Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More F508del Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial

• Type of Material: Article
• Authors: Matthias Griese, Ludwig Maximilians Universitat Munchen; Stefano Costa, University of Messina; Rachel Linnemann, Emory University; Marcus Mall, Charité–Universitätsmedizin Berlin; Edward F. McKone, St Vincents University Hospital; Deepika Polineni, University of Kansas; Bradley S. Quon, University of British Columbia; Felix C. Ringshausen, Hannover Medical School; Jennifer L. Taylor-Cousar, National Jewish Health; Nicholas J. Withers, Royal Devon & Exeter NHS Foundation Trust; Samuel M. Moskowitz, Vertex Pharmaceuticals Inc; Cori L. Daines, University of Arizona
• Language: English
• Date: 2021-02-01
• Publisher: American Thoracic Society
• Publication Version: Final Published Version
• Copyright Statement: © 2021 by the American Thoracic Society
• License: Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1164/rccm.202008-3176LE
• Title of Journal or Parent Work: American Journal of Respiratory and Critical Care Medicine
• Volume: 203
• Issue: 3
• Start Page: 381
• End Page: 385
• Grant/Funding Information: Supported by Vertex Pharmaceuticals Inc., which participated in the design, statistical analysis, and interpretation of the data and provided editorial and writing assistance.
• Keywords: Science & Technology; Critical Care Medicine; General & Internal Medicine; Respiratory System; Life Sciences & Biomedicine
• Research Categories: Health Sciences, Medicine and Surgery; Chemistry, Pharmaceutical; Health Sciences, Health Care Management

Abstract

To the Editor:Cystic fibrosis (CF) is caused by mutations in the CFTR (CF transmembrane conductance regulator) gene (1). The most common CFTR mutation in populations of European descent is F508del, with up to 90% of people with CF (pwCF) having one or more F508del alleles (2–4).