Research Progress of Enzyme Replacement Therapy for Rare Diseases Products and Their Non-Clinical Evaluation
10.3870/j.issn.1004-0781.2024.06.009
- VernacularTitle:罕见病酶替代疗法药物研究进展及非临床评价思考
- Author:
Yi RONG
1
;
Xiaocong LIU
;
Huajing YIN
Author Information
1. 国家药品监督管理局药品审评中心,北京 100076
- Keywords:
Rare disease;
Enzyme replacement therapy;
Nonclinical evaluation
- From:
Herald of Medicine
2024;43(6):895-903
- CountryChina
- Language:Chinese
-
Abstract:
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.
