Efficacy and safety of pitolisant in children above 6 years with narcolepsy

INTRODUCTION: The newly approved use of pitolisant in pediatric narcolepsy marks a significant advancement for patients and clinicians, given the scarcity of medications for this age group that are both safe and effective in reducing narcolepsy symptoms and improving quality of life. AREAS COVERED: This article covers the use of pitolisant for treating narcolepsy type 1 (NT1) and 2 (NT2) in pediatric patients considering drug's pharmacokinetics and pharmacodynamics. By integrating recent literature and real-world data, the safety, the tolerability and the efficacy of this drug have been analyzed, also including evidence drawn from studies involving patients with Prader-Willi Syndrome. EXPERT OPINION: Pitolisant represents a groundbreaking treatment for pediatric narcolepsy, addressing the paucity of safe and effective options for this age group. Its unique mechanism as a histamine H3 receptor antagonist reduces excessive daytime sleepiness and cataplexy while offering cognitive benefits. With a favorable safety profile and good tolerability, pitolisant efficiently outperforms traditional therapies, which often have distressing side effects. Its use is particularly critical in childhood, a developmental stage where factors like growth, school performance, and socialization must be carefully considered, making it a transformative option for pediatric care.