Heijerman HGM, McKone EF, Downey DG, et al. - In this phase 3, multicentre, randomised, double-blind, active-controlled trial of elexacaftor in combination with tezacaftor plus ivacaftor carried out at 44 sites in four countries of people with cystic fibrosis homozygous for the F508del mutation, aged 12 years or older with stable disease, and with a percentage prognosticated forced expiratory volume in 1 s (ppFEV 1) of 40–90%, inclusive, researchers determined the efficiency and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in such individuals. A total of 113 participants were recruited, of which 107 were randomized in the elexacaftor plus tezacaftor plus ivacaftor group (n = 55) and the tezacaftor plus ivacaftor group (n = 52). In comparison with tezacaftor plus ivacaftor alone, elexacaftor plus tezacaftor plus ivacaftor gave a clinically strong advantage, along with a favorable safety profile, and hence, exhibiting the potential to result in transformative advancements in the lives of individuals with cystic fibrosis who are homozygous for the F508del mutation.