Elexacaftor

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., It increases CFTR levels in bronchial epithelial cells isolated from patients with F508del-CFTR mutations when used at a concentration of 2 µM. Elexacaftor potentiates the transepithelial current in primary human nasal epithelial cells (EC50 = 1.5 nM). 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.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. CAS Number: 2216712-66-0. Synonyms: VX-445. Molecular Formula: C26H34F3N7O4S. Formula Weight: 597.7. Purity: >98%. Formulation: (Request formulation change), A solid. Solubility: DMSO: soluble. lambdamax: 279, 350 nm. SMILES: CC(C(F)(F)F)(C)COC1=NN(C2=NC(N3C(C)(C)C[C@H](C)C3)=C(C(NS(C4=CN(C)N=C4C)(=O)=O)=O)C=C2)C=C1. InChi Code: InChI=1S/C26H34F3N7O4S/c1-16-12-25(5,6)35(13-16)22-18(23(37)33-41(38,39)19-14-34(7)31-17(19)2)8-9-20(30-22)36-11-10-21(32-36)40-15-24(3,4)26(27,28)29/h8-11,14,16H,12-13,15H2,1-7H3,(H,33,37)/t16-/m0/s1. InChi Key: MVRHVFSOIWFBTE-INIZCTEOSA-N.