Avadel Pharmaceuticals has submitted a supplemental new drug application to the US Food and Drug Administration (FDA) for Lumryz for the treatment of cataplexy or excessive daytime sleepiness in the pediatric narcolepsy population. 

According to a release from the company, Lumryz has the potential to significantly alleviate the burden placed on families and caregivers of children with narcolepsy who are responsible for waking up in the middle of the night to administer a second dose. Pediatric patients currently represent approximately 5% of all oxybate-treated narcolepsy patients.

An approval decision is expected in the second half of 2024.

Additionally, the company reported in its third-quarter results that Lumryz will move to a preferred position on CVS commercial formularies starting Jan 1, 2024, following its US launch this summer and increasing patient enrollment.

“We are very pleased with the significant progress we made during the first full quarter of the launch of Lumryz, underscored by the early patient demand and the overwhelmingly positive feedback received from across the narcolepsy community,” says Greg Divis, CEO of Avadel, in a release.

Since the launch of Lumryz through Sept 30, more than 1,000 patients enrolled in Avadel’s RYZUP patient support services, and more than 400 patients initiated therapy with Lumryz during the quarter. Additionally, approximately 600 patients remained in the RYZUP process, going through the benefits investigation or pending their first shipment.

RYZUP enrollments and patients currently being treated with Lumryz include a majority of switch patients from first-generation oxybates, followed by patients who previously tried and discontinued a first-generation oxybate, and patients who are new to oxybates.

Other highlights from the quarter include: 

  • Secured Lumryz coverage policies for over 100 million commercial lives representing approximately 60% of the total commercially insured lives across the country.
  • Announced that Lumryz is moving to preferred status within the CVS commercial formularies effective Jan 1, 2024.
  • Nearly 1,400 health care providers have completed the Lumryz Risk Evaluation and Mitigation Strategy certification process, including both experienced oxybate prescribers as well as providers who have never previously prescribed an oxybate.

“Our team continues to execute on our launch plan, actively engaging with all stakeholders, driving patient enrollments, converting RYZUP enrollments to patients on therapy, and securing key coverage policy decisions with payers, such as the recent CVS decision to move Lumryz to preferred status effective Jan 1, 2024,” says Divis in the release. 

Clinical updates:

  • In October, Avadel announced new and encore data supporting the clinical profile for Lumryz and patient preference for a once-nightly oxybate in 15 poster presentations and two oral presentations, at World Sleep 2023.
    • New post-hoc analyses demonstrated the robust clinical efficacy of Lumryz and provided further insight into the improvements on measures of excessive daytime sleepiness and cataplexy compared with placebo in different demographic and clinical subgroups.
    • Post-hoc analyses reinforced data from the completed pivotal phase 3 REST-ON trial, demonstrating that treatment with Lumryz resulted in statistically significant and clinically meaningful improvement in excessive daytime sleepiness.
    • RESTORE study poster demonstrated long-term tolerability and clinically significant improvement in symptoms.

 The FDA approval of Lumryz was supported by results from REST-ON, a randomized, double-blind, placebo-controlled, pivotal phase 3 trial in adults with narcolepsy. Lumryz demonstrated statistically significant and clinically meaningful improvements in the three co-primary endpoints: excessive daytime sleepiness, clinicians’ overall assessment of patients’ functioning, and cataplexy attacks, for all three evaluated doses when compared to placebo.

With its approval, the FDA also granted seven years of Orphan Drug Exclusivity to Lumryz for the treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy 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.