替洛利生治疗发作性睡病的基础与临床研究进展

doi:10.12092/j.issn.1009-2501.2025.08.015

中国临床药理学与治疗学 ›› 2025, Vol. 30 ›› Issue (8): 1127-1132.doi: 10.12092/j.issn.1009-2501.2025.08.015

替洛利生治疗发作性睡病的基础与临床研究进展

张慧敏, 詹淑琴

首都医科大学宣武医院神经内科,北京 100053

收稿日期:2025-05-06 修回日期:2025-06-28 发布日期:2025-08-12
通讯作者: 詹淑琴,女,教授,研究方向：睡眠障碍,发作性睡病。E-mail: shqzhan@hotmail.com
作者简介:张慧敏,女,硕士,研究方向：睡眠障碍,发作性睡病。E-mail: zhanghuimin199803@163.com
基金资助:
国家自然科学基金（82371489）; 国家重点研发计划项目（2021YFC2501400）; 国家科技创新2030-“脑科学与类脑研究”重大项目（2021ZD200600）

Progress in basic and clinical research of pitolisant in treating narcolepsy

ZHANG Huimin, ZHAN Shuqin

Department of Neurology, Xuanwu Hospital, Capital Medical University, Beijing 100053, China

Received:2025-05-06 Revised:2025-06-28 Published:2025-08-12

摘要/Abstract

摘要： 替洛利生是一种H3受体反向激动剂,通过阻断H3受体的负反馈作用,增加大脑中的组胺水平,从而提高觉醒度,被用于发作性睡病的促觉醒治疗。替洛利生的研发历程跨越了二十多年,这一过程中进行了大量的基础与临床研究。这些研究不仅证实了替洛利生在治疗发作性睡病方面的长期有效性,也证明了其良好的安全性。特别是在中国,替洛利生获得了国家药品监督管理局（NMPA）的正式注册批准,用于治疗发作性睡病成人患者的日间过度思睡或猝倒,填补了我国在这一治疗领域的空白,为患者提供了一个安全、有效和便捷的治疗新选择。本文将从研发历史、临床前研究与药理作用、临床研究及适应症和用药指南、安全性等方面总结替洛利生的基础和临床研究进展,以期为发作性睡病的临床治疗提供科学依据。

关键词: 替洛利生, 发作性睡病, H3受体反向激动剂

Abstract: Pitolisant, a selective histamine H3 receptor inverse agonist, promotes wakefulness by blocking H3 autoreceptor-mediated feedback inhibition to elevate central histamine levels. It is clinically indicated for wake promotion in narcolepsy. The development journey of pitolisant spans over two decades, encompassing extensive preclinical and clinical research. These studies have demonstrated both sustained efficacy in managing narcolepsy and a favorable long-term safety profile. Notably, pitolisant received formal marketing authorization from China's National Medical Products Administration (NMPA) for treating excessive daytime sleepiness or cataplexy in adult narcolepsy patients. 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 summarize the basic and clinical research progress of pitolisant from the aspects of development history, preclinical studies and pharmacological effects, clinical research and indications, medication guidelines, and safety, aiming to provide a scientific basis for the clinical treatment of narcolepsy.

Key words: pitolisant, narcolepsy, H3 receptor inverse agonist

中图分类号:

R740

张慧敏, 詹淑琴. 替洛利生治疗发作性睡病的基础与临床研究进展[J]. 中国临床药理学与治疗学, 2025, 30(8): 1127-1132.

ZHANG Huimin, ZHAN Shuqin. Progress in basic and clinical research of pitolisant in treating narcolepsy[J]. Chinese Journal of Clinical Pharmacology and Therapeutics, 2025, 30(8): 1127-1132.

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替洛利生治疗发作性睡病的基础与临床研究进展

Progress in basic and clinical research of pitolisant in treating narcolepsy

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