Mechanism and clinical evaluation of a novel drug nedosiran for the treatment of primary hyperoxaluria type 1
DU Wen-lin
LUO Chun-mei
WU Xiao-yan
ZHANG Rong
Abstract：Nedosiran is a novel small interfering RNA drug developed by Novo Nordisk Pharmaceutical company for the treatment of primary hyperoxaluria type 1.It targets lactate dehydrogenase A in liver cells and reduces the expression of lactate dehydrogenase.Nedosiran effectively reduces oxalate production and prevents calcium oxalate crystal deposition,thereby reducing urinary oxalate levels.It was approved by the US FDA on September 29th,2023 and indicated to lower urinary oxalate levels in children at 9 years of age and adults with primary hyperoxaluria type 1.This article summarizes the use and dosage,mechanism of action,pharmacokinetics,clinical evaluation and safety of nedosiran to provide a theoretical reference for clinically safe and rational use of this drug.
Keywords：nedosiranprimary hyperoxaluria type 1mechanism of actionclinical evaluation
Publication Date：2024-10-15
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：5( 2023-2027 )