中国临床药理学杂志2026，Vol.42Issue(10)：1457-1461,5.DOI:10.13699/j.cnki.1001-6821.2026.10.017
治疗软骨发育不全的新药—长效C型利钠肽前药Navepegritide
Long-acting C-type natriuretic peptide prodrug Navepegritide,a new approveddrug for the treatment of achondroplasia
李妍 1何心 2胡爽2
作者信息
- 1. 山西省儿童医院、山西省妇幼保健院药剂科,山西太原 030013
- 2. 南京医科大学附属妇产医院、南京市妇幼保健院药学部,江苏南京 210004
- 折叠
摘要
Abstract
Achondroplasia(ACH)is the most common hereditary short limb dwarfism,caused by functional acquired mutations in the fibroblast growth factor receptor 3(FGFR3)gene.In recent years,significant breakthroughs have been made in precision therapy targeting downstream pathways of FGFR3.Navepegritide(product name:YUVIWEL)is a long-acting C-type natriuretic peptide(CNP)prodrug that uses transient conjugation(TransCon)technology to couple CNP to PEG carrier molecules through a cleavable linker,achieving weekly subcutaneous administration.It was approved by the US Food and Drug Administration(FDA)in February 2026 to promote linear growth in children aged 2 years and above with non-closed epiphyseal plates and cartilage dysplasia.This article introduces the pharmacological properties,key clinical trial evidence,safety features,and clinical value of navepegritide,providing reference for clinical medication.关键词
软骨发育不全/Navepegritide/TransCon CNP/YUVIWELKey words
achondroplasia/Navepegritide/TransCon CNP/YUVIWEL分类
医药卫生引用本文复制引用
李妍,何心,胡爽..治疗软骨发育不全的新药—长效C型利钠肽前药Navepegritide[J].中国临床药理学杂志,2026,42(10):1457-1461,5.
