摘要
近年来,全球多款干细胞衍生胰岛细胞药物快速推进临床,在糖尿病治疗及功能性治愈方面展现出较大应用潜力。目前,在我国已获批开展注册临床研究的同类产品尚处于早期临床研究阶段。鉴于对该类药物的科学认知及人用经验有限,其关键质量属性与临床安全有效性的相关性尚未建立,药学研发及监管均面临较大挑战。此类药物在活性成分、结构、功能与作用机制等方面与其他细胞治疗药品存在一定差异,因此,在评价其安全性、有效性及质量可控性方面应特别关注。基于此类药物的特点及研发申报进展,参考细胞治疗药品相关指南以及胰岛移植技术质量控制规范,本文旨在重点阐述干细胞衍生胰岛细胞药物开发及申报中的质量风险管理策略及药学评价考虑,为此类药物的研发及审评提供参考。
In recent years,multiple stem cell-derived pancreatic islet cell medicinal products globally have advanced rapidly into clinical trials,demonstrating great application potential in the treatment and functional cure of diabetes mellitus.Currently,the similar medicinal products that have been approved for registration clinical trials in China are still in the early clinical research stages.Given the limited scientific cognition and application experience in human beings,the correlation between critical quality attributes of such products and their clinical safety and efficacy has not been established.Both the pharmaceutical research and development as well as regulation face major challenges.In view of differences between such medicinal products and other cell therapy medicinal products in terms of active ingredients,structures,functions and mechanisms of action,particular attention should be given to the evaluation of safety,efficacy,and quality controllability.Based on the characteristics,the progress in research and development as well as application of such products,including relevant guidelines for cell therapy medicinal products and the quality control practices for pancreatic islet transplantation technology for reference,this article aims at focusing on the quality risk management strategies and pharmaceutical evaluation considerations during the development and application of stem cell-derived pancreatic islet cell medicinal products,providing references for the research and development,as well as evaluation of such products.
作者
刘丹
卢加琪
尹慧芳
韦薇
LIU Dan;LU Jia-qi;YIN Hui-fang;WEI Wei(Center for Drug Evaluation,National Medical Products Administration,Beijing 100076,China;Yangtze River Delta Center for Drug Evaluation and Inspection of National Medical Products Administration,Shanghai 201210,China)
出处
《中国新药杂志》
北大核心
2025年第24期2598-2604,共7页
Chinese Journal of New Drugs
关键词
糖尿病
干细胞衍生胰岛细胞药物
质量风险管理
药学评价
diabetes mellitus
stem cell-derived pancreatic islet cell medicinal products
quality risk management
pharmaceutical evaluation
