Positive topline data from the phase 3 REST-ON trial evaluating FT218 (Avadel), an investigational, once-nightly formulation of sodium oxybate, showed that the treatment led to clinically meaningful improvement in patients with narcolepsy.
The multicenter, double-blind, placebo-controlled trial evaluated the efficacy and safety of FT218 in 212 patients with narcolepsy. Patients were randomized to receive FT218 4.5g, 6.0g, 7.5g, 9.0g, or placebo once nightly for 13 weeks. The primary end points of the study included improvements on the maintenance of wakefulness test (MWT), clinical global impression of improvement (CGI-I) in daytime sleepiness, and mean weekly cataplexy attacks recorded on the Sleep and Symptom Daily Diary.
Results showed that patients treated with FT218 9g achieved highly significant and clinically meaningful improvement across all 3 end points compared with placebo at week 13:
- MWT (minutes): 10.82 vs 4.69, respectively; LS Mean 6.13; P <.001.
- CGI-I (% of patients much/very much improved): 72% vs 31.6%, respectively; odds ratio 5.56; P <.001.
- Mean weekly cataplexy attacks: -11.51 vs -4.86; LS Mean -6.65; P <.001.
Additionally, the 6g and 7.5g doses of FT218 demonstrated highly statistically significant, clinically meaningful improvements for all 3 end points compared with placebo (P <.001).
With regard to safety, the 9g dose of FT218 was generally well tolerated with the most common adverse reactions being nausea (1.3%), vomiting (5.2%), decreased appetite (2.6%), dizziness (5.2%), somnolence (3.9%), tremor (1.3%), and enuresis (9%).
According to Avadel, full safety data and additional secondary end point analysis will be presented at future scientific meetings once it becomes available.
“We are excited to see these positive topline data from the REST-ON study, where all 3 dose levels of once-nightly FT218 demonstrated a statistically significant and clinically meaningful improvement on the measures of the 2 prominent symptoms of narcolepsy, as well as an improvement in overall functioning compared to placebo,” said Jordan Dubow, MD, Chief Medical Officer of Avadel. “Once-nightly FT218 delivered a clinically meaningful response within 3 weeks of treatment initiation, which was sustained through each treatment period.”
For more information visit avadel.com.