为科学指导和规范用于罕见病的酶替代疗法（ERT）药物的非临床研究与评价，促进罕见病治疗药物的研发，中国于2024年2月23日发布了《罕见病酶替代疗法药物非临床研究指导原则》，对该指导原则进行全面解读，包括起草背景、起草过程及思路和相关内容，并结合具体案例重点阐述罕见病ERT药物非临床药效学、药动学和毒理学研究的关注要点，旨在为该类药物的研发提供参考。

To provide scientific guidance and standardize the nonclinical investigation and evaluation of enzyme replacement therapy（ERT） products intended for rare diseases and foster the development of products for rare diseases, China issued Guidance on Nonclinical Evaluation of Enzyme Replacement Therapy Products for Rare Diseases on 23 February, 2024. This article presents a comprehensive interpretation of the guidance, covering the drafting background, idea and related content. It highlights the pivotal scientific considerations for the nonclinical pharmacodynamics, pharmacokinetics and toxicology research evaluation of ERT products drawing on concrete case studies, combined with some cases, aiming to provide reference for drug researchers.

R95