Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor–tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials

Background The goal of cystic fibrosis transmembrane conductance regulator (CFTR) modulators is to reach normal CFTR function in people with cystic fibrosis.Vanzacaftor-tezacaftor-deutivacaftor restored CFTR function in vitro and in phase 2 trials in participants aged 18 years and older resulting in improvements in CFTR function, as measured by sweat chloride concentrations and lung function as measured by spirometry.We aimed to evaluate the efficacy and safety of vanzacaftor-tezacaftor-deutivacaftor compared with standard of care elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older.Methods In two randomised, active-controlled, double-blind, phase 3 trials, individuals…

• AT
  American Thoracic Society
• A
  AstraZeneca
• CF
  Cystic Fibrosis Foundation Therapeutics
• ER
  European Respiratory Society
• VP
  Vertex Pharmaceuticals
• AP
  Arrowhead Pharmaceuticals
• M
  Mylan
• I
  Insmed
• NI
  National Institute for Health and Care Research
• DO
  Department of Health and Social Care
• CF
  Cystic Fibrosis Trust
• DF
  Deutsche Forschungsgemeinschaft
• FW
  Fonds Wetenschappelijk Onderzoek
• KL
  KU Leuven
• KB
  Koning Boudewijnstichting
• VR
  Vlaamse regering
• NI
  National Institutes of Health
• MB
  Manchester Biomedical Research Centre
• MC
  Manchester Clinical Research Facility