FDA Approves Wakix for Cataplexy in Pediatric Narcolepsy Patients
The FDA approved pitolisant (Wakix) for treating cataplexy in children ages 6 and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
On Feb. 17, 2026, 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. With this approval, Wakix becomes the first and only FDA-approved non-scheduled treatment for narcolepsy in both pediatric and adult patients, with or without cataplexy.
Wakix was first approved in August 2019 for the treatment of excessive daytime sleepiness in adults with narcolepsy. The indication was expanded in October 2020 to include cataplexy in adults. In June 2024, the FDA approved pitolisant for the treatment of excessive daytime sleepiness in pediatric patients aged 6 years or older with narcolepsy.
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. In a randomized, placebo-controlled trial of children aged 6-17 years with narcolepsy with or without cataplexy, pitolisant significantly reduced narcolepsy symptoms compared with placebo (P = .007). Findings from the trial were published in The Lancet Neurology and supported the safety and efficacy of pitolisant in children and adolescents.
The most common adverse reactions (≥ 5% and greater than placebo) observed with pitolisant in children were headache (19%) and insomnia (7%). The overall adverse reaction profile of the drug in the pediatric clinical trial was similar to that seen in the adult clinical trial program.
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.
Wakix received orphan drug designation for narcolepsy in 2010 and breakthrough therapy designation for cataplexy in 2018.
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.