Ivacaftor (VX-770)

Ivacaftor (VX-770) is a CFTR potentiator with an IC50 of 43 ± 38 nM. In recombinant cells Ivacaftor (VX-770) increased CFTR channel open probability (P(o)) in both the F508del processing mutation and the G551D gating mutation. Ivacaftor (VX-770) also increased Cl(-) secretion in cultured human CF bronchial epithelia (HBE) carrying the G551D gating mutation on one allele and the F508del processing mutation on the other allele by approximately 10-fold, to approximately 50% of that observed in HBE isolated from individuals without CF. Furthermore,Ivacaftor (VX-770) reduced excessive Na(+) and fluid absorption to prevent dehydration of the apical surface and increased cilia beating in these epithelial cultures.