VX-770

VX-770 (Ivacaftor) is a potentiator of CFTR, which targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively. VX-770 (Ivacaftor) inhibits excessive ENaC-mediated Na+ and fluid absorption with an IC50 of 43 nM, and decreases the amiloride response, resulting in an increase in the surface fluid and cilia beat frequency (CBF) in G551D/F508del HBE. VX-770 (Ivacaftor) is the first potent and orally available CFTR potentiator to enter human clinical trials. It showed that VX-770 (Ivacaftor) was associated with within-subject improvements in CFTR and lung function. These findings provide support for further studies of pharmacologic potentiation of CFTR as a means to treat cystic fibrosis.