• is an online educational resource on the genetics of cystic fibrosis (CF) and the role of CF transmembrane conductance regulator (CFTR) in normal human physiology as well as in CF.
  • The information contained herein is intended for healthcare professionals working within the European Union (EU).
  • The content of this website has been developed independently by a group of CF healthcare professionals and is for general information purposes only.
  • All of the information about CFTR and CFTR modulators has been obtained from referenced sources and is in the public domain.
  • This website is not intended to promote or advocate a particular course of treatment or any specific drug, whether licensed or unlicensed, nor is it intended to imply or suggest that any drug be used for any purpose other than that which for which it is licensed in the EU.


  • While we endeavour to keep the information up to date and correct, we make no representations or warranties of any kind, express or implied, about the completeness, accuracy, reliability, suitability or availability with respect to the website or the information and related graphics, or products and treatments, contained on the website for any purpose. Any reliance you place on such information is therefore strictly at your own risk.
  • In no event will we be liable for any loss or damage including without limitation, indirect or consequential loss or damage, or any loss or damage whatsoever arising from, or in connection with, the use of this website.
  • Through this website you are able to link to other websites which are not under our control. We have no control over the nature, content and availability of those sites. The inclusion of any links does not necessarily imply a recommendation or endorse the views expressed within them.
  • Every effort is made to keep the website up and running smoothly. However, we take no responsibility for, and will not be liable for, the website being temporarily unavailable due to technical issues beyond our control.

Please see the full Terms & Conditions before using this website.


I have read and understood the above

Clinical management of CF » CFTR modulators » CFTR modulators approved

CFTR modulators approved

The following CFTR modulator is currently approved for clinical use.


Ivacaftor (previously VX-770, commercialised as Kalydeco™) is currently the only approved CFTR potentiator by the US FDA and European Commission for the treatment of patients with CF aged 6 years and older who have at least one copy of the G551D mutation or any of the following gating mutations: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.1,2 In Canada, ivacaftor was approved for use in patients with CF aged 6 years and older who have the aforementioned CTFR mutations in addition to G970R.2

Ivacaftor and lumacaftor combination
In July 2015, the US FDA approved the first drug,
lumacaftor 200 mg/ivacaftor 125 mg, for the treatment of patients with CF aged 12 years and older who have two copies of the F508del mutation.3 Patients with two copies of this mutation (one inherited from each parent) represent the largest population of CF patients.3

  1. US Food & Drug Administration Approves Kalydeco™ (ivacaftor) for Use in Eight Additional Mutations that Cause Cystic Fibrosis. 21 February 2014.
  2. Vertex Receives European Approval for Kalydeco™ (ivacaftor) in Eight Non-G551D Gating Mutations.
  3. FDA News Release. FDA approves new treatment for cystic fibrosis. Available at: July 2, 2015