Progress in basic and clinical research of pitolisant in treating narco-lepsy
10.12092/j.issn.1009-2501.2025.08.015
- VernacularTitle:替洛利生治疗发作性睡病的基础与临床研究进展
- Author:
Huimin ZHANG
1
;
Shuqin ZHAN
1
Author Information
1. 首都医科大学宣武医院神经内科,北京 100053
- Publication Type:Journal Article
- Keywords:
pitolisant;
narcolepsy;
H3 receptor in-verse agonist
- From:
Chinese Journal of Clinical Pharmacology and Therapeutics
2025;30(8):1127-1132
- CountryChina
- Language:Chinese
-
Abstract:
Pitolisant,a selective histamine H3 re-ceptor inverse agonist,promotes wakefulness by blocking H3 autoreceptor-mediated feedback inhi-bition to elevate central histamine levels.It is clini-cally indicated for wake promotion in narcolepsy.The development journey of pitolisant spans over two decades,encompassing extensive preclinical and clinical research.These studies have demon-strated both sustained efficacy in managing narco-lepsy and a favorable long-term safety profile.No-tably,pitolisant received formal marketing authori-zation from China's National Medical Products Ad-ministration(NMPA)for treating excessive daytime sleepiness or cataplexy in adult narcolepsy pa-tients.This approval addresses an unmet medical need in China's therapeutic landscape,providing patients with a safe,effective,and well-tolerated novel therapeutic option.This article will summa-rize the basic and clinical research progress of pi-tolisant from the aspects of development history,preclinical studies and pharmacological effects,clin-ical research and indications,medication guide-lines,and safety,aiming to provide a scientific basis for the clinical treatment of narcolepsy.
