FDA Approves Wakix for Cataplexy in Pediatric Narcolepsy
The FDA has expanded approval of Wakix (pitolisant) to treat cataplexy in pediatric patients aged 6 years and older with narcolepsy, making it the first non-controlled substance approved for both pediatric and adult narcolepsy patients with or without cataplexy.
The Food and Drug Administration (FDA) has expanded the approval of Wakix (pitolisant) to include treatment of cataplexy in pediatric patients aged 6 years and older with narcolepsy. Wakix is an antagonist/inverse agonist of the histamine-3 receptor.
With this approval, it is now indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in patients aged 6 years and older with narcolepsy.
The expanded use for cataplexy in pediatric narcolepsy patients was supported by data from the double-blind, placebo-controlled phase 3 study that led to the approval of Wakix for EDS in these patients (ClinicalTrials.gov Identifier: NCT02611687).
Results showed that among the 95 patients with a history of cataplexy, treatment with pitolisant significantly reduced the average number of cataplexy attacks per week compared with placebo (weekly rate of cataplexy [WRC]: 2.2 vs 5.6; WRC ratio, 0.39 [95% CI, 0.17-0.90]). In pediatric patients, the most common adverse reactions reported were headache and insomnia.
Wakix is now the first-and-only FDA-approved treatment option for both pediatric and adult narcolepsy patients with or without cataplexy that is not scheduled as a controlled substance, an important distinction that supports its clinical utility.