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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

Click here for full Prescribing Information.

 

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