On Feb. 17, 2026, Harmony Biosciences announced that the U.S. Food and Drug Administration approved a supplemental new drug application for Wakix (pitolisant) tablets to treat cataplexy in children ages 6 years and older with narcolepsy.
About Wakix
Wakix is a selective histamine-3 (H₃) receptor antagonist/inverse agonist. While its precise mechanism of action is not fully understood, its therapeutic effects are believed to be mediated through increased synthesis and release of histamine, a wake-promoting neurotransmitter.
Wakix is administered once daily in the morning. Unlike many therapies used to manage narcolepsy symptoms, Wakix is not a stimulant. In an adult clinical study in which Wakix was taken alongside sodium oxybate or modafinil, there were no significant effects on the levels of medications in the body.
The pediatric approval for cataplexy was supported by results from a phase 3 clinical trial (NCT02611687) involving patients ages 6 to 17 years with narcolepsy. The study demonstrated statistically significant reductions in excessive daytime sleepiness and cataplexy at daily doses ranging from 5 mg to 40 mg. Findings from the trial were published in The Lancet Neurology and supported the safety and efficacy of pitolisant in children and adolescents.
Wakix received orphan drug designation for narcolepsy in 2010 and breakthrough therapy designation for cataplexy in 2018. The FDA initially approved Wakix in August 2019 for the treatment of excessive daytime sleepiness in adults with narcolepsy, with an expanded adult indication for cataplexy granted in October 2020. In June 2024, approval was extended to include excessive daytime sleepiness in pediatric patients ages 6 years and older.
With this latest approval, Wakix becomes the first and only FDA-approved non-scheduled treatment for narcolepsy in both pediatric and adult patients, with or without cataplexy.
About narcolepsy
Narcolepsy is a chronic neurologic disorder characterized by dysregulation of the sleep–wake cycle. Common symptoms include excessive daytime sleepiness and, in some patients, episodes of sudden muscle weakness known as cataplexy. Affecting an estimated one in 2,000 individuals, narcolepsy is frequently underdiagnosed and can significantly interfere with daily functioning, underscoring the importance of timely recognition and appropriate management.
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