Small molecule
PubChem CID 16220172
Ivacaftor
Kalydeco
- Mechanism of action
- CFTR potentiator, increases chloride channel open probability
- In plain language
- Props open a faulty channel on the surface of lung and other cells (called CFTR) that's supposed to move salt and water in and out, so mucus stays thinner instead of getting thick and sticky, but only in people whose specific CFTR gene mutation is one this drug can act on.
- How it works
- Ivacaftor (Kalydeco) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. Per the FDA label, ivacaftor facilitates increased chloride transport by potentiating the channel-open probability (gating) of the CFTR protein at the cell surface. The overall level of ivacaftor-mediated CFTR chloride transport depends on the amount of CFTR protein present at the cell surface and how responsive a given mutant CFTR protein is to ivacaftor potentiation.
- Therapeutic applications
- Ivacaftor is FDA-indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor, based on clinical and/or in vitro assay data. The label requires baseline and periodic liver function monitoring (ALT/AST at baseline, every 3 months during the first year, and annually thereafter), since elevated transaminases have been reported in patients receiving ivacaftor.
- Class
- Small molecule
- Therapeutic area
- Respiratory
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