Jennifer Gudeman, PharmD, Vice President of Medical and Clinical Affairs at Avadel Pharmaceuticals, discusses the post hoc analyses of data from the REST-ON clinical trial of FT218, an investigational treatment for narcolepsy.
Narcolepsy is a rare neurological disorder characterized by excessive sleepiness during the day, often with periods of brief involuntary sleep and/or cataplexy.
As Dr. Gudeman explains, FT218 is a long-acting form of sodium oxybate. A quick-release form of sodium oxybate was FDA approved in 2002 but, due to a short half-life, had to be taken twice a night. With FT218, the long-acting nature allows it to be taken once before bed.
The REST-ON study was a double-blind, randomized, placebo-controlled phase 3 trial assessing the efficacy and safety of FT218 in the treatment of excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy. The trial design allowed 3 doses to be evaluated (6 g, 7.5 g, 9 g) within each patient. The three co-primary endpoints were: maintenance of wakefulness test (MWT), Clinical Global Impression-Improvement (CGI-I), and weekly number of cataplexy attacks.
As Dr. Gudeman notes, FT218 demonstrated statistically significant improvement in excessive daytime sleepiness (EDS) at all evaluated doses in patients with both narcolepsy subtypes 1 (NT1, with cataplexy) and 2 (NT2, without cataplexy), with greater improvement in measures of EDS, including mean sleep latency on maintenance of wakefulness test (MWT) and Clinical Global Impression-Improvement (CGI-I) in overall condition, compared to placebo. Additionally, a significantly greater percentage of patients with NT1 receiving FT218 were rated as much or very much improved on the CGI-I compared to placebo: 75.5% vs. 35.9% at 9 g, 66.9% vs. 27.9% at 7.5 g, and 39.9% vs. 7.8% at 6 g (all P < .001). A greater percentage of NT2 patients receiving FT218 were rated as much/very much improved at all three doses compared to placebo.
The Epworth Sleepiness Scale was a secondary endpoint in the study. In this scale, the highest score achievable is 24, which indicates incredible sleepiness, while anything < 10 is average. At baseline, participants on average scored a 16 on this scale. Following treatment with FT218, the average score was 10.4, which indicates that approximately half of the FT218 arm were experiencing average or better than average sleepiness. This marks a significant improvement in daytime sleepiness with FT218 compared to placebo.
To learn more about narcolepsy and other rare neurological conditions, go to checkrare.com/diseases/neurology