Formulation: A solid
Formal Name: N-[(1,3-dimethyl-1H-pyrazol-4-yl)sulfonyl]-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethyl-1-pyrrolidinyl]-3-pyridinecarboxamide
Purity: ≥98%
Formula Markup: C26H34F3N7O4S
Formula Weight: 597,7
Shelf life (days): 1460
CAS Number: 2216712-66-0
Notes: 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.{62803},{62804} It increases CFTR levels in bronchial epithelial cells isolated from patients with F508del-CFTR mutations when used at a concentration of 2 µM.{62803} Elexacaftor potentiates the transepithelial current in primary human nasal epithelial cells (EC50 = 1.5 nM).{62804} 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.