Ivacaftor hydrate (VX-770 hydrate) is an orally bioavailable CFTR potentiator, used for cystic fibrosis treatment. In Vitro Ivacaftor (10 µM) increases 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. Ivacaftor (10 μM) significantly increases CFTR activity in W1282X-expressing cells compared to R1162X CFTR cells. Ivacaftor shows no significant activity against 160 targets tested including the GABA A benzodiazepine receptor. Ivacaftor increases the chloride secretion with an EC 50 of 0.236 ± 0.200 μM, a 10-fold shift in potency compared to the F508del HBEs. In recombinant cells, VX-770 increases CFTR channel open probability (Po) in both the F508del processing mutation and the G551D gating mutation. VX-770 increases forskolin-stimulated I T in temperature-corrected F508del-FRT cells by appr 6-fold with an EC 50 of 25 nM . MCE has not independently confirmed the accuracy of these methods. They are for reference only. In Vivo Ivacaftor (1-200 mg/kg, p.o.) exhibits good oral bioavailability in rat. MCE has not independently confirmed the accuracy of these methods. They are for reference only.