Wakix Treatment for Excessive Daytime Sleepiness in Pediatric Patients with Narcolepsy Approved

The Food and Drug Administration (FDA) has approved Harmony Biosciences’ supplemental New Drug Application (sNDA) for Wakix (pitolisant; Harmony Biosciences, Plymouth Meeting, PA) for the treatment of excessive daytime sleepiness (EDS) in pediatric patients (aged 6 to <18 years) with narcolepsy. The FDA previously approved Wakix, a selective histamine 3 receptor antagonist/inverse agonist, for the treatment of EDS (approved August 2019) and cataplexy (approved October 2020) in adults with narcolepsy.

The FDA granted priority review of the sNDA for Wakix based on results of a double-blind, randomized, placebo-controlled phase 3 study (NCT02611687) which demonstrated the safety and efficacy of Wakix treatment in pediatric patients with narcolepsy.

"The unique mechanism of action of WAKIX and its non-scheduled status are especially important for a pediatric population that has had limited treatment options, all of which are controlled substances," said Jeffrey M. Dayno, MD, President and Chief Executive Officer of Harmony Biosciences. "EDS is the primary symptom experienced by all patients with narcolepsy and this approval for WAKIX, as the first-and-only FDA-approved non-scheduled treatment option for narcolepsy, makes this important treatment option available to pediatric patients 6 years and older living with narcolepsy."