治疗软骨发育不全的新药—长效C型利钠肽前药NavepegritideOA

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.