Elexacaftor is a corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) bearing the F508 deletion (F508del) mutation, which is carried by 90% of cystic fibrosis patients, and a CFTR potentiator.1,2 It increases CFTR levels in bronchial epithelial cells isolated from patients with F508del-CFTR mutations when used at a concentration of 2 ?M.1 Elexacaftor potentiates the transepithelial current in primary human nasal epithelial cells (EC50 = 1.5 nM).2 Formulations containing elexacaftor in combination with ivacaftor and tezacaftor have been used in the treatment of cystic fibrosis in patients carrying one or more mutations in the CFTR gene.
1.Keating, D.J., Marigowda, G., Burr, L.E., et al.VX-445–tezacaftor–ivacaftor in patients with cystic fibrosis and one or two Phe508del allelesN. Engl. J. Med.379(17)1612-1620(2018) 2.Shaughnessy, C.A., Zeitlin, P.L., and Bratcher, P.E.Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatmentSci. Rep.11(1)19810(2021)