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Papers of the Week

January 27, 2023

J Cyst Fibros


Long-term tezacaftor/ivacaftor safety and efficacy in people with cystic fibrosis and an F508del-CFTR mutation: 96-week, open-label extension of the EXTEND trial.


Flume PA, Harris SR, Paz-Diaz H, Ahluwalia N, Higgins M, Campbell D, Berhane I, Shih JL, Sawicki G
J Cyst Fibros. 2022 Dec 27.
PMID: 36581484.


Study 661-110 (EXTEND) is a phase 3, open-label, three-part rollover study designed to assess the long-term safety and efficacy of tezacaftor/ivacaftor (TEZ/IVA) in participants aged ≥12 years homozygous for F508del (F/F) or heterozygous for F508del and a residual function mutation (F/RF). TEZ/IVA was shown to be safe and efficacious for up to 120 weeks in Part A. Here we report results from Part B, which evaluated safety and efficacy for an additional 96 weeks.