Harmony Biosciences Holdings (NASDAQ:HRMY) announced Tuesday that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant) to treat cataplexy in pediatric patients aged six years and older.
The decision marks a significant shift in the pediatric narcolepsy landscape.
WAKIX is now the first and only FDA-approved treatment for both adult and pediatric patients that is not classified as a controlled substance by the DEA.
Unlike traditional stimulants or oxybate-based therapies, WAKIX functions as a selective histamine 3 (H₃) receptor antagonist/inverse agonist, increasing the natural release of histamine—a key wake-promoting neurotransmitter—in the brain.
The approval of the cataplexy indication completes the pediatric profile for WAKIX, which received FDA clearance for excessive daytime sleepiness (EDS) in the same age group in June 2024.
Clinicians can now utilize a single, once-daily oral medication to manage the dual hallmark symptoms of Type 1 narcolepsy in children.
The regulatory win comes as Harmony Biosciences demonstrates robust commercial momentum.