Lumryz Receives Expanded Indication and Was Shown to Improve Symptoms of Narcolepsy Regardless of Concomitant Use of Alerting Agents

The Food and Drug Administration (FDA) has approved a supplemental new drug application (sNDA) for Lumryz (sodium oxybate; Avadel Pharmaceuticals, Dublin, Ireland), expanding the medication’s indication to the treatment of cataplexy or excessive daytime sleepiness (EDS) in people aged ≥7 years with narcolepsy. Lumryz was previously only approved to treat cataplexy or EDS in adults with narcolepsy.

Additionally, new results published in Sleep Medicine demonstrated clinically significant improvements in wakefulness, overall condition, EDS, and number of weekly cataplexy episodes in people taking Lumryz compared with placebo, regardless of the concomitant use of alerting agents.

The new findings result from a post-hoc analysis of data from the pivotal phase 3 REST-ON clinical study (NCT02720744), which was a randomized, placebo-controlled study evaluating the safety and efficacy of Lumryz treatment for 190 participants aged ≥16 years with narcolepsy type 1 or 2. In the post-hoc analysis population, 119 (63%) participants (Lumryz, n=66; placebo, n=53) concomitantly used alerting agents and 71 (37%) did not (Lumryz, n=31; placebo, n=40). Primary endpoints included change from baseline in sleep latency on the Maintenance of Wakefulness Test (MWT), overall condition according to Clinical Global Impression–Improvement (CGI-I) rating, and number of weekly cataplexy episodes.

• At week 13, participants taking Lumryz showed a significantly improved least squares mean (LSM) change in sleep latency on the MWT vs placebo in those with (10.7 minutes vs 4.7 minutes; P=.001) and without (10.9 minutes vs 4.6 minutes; P=.001) concomitant use of alerting agents.
• At weeks 3, 8, and 13, regardless of use of alerting agents, a greater proportion of participants who received Lumryz vs placebo showed CGI-I ratings of “much improved” or “very much improved”.
• At week 13, participants taking Lumryz showed a significantly reduced LSM change from baseline in the number of cataplexy episodes vs placebo in those with (-11.7 vs -5.2; P<.05) and without (-10.7 vs -5.2; P<.05) concomitant use of alerting agents.
• Mean scores for the key secondary endpoint of Epworth Sleepiness Scale (ESS) were significantly improved for participants receiving Lumryz at all doses vs placebo in those that did not take alerting agents (P<.05).
• For participants who concomitantly used alerting agents, the 7.5 g and 9 g dose levels were associated with significant improvements in ESS score.

“The expanded FDA approval for LUMRYZ allows me to now share with my patients and their families that there is an FDA-approved treatment that offers a single bedtime dose of medication, provided in a pre-filled packet,” said Anne Marie Morse, DO, a pediatric neurologist and sleep medicine specialist at Geisinger Health System. “I can now offer more options to more patients which allows me to continue my role as a partner in my patients' journeys."

Lumryz was also granted Orphan Drug Exclusivity by the FDA through October 16, 2031. Lumryz’s label contains a Boxed Warning for central nervous system (CNS) depression and abuse and misuse.