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Clinical management of CF » CFTR modulators » Summary


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To summarise:

Clinical investigations are underway to evaluate the safety and efficacy of combining potentiators and correctors together. It is important to note, however, that CFTR modulators have been developed to ‘repair’ specific mutation defects and not entire classes of mutations.


Potentiators and correctors


Potentiators increase the flow of Cl through CFTR channels at the cell surface.




Correctors increase the cellular processing and delivery of CFTR proteins to the cell surface.


Combination therapies

There is increasing interest in the use of combination therapies. Combining a corrector and potentiator may serve to augment CFTR function and is currently under investigation in clinical trials.5

Comparison chart

This table shows an overview of CFTR modulators that are either approved or are in development for the treatment of CF.6


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  1. Sloane PA. Curr Opin Pulm Med 2010;16:591–7
  2. O’Sullivan BP & Freedman SD. Lancet 2009;373:1891–1904
  3. Rogan MP. Chest 2011;139;1480–90
  4. Welsh MJ et al. Cystic fibrosis. In Valle D et al (Eds). OMMBID. The McGraw-Hill Companies Inc 2004. Part 21, chapter 201
  6. Adapted from Rogan MP et al. Chest 2011;139;1480–90