Abstract Details

Twice-nightly SO is an effective treatment for EDS and cataplexy in patients with narcolepsy. FT218 is an investigational once-nightly controlled-release formulation of SO.

To evaluate the efficacy of FT218, a once-nightly sodium oxybate (SO) formulation, on excessive daytime sleepiness (EDS), self-reported sleep quality/refreshing nature of sleep, sleep paralysis (SP), and hypnagogic hallucinations (HH) in patients with narcolepsy.

This was a randomized, double-blind, placebo-controlled, multicenter, parallel-group study in patients with narcolepsy ≥16 years old (randomized, n=212). The 4-period uptitration dosing schedule was as follows: 4.5 g for 1 week, 6 g for 2 weeks, 7.5 g for 5 weeks, and 9 g for 5 weeks (total study duration, 17 weeks). Secondary endpoints included Epworth Sleepiness Scale (ESS), sleep quality/refreshing nature of sleep using a visual analogue scale (VAS), and SP and HH using a sleep symptom diary (SSD).

FT218 demonstrated significant (P<0.001) improvement in ESS vs placebo at all doses tested: LS mean difference –3.86 at 9 g (week 13), –3.16 at 7.5 g (week 8), and –2.06 at 6 g (week 3). FT218 was significantly (P<0.001) better than placebo at all doses tested for sleep quality/refreshing nature of sleep on VAS, and for SP on SSD (P=0.037, 0.021, 0.039 at 9, 7.5, 6 g, respectively). HH was similar for FT218 and placebo. The most common adverse reactions with FT218 treatment were nausea, dizziness, enuresis, headache, decreased appetite, and vomiting.

FT218 demonstrated significant and clinically meaningful results vs placebo at all doses tested for ESS, sleep quality/refreshing nature of sleep, and SP, representing improvement on important narcolepsy symptoms. FT218 was generally well tolerated, and the most common adverse reactions were well-known and established SO adverse reactions.