Vanzacaftor–tezacaftor–deutivacaftor for children aged 6–11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial
Vanzacaftor–tezacaftor–deutivacaftor was safe and well tolerated and maintained FEV1 % predicted from elexacaftor–tezacaftor–ivacaftor baseline with further improved CFTR function. Improvements in CFTR function compared with baseline elexacaftor–tezacaftor–ivacaftor values demonstrate the potential opportunity to restore normal physiology early and prevent development or progression of cystic fibrosis. Nearly all participants had sweat chloride below the diagnostic threshold for cytstic fibrosis (<60 mmol/L) and more than half had normal levels (<30 mmol/L).
