Ivacaftor displays no significant activity against 160 targets tested including the GABAA benzodiazepine receptor. Ivacaftor enhances the chloride secretion (EC50 of 0.236 ± 0.200 μM). Compared to the F508del HBEs, it has a 10-fold shift in potency [3]. Ivacaftor (10?μM) enhances the PC secretion activity by 3-fold for ABCB4-G535D, 13.7-fold for ABCB4-G536R, 6.7-fold for ABCB4-S1076C, 9.4-fold for ABCB4-S1176L, and 5.7-fold for ABCB4-G1178S. Ivacaftor corrects the functional defect of ABCB4 mutants[1]. Ivacaftor (10 μM) obviously increases CFTR activity in W1282X-expressing cells compared to R1162X CFTR cells[2]. VX-770 increases CFTR channel open probability (Po) in both the F508del processing mutation and the G551D gating mutation, in recombinant cells. VX-770 increases forskolin-stimulated IT in temperature-corrected F508del-FRT cells by appr 6-fold with an EC50 of 25 nM[4].

In rat, Ivacaftor (1-200 mg/kg, p.o.) shows good oral bioavailability[3].