Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251).

doi:10.1016/j.jcf.2020.11.002

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Article

Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251).

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2021

Kazani Shamsah, Rowlands David J, Bottoli Ivan, Milojevic Julie, Alcantara Jose, Jones Ieuan, Kulmatycki Kenneth, Machineni Surendra, Mostovy Lidia, Nicholls Ian, Nick Jerry A, Rowe Steven M, Simmonds Nicholas J, Vegesna Raju, Verheijen Jeroen, Danahay Henry, Gosling Martin, Ayalavajjala Phaninatha Sarma, Salman Mohammed, Strieter Robert

Abstract excerpt

BACKGROUND: This is the first-in-human study of icenticaftor, an oral potentiator of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) channel. Restoration of CFTR activity has shown significant clinical benefits, but more studies are needed to address all CFTR mutations. METHODS: Safety, pharmacodynamics/pharmacokinetics of icenticaftor were evaluated in a randomized, double-blind,...

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PMID: 33293212
DOI: 10.1016/j.jcf.2020.11.002
PMCID: PMC8935475

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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