiness in both adults and children with narcolepsy.
  • The once-nightly formulation is seen as clinically superior to twice-nightly oxybate treatments by eliminating the need for nocturnal awakenings.
  • The REVITALYZ phase 3 trial is underway to evaluate LUMRYZ’s efficacy and safety in adults with IH, with enrollment expected to complete by the end of 2025.
  • Orphan Drug Designation offers benefits like market exclusivity, FDA trial design assistance, and waived user fees, potentially accelerating development and commercialization.

The US Food & Drug Administration (FDA) has granted Avadel Pharmaceuticals plc’s LUMRYZ Orphan Drug Designation for the treatment of idiopathic hypersomnia (IH). The Orphan Drug Designation was granted based on the plausible hypothesis that LUMRYZ may be clinically superior to the same drug(s) already approved for the same indication because LUMRYZ may provide a major contribution to patient care due to its once-nightly dosing for patients with IH.

“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,” says Greg Divis, CEO of Avadel Pharmaceuticals, in a release. “Receipt of Orphan Drug Designation highlights the unique value of our next-generation, extended-release oxybate and its potential for IH patients, if approved. We continually hear 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.”

LUMRYZ is currently being evaluated for the treatment of IH in the REVITALYZ trial, which is a double-blind, placebo-controlled, randomized withdrawal, multicenter phase 3 study designed to evaluate the efficacy and safety of LUMRYZ when given as a once-at-bedtime dose. The study will enroll approximately 150 adults who are diagnosed with IH. REVITALYZ enrollment is open for both participants switching from immediate-release oxybates as well as those not currently taking oxybates, and is on track to be completed by year end 2025.

Orphan Drug Designation is granted by the FDA to support drug development for rare diseases and is assigned to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States. The designation can provide several benefits for the development and commercialization of indicated compounds and medicine,s which include eligibility for a seven-year period of market exclusivity in the U.S. following product approval, FDA assistance in clinical trial design, and an exemption from FDA user fees.

LUMRYZ is an extended-release sodium oxybate medication approved by the FDA on May 1, 2023, as a once-at-bedtime treatment for cataplexy or excessive daytime sleepiness in adults with narcolepsy. On October 16, 2024, LUMRYZ was additionally approved as a once-at-bedtime treatment for cataplexy or EDS in pediatric patients 7 years of age and older with narcolepsy.

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With its approvals in May 2023 and October 2024, the FDA also granted 7 years of Orphan Drug Exclusivity to LUMRYZ for the treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy and in pediatric patients 7 years of age and older with narcolepsy (respectively) due to a finding of clinical superiority of LUMRYZ relative to currently available oxybate treatments. In particular, the FDA found that LUMRYZ makes a major contribution to patient care over currently available, twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose.

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