The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for FT218, a once-a-night investigational formulation of sodium oxybate for the treatment of excessive daytime sleepiness and cataplexy in adults with narcolepsy.
The NDA filing is supported by data from the Phase 3 REST-ON study (ClinicalTrials.gov: NCT02720744), which evaluated the efficacy and safety of FT218 in 212 patients with narcolepsy. Patients were randomly given FT218 4.5 g, 6.0 g, 7.5 g, 9.0 g, or placebo once per night for 13 weeks. The primary endpoints of the study included improvements in maintenance of the wakefulness test (MWT), overall clinical impression of improvement (CGI-I) in daytime sleepiness, and mean weekly cataplexy attacks recorded in the daily sleep and symptoms diary.
The results showed that patients treated with FT218 9g achieved a highly significant and clinically meaningful improvement across all 3 endpoints compared to placebo at week 13:
- MWT (minutes: minutes): 10.82 versus 4.69; LS mean 6.13; P <0.001.
- CGI-I (% of patients greatly / very much improved): 72% versus 31.6%; Odds ratio 5.56; P <0.001.
- Mean weekly cataplexy attacks: -11.51 vs -4.86; LS mean -6.65; P <0.001.
In addition, the 6 g and 7.5 g doses of FT218 showed statistically highly significant, clinically meaningful improvements for all 3 endpoints compared to placebo (P <0.001).
The 9 g dose was found to be well tolerated. The most common side effects were nausea (1.3%), vomiting (5.2%), decreased appetite (2.6%), dizziness (5.2%), drowsiness (3.9%), tremors (1.3 %) and enuresis (9%).
A target date of the Prescription Drug User Fee Act (PDUFA) of October 15, 2021 has been set for this application. “If approved, FT218 will be the first and only once-a-night oxybate drug. This is a significant step up from the twice-weekly regimen that has been required for nearly 20 years,” said Greg Divis, CEO of Avadel.
Avadel Pharmaceuticals Announces FDA Approval of FT218’s new drug application in adults with narcolepsy for the treatment of excessive daytime sleepiness and cataplexy. [press release]. Dublin, Ireland: Avadel Pharmaceuticals; March 1, 2021.
This article originally appeared on MPR