Elexacaftor/tezacaftor/ivacaftor in children aged

AIMS: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious and safe in children aged 6-11 years with cystic fibrosis (CF) heterozygous for F508del and a minimal function CFTR variant (F/MF genotypes) in a 24-week, placebo-controlled trial. We conducted a 96-week open-label extension study for children who completed the 24-week parent study. METHODS: In this phase 3b extension study, dosing was based on weight and age with children weighing <30 kg and aged <12 years receiving ELX 100 mg once daily (qd), TEZ 50 mg qd, and IVA 75 mg every 12 h (q12) and children

• Mall, M. A.
• Wainwright, C. E.
• Legg, J.
• Chilvers, M.
• Gartner, S.
• Dittrich, A. M.
• Stehling, F.
• Conner, S.
• Grant, S.
• Suresh, N.
• Weinstock, T. G.
• Davies, J. C.