Table 1. Mechanism of action and targets of emerging cystic fibrosis transmembrane conductance regulator (CFTR) modulators
Mutation classNature of defectExample mutationsPancreatic function#Modulator strategyAgents in development
Suppressor and potentiator
Corrector plus potentiator
VX-809 plus ivacaftor+
VX-661 plus ivacaftor+
IIIChannel gatingG551DInsufficientPotentiatorIvacaftor (FDA approved, G551D)
Other gating mutations?
IVChannel conductanceR117HSufficientPotentiator?No clinical data available
VLow transcript levels3849+10kb C->TSufficientPotentiator?No clinical data available
  • FDA: US Food and Drug Administration. #: when in trans with a non-functional CFTR mutation; : theoretical approach based on nature of defect and known drug mechanism; +: currently in phase II trials. Reproduced from [15] with permission from the publisher.