Page 17 - 《中国药科大学学报》2026年第1期

学报
Journal of China Pharmaceutical University 2026, 57(1): 11 − 18 11

2020−2024 年：解锁 “老药新用” 新趋势与新策略

陈方溯 ，杨俊杰 ，杜佳玉 ，黄诗淼 ，张宇轩 ，尤启冬 ，王 磊 ，张秋月 1,2**
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（ 中国药科大学江苏省创新药物设计与成药性优化重点实验室, 南京 210009； 中国药科大学药学院, 南京 210009）
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摘 要 创新药物研究和开发已经取得显著进展，但漫长的研发周期和高失败率成为其有待突破的瓶颈。老药新用作为克
服该障碍的战略路径，通过发掘已获批药物的新适应证，近年来在基础研究和临床转化方面发展迅速。该策略依托丰富的药
物库资源，确证的安全性、高研发效率和低成本等核心优势，成为优化药物研发流程的重要方向。本综述系统梳理了近五年
获批临床应用的老药新用案例，归纳出以靶点、通路、药物相似性、用药后表型及临床不良反应为核心策略的老药新用研究思
路，以期为老药新用提供策略性指导。
关键词 老药新用；药物研发策略；新适应证；研究进展
中图分类号 R914;R969 文献标志码 A 文章编号 1000−5048(2026)01−0011−08
doi：10.11665/j.issn.1000−5048.2025072102

引用本文 陈方溯，杨俊杰，杜佳玉，等. 2020−2024 年：解锁 “老药新用” 新趋势与新策略 [J]. 中国药科大学学报，2026，57（1）：11-18.

Cite this article as: CHEN Fangsu, YANG Junjie, DU Jiayu, et al. New trends and new strategies of drug repurposing: 2020–2024[J]. J China
Pharm Univ, 2026, 57(1): 11-18.

New trends and new strategies of drug repurposing: 2020–2024
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CHEN Fangsu , YANG Junjie , DU Jiayu , HUANG Shimiao , ZHANG Yuxuan , YOU Qidong ,
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WANG Lei , ZHANG Qiuyue 1,2**
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Jiangsu Provincial Key Laboratory of Drug Design and Optimization, China Pharmaceutical University, Nanjing 210009;
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School of Pharmacy, China Pharmaceutical University, Nanjing 210009, China
Abstract The research and development of innovative drug have progressed remarkably, but the long
development circle and high failure rate have become the bottleneck. Drug repurposing, discovering new
indications of approved drugs, is a strategy to overcome these obstacles. By exploring new indications for
approved drugs, rapid progress has been made in basic research and clinical translation in recent years. Rich
resources of drugs, proven security, efficient development workflow and reduced cost are core advantages of this
strategy, making the strategy a crucial direction of optimizing the pipeline of drug research and development.
This review systematically summarizes drug repurposing cases that have received clinical approval over the past
five years, and proposes core strategies for drug repurposing, including approaches based on targets, pathways,
drug similarity, post-treatment phenotypes, and clinical side effects, aiming to provide some strategic guidance
for drug repurposing efforts.
Key words drug repurposing; drug development strategies; new indications; research progress

This study was supported by the National Natural Science Foundation of China (No.82173741，No.82304309)；the Natural Science
Foundation of Jiangsu Province (BK20231014)； and the National Innovation and Entrepreneurship Training Program for
Undergraduate（No.202510316017）

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收稿日期 2025-07-21 通信作者 E-mail：leiwang.91@cpu.edu.cn
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E-mail：zhangqiuyue_1994@163.com
基金项目 国家自然科学基金项目 (No.82173741, No.82304309)；江苏省自然科学基金项目 (BK20231014)；国家级大学生创新创业训练
计划项目（No.202510316017）