Elexacaftor/Tezacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients With Cystic Fibrosis.

ABSTRACT: Elexacaftor/tezacaftor/ivacaftor (ETI) significantly improves treatment outcomes for people with cystic fibrosis (pwCF) with at least one F508del allele. In 2023, the Food and Drug Administration approved ETI for children with CF aged 2-5 years. However, real-world pharmacokinetic-pharmacodynamic data for ETI in pediatric and adult populations are still limited. This study aimed to characterize the population PK of ETI in children with CF (chCF) and evaluate current dosing recommendations. Population PK modeling was conducted using Monolix software on 150 ETI concentrations obtained from therapeutic drug (TDM) monitoring in 96 children with CF aged 2-18 years, as part of the MODUL-CF study. Area under the curve was derived from individual Bayesian pharmacokinetic estimates. A one-compartme

SUBMITTER: Truong NH 

PROVIDER: S-EPMC12063479 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature