Ivacaftor for cystic fibrosis in patients aged 12 or over with G551D - CFTR mutation

Respiratory Disease and Thoracic Surgery

Date of publication: 1st December 2010

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Technology description:

Ivacaftor (VX-770) is a CF transmembrane conductance regulator agonist. It acts as a `potentiator’ increasing the activity of the defective CF transmembrane regulator (CFTR) protein. Ivacaftor is designed to restore the function of the mutated CFTR protein. In clinical trials, ivacaftor is administered orally at 150mg twice daily in addition to standard care.