VX-809 (Lumacaftor) is a small molecular corrector and rescues folding defects of F508del mutation in cystic fibrosis transmembrane conductance regulator (CFTR). F508del is the most frequent mutation in CFTR gene that leads to cystic fibrosis (CF). VX-809 (Lumacaftor) directly binds to the pocket created by the deleted F508 in CFTR and promotes the cellular processing and maturation of F508del-CFTR in primary human bronchial epithelia (EC50 81 nM). [1] [2]
VX-809 (Lumacaftor) has been approved for cystic fibrosis treatment and also subjected to multiple Phase III clinical trials in cystic fibrosis patients. [3] [4]