TABLE 3

Nonclinical end-points used in different cell lines to demonstrate efficacy of ivacaftor, lumacaftor and orkambi

Nonclinical end-pointCell mutationIvacaftorLumacaftorOrkambi
Total ionic current (IT) (FRT cells)G551D
F508del
Total ionic current (IT) (HBE cells)F508del
G551D/F508del
Open probability (PO) (NIH-3T3 cells)G551D
F508del#
CFTR maturation (FRT cells)F508del
CFTR maturation (HBE cells)F508del

IT: total ionic current; FRT: Fischer rat thyroid; HBE: human bronchial epithelial; PO: open channel probability; CFTR: cystic fibrosis transmembrane conductance regulator. #: type of cells not reported.