罕见病酶替代疗法药物研究进展及非临床评价思考  

Research Progress of Enzyme Replacement Therapy for Rare Diseases Products and Their Non-Clinical Evaluation

作　　者：戎怿 刘小聪 尹华静 RONG Yi;LIU Xiaocong;YIN Huajing(Office of Pharmacotoxicology,Center for Drug Evaluation,National Medical Products Administration,Beijing 100076,China)

机构地区：[1]国家药品监督管理局药品审评中心,北京100076

出　　处：《医药导报》2024年第6期895-903,共9页Herald of Medicine

摘　　要：近年来,为缓解中国罕见病药物缺乏现状,满足临床迫切需求,国家出台一系列政策鼓励罕见病药物的研发。酶替代疗法是罕见先天性代谢异常疾病(尤其是酶缺陷类疾病)的标准疗法。由于罕见病酶替代疗法药物分子的“复杂性”与“异质性”,其疾病动物模型的评价是非临床开发与评价的主要难点。该文介绍国内外罕见病酶替代疗法药物开发的最新研究进展,并以国内外有代表性的文献为基础,结合审评实践,简要就此类产品的非临床评价(药理学、药动学和毒理学)提出一些思考和建议。In recent years,in order to meet urgent clinical needs alleviate the shortage of rare disease drugs in China,a series of policies encouraging the research and development of rare disease drugs has been introduced.Enzyme replacement therapy(ERT)is a standard treatment for rare congenital metabolic disorders,especially for enzyme deficiency diseases.Due to the complexity and heterogeneity of drug molecules for rare enzyme replacement therapy,the evaluation of animal models is a major challenge in non-clinical development and evaluation.This article introduces the latest research progress in the development of rare enzyme replacement therapy drugs at home and abroad,put forward some thoughts and suggestions on the non-clinical evaluation(pharmacology,pharmacokinetics,and toxicology)of such products based on representative literature,combined with review practice.

关 键 词：罕见病 酶替代疗法 非临床评价 

分 类 号：R971[医药卫生—药品]