Description

Ivacaftor is a CFTR activator (IC₅₀ of 43 ± 38 nM). In some cells Ivacaftor was shown to increase CFTR channel open probability in the F508del processing and the G551D gating mutations. In cultured human CF bronchial epithelia (HBE) expressing the F551D gating mutation on one allele and F508del processing mutation on the other allele, Ivacaftor was shown to increase Cl^– secretion by about 10-fold, to about 50% of that observed in HBE isolated from individuals without CF, in addition, Ivacaftor reduced excessive Na⁺ and fluid absorption preventing dehydration of the apical surface and increased cilia beating in these epithelial cultures.