Icenticaftor is a new CTFR potentiator whose safety and efficacy were demonstrated by Kazani et al. in their first-in-human RCT of this molecule (NCT02190604) that included both healthy subjects and CF patients with ≥1 pre-specified CFTR Class III or IV mutation, or homozygous for F508del mutation [28]. This evidence concerns the gene CFTR and cystic fibrosis.