FDA
Wakix now approved for cataplexy in pediatric patients with narcolepsy
On February 17, 2026, FDA approved its supplemental new drug application (sNDA) for Wakix (pitolisant) for the treatment of cataplexy in pediatric patients ≥6 years of age with narcolepsy.
Efficacy
The efficacy of Wakix for the treatment of excessive daytime sleepiness in pediatric patients ≥6 years of age with narcolepsy was evaluated in a multicenter, randomized, double-blind, placebo-controlled study (Study 4; NCT02611687). Pediatric patients 6 to 17 years who met the International Classification of Sleep Disorders (ICSD-3) criteria for narcolepsy (with or without cataplexy) and who had a Pediatric Daytime Sleepiness Scale (PDSS) score ≥15 were eligible to enroll in the study. EDS was assessed with the PDSS, an 8-item questionnaire in which patients report their frequency of EDS-related symptoms. Each of the 8 items on the PDSS is rated from 0 (never) to 4 (very often, always); the maximum score is 32, with higher scores representing greater severity of symptoms. The study included an 8-week treatment period: a 4-week dose titration phase followed by a 4-week stable dose phase.
In Study 4, 110 pediatric patients 6 to 17 years were randomized to receive Wakix or placebo. The dose of Wakix was initiated at 4.45 mg once daily and could be increased at weekly intervals to 17.8 mg for patients weighing ≥40 kg, based on clinical response and tolerability. Median age in the study was 13 years, 56% of the patients were male, and 86% of the population had a history of cataplexy. Race and ethnicity weren't collected in the study.
Wakix demonstrated statistically significantly greater improvement on the least squares mean change of -3.41 points (95% confidence interval, -5.52, -1.31) from baseline to the end of treatment in final PDSS total score compared with placebo. Study 4 included global assessments, which showed positive trends supporting PDSS total score of improvement.
Safety
The common adverse reactions (≥5% and greater than placebo) in pediatric patients were headache and insomnia.
Sources:
Wakix (pitolisant) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/211150s006lbl.pdf Revised February 2026. Accessed February 18, 2026.
Harmony Biosciences receives US Food and Drug Administration approval for Wakix® (pitolisant) for the treatment of cataplexy in pediatric narcolepsy. [News release]. 2025. https://ir.harmonybiosciences.com/news-releases/news-release-details/harmony-biosciences-receives-us-food-and-drug-administration-1