AASM Includes Wakix on Guidelines for Narcolepsy

The American Academy of Sleep Medicine has published an updated clinical practice guideline, which includes Harmony Biosciences’ Wakix (pitolisant) as a recommended treatment option for adults living with narcolepsy.

The new clinical practice guideline was published in the Journal of Clinical Sleep Medicine along with an accompanying article that reviews supporting evidence for the guidelines. The American Academy of Sleep Medicine commissioned a task force of experts in sleep medicine to perform a systematic review of studies for both pharmacological and nonpharmacological interventions.

In June 2021, Harmony Bioscience presented data at the 35th Annual Meeting of the Associated Professional Sleep Societies. In a poster, investigators presented new analysis that evaluated the clinical impact of Wakix in adults with narcolepsy using number needed to treat (NNT) and effect sizes for treatment as measures of treatment response based on data from the HARMONY 1 and HARMONY CTP randomized, controlled clinical trials.

NNT measures the number of patients that need to be treated to achieve a specific outcome for one person and effect size measures the magnitude of drug–placebo difference on outcome measures. NNT values less than 10 are considered to represent a clinically meaningful difference. Thresholds for effect size are 0.2 for a small effect, 0.5 for a medium effect, and 0.8 representing a large effect size, with larger effect sizes signifying greater efficacy.

For excessive daytime sleepiness umber needed to treat was five in one trial and three in the other. The effect size was 0.61 in the first trial and 0.86 in the second.

Jeffrey Dayno, M.D.

Cataplexy is a condition in which strong emotion or laughter causes a person to suffer sudden physical collapse. The number needed to treat in the HARMONY CTP trial was 3 for a 50% reduction in the weekly rate of cataplexy.

"This analysis utilizing NNT and effect size offers a different perspective on the efficacy profile of Wakix compared with traditional clinical trial end points, and is one that is clinically relevant,” Harmony’s chief medical officer, Jeffrey Dayno, M.D., said in a statement.

The FDA approved Wakix in 2019 for the treatment of excessive daytime sleepiness in adult patients with narcolepsy.