Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children ≥6 Years with Cystic Fibrosis and at Least One F508del Allele: A 192-Week, Phase 3, Open-Label Extension Study.

doi:10.1164/rccm.202502-0512OC

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Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children ≥6 Years with Cystic Fibrosis and at Least One F508del Allele: A 192-Week, Phase 3, Open-Label Extension Study.

American journal of respiratory and critical care medicine - 1 Oct 2025

Wainwright Claire, McColley Susanna A, McNally Paul, Powers Michael, Ratjen Felix, Rayment Jonathan H, Retsch-Bogart George, Roesch Erica, Ramsey Bonnie, McKone Edward F, Tullis Elizabeth, Mall Marcus A, Taylor-Cousar Jennifer L, Waltz David, Ahluwalia Neil, Chu Chenghao, Scirica Christina V, Davies Jane C

Abstract excerpt

Rationale: Elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) was shown to be safe and efficacious in children 6 through 11 years of age with cystic fibrosis (CF) and at least one F508del allele in a 24-week phase 3 study. Children completing this study could enroll into a 192-week extension study. Objectives: To evaluate the long-term safety and efficacy of ELX/TEZ/IVA in children ≥6 years of age. Methods: In...

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PMID: 40454869
DOI: 10.1164/rccm.202502-0512OC

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CFTR

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