Harmony Biosciences announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for WAKIX (pitolisant) tablets for the treatment of excessive daytime sleepiness (EDS) in pediatric patients 6 years of age and older with narcolepsy. The FDA separated the submission into two sNDAs for administrative purposes to issue an approval for the treatment of EDS and a complete response for the treatment of cataplexy in pediatric patients (6 to <18 years of age) with narcolepsy. The FDA granted priority review of the sNDA based on a Phase 3 study conducted by Bioprojet, which evaluated the safety and efficacy of pitolisant in patients ages 6 to under 18 years with narcolepsy, with or without cataplexy.

Based on the results of this study, Bioprojet received approval from the European Medicines Agency last year extending the indication for WAKIX to include the treatment of narcolepsy in children ages 6 years of age and older, with or without cataplexy. WAKIX was first approved by the FDA in August 2019 for the treatment of EDS in adult patients with narcolepsy, followed by FDA approval for the treatment of cataplexy in adult patients with narcolepsy in October 2020. A first-in-class treatment with a novel mechanism of action, WAKIX functions as a selective histamine 3 (H3) receptor antagonist/inverse agonist that is believed to target the histamine system to promote wakefulness.