Pitolisant (WAKIX^®), a histamine H₃ receptor antagonist/inverse agonist that has been developed by Bioprojet Pharma, is approved in the EU and USA and elsewhere for use in adults with narcolepsy with or without cataplexy. In February 2023, based on clinical data in patients aged 6 to < 18 years, pitolisant received its first approval in adolescents and children from the age of 6 years for the treatment of narcolepsy with or without cataplexy in the EU. This article summarizes the milestones in the development of pitolisant leading to this pediatric first approval for narcolepsy with or without cataplexy.

Pitolisant (WAKIX ^® ) 是一种组胺 H ₃受体拮抗剂/反向激动剂，由 Bioprojet Pharma 开发，已在欧盟、美国和其他地方批准用于治疗伴有或不伴有猝倒的发作性睡病成人。2023 年 2 月，基于 6 岁至 18 岁以下患者的临床数据，pitolisant 在欧盟首次获批用于治疗 6 岁以上青少年和儿童，用于治疗伴有或不伴有猝倒的发作性睡病。本文总结了pitolisant开发的里程碑，该药物首次被批准用于治疗伴有或不伴有猝倒的发作性睡病。