A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele

Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in patients ≥12 years of age with cystic fibrosis and at least one F508del-CFTR (cystic fibrosis transmembrane conductance regulator) allele, but it has not been evaluated in children <12 years of age. Objectives: To assess the safety, pharmacokinetics, and efficacy of ELX/TEZ/IVA in children 6 through 11 years of age with F508del-minimal function or F508del-F508del genotypes. Methods: In this 24-week open-label phase 3 study, children (N = 66) weighing <30 kg received 50% of the ELX/TEZ/IVA adult daily dose (ELX 100 mg once daily, TEZ 50 mg once daily, and IVA 75 mg every 12 h) whereas children weighing

• Zemanick, E. T.
• Taylor-Cousar, J. L.
• Davies, J.
• Gibson, R. L.
• Mall, M. A.
• McKone, E. F.
• McNally, P.
• Ramsey, B. W.
• Rayment, J. H.
• Rowe, S. M.
• Tullis, E.
• Ahluwalia, N.
• Chu, C.
• Ho, T.
• Moskowitz, S. M.
• Noel, S.
• Tian, S.
• Waltz, D.
• Weinstock, T. G.
• Xuan, F.
• Wainwright, C. E.
• McColley, S. A.