Word, table, figure count
Bullet point summary:
  1. What is already known about this subject :
  2. The clinical efficacy and safety of tezacaftor-ivacaftor in children with cystic fibrosis is highly variable in clinical practice.
  3. Tezacaftor-ivacaftor has two fixed dosing regimens in children: ≥30kg receive the adult dose, and <30kg receive half of the adult dose.
  4. A limited number of pharmacokinetic studies has been performed in children; data are sparsely published. No population pharmacokinetic data of tezacaftor-ivacaftor in children have been published so far.
  5. What this study adds :
  6. The first population pharmacokinetic models with real-world data were developed for tezacaftor-ivacaftor and its active metabolites using prior information from adolescents/adults.
  7. AUC of tezacaftor-ivacaftor varied greatly within and across age groups; a strong correlation between Cmin and AUC was observed.
  8. The developed population pharmacokinetic models for tezacaftor-ivacaftor can be used in future studies evaluating the exposure-response relationship and its variability as a basis for more personalized dosing.