ORKAMBI Targets the Complex Protein Defects of the F5O8del-CFTR Protein1
- In vitro responses do not necessarily correspond to in vivo pharmacodynamic response or clinical benefit
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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, and how lumacaftor and ivacaftor work together to increase the quantity, stability, and function of F508del-CFTR protein resulting in increased chloride ion transport. In vitro responses do not necessarily correspond to in vivo pharmacodynamic response or clinical benefit.