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The FDA has approved a supplemental new drug application for pitolisant (Wakix, Harmony Biosciences) for the treatment of cataplexy in children as young as 6 years with narcolepsy.
Pitolisant was first approved in August 2019 for the treatment of excessive daytime sleepiness (EDS) 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 EDS in pediatric patients aged 6 years or older with narcolepsy.
With this new indication, pitolisant is now the “first-and-only FDA-approved treatment option for both pediatric and adult narcolepsy patients with or without cataplexy that is not scheduled as a controlled substance, an important distinction that supports its clinical utility,” Jeffrey M. Dayno, MD, president and CEO of Harmony Biosciences, said in a statement.
Pitolisant is a selective histamine 3 receptor antagonist/inverse agonist that works through a novel mechanism of action to increase the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain. It is administered orally once daily in the morning upon waking.
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).
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.
Complete prescribing information is available online.
