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Mechanism of Action

ORKAMBI Targets the Complex Protein Defects of the F508del-CFTR Protein¹

In vitro responses do not necessarily correspond to in vivo pharmacodynamic response or clinical benefit

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Downloadable Overview

View this document to explore the mechanism of action of ORKAMBI at the cellular level. 

Topics include:

  • Normal CFTR protein function
  • The F508del-CFTR mutation
  • How lumacaftor and ivacaftor work together to increase the quantity, stability, and function of F508del-CFTR protein resulting in increased chloride ion transport


MOA Downloadable Overview