Synonyms: Kalydeco® | VX-770
ivacaftor is an approved drug (FDA and EMA (2012))
Compound class:
Synthetic organic
Comment: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, the net effect of which is increased chloride channel opening.
![]() View more information in the IUPHAR Pharmacology Education Project: ivacaftor |
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References |
1. Eckford PD, Li C, Ramjeesingh M, Bear CE. (2012)
Cystic fibrosis transmembrane conductance regulator (CFTR) potentiator VX-770 (ivacaftor) opens the defective channel gate of mutant CFTR in a phosphorylation-dependent but ATP-independent manner. J Biol Chem, 287 (44): 36639-49. [PMID:22942289] |
2. Levring J, Terry DS, Kilic Z, Fitzgerald G, Blanchard SC, Chen J. (2023)
CFTR function, pathology and pharmacology at single-molecule resolution. Nature, 616 (7957): 606-614. [PMID:36949202] |
3. Van Goor F, Hadida S, Grootenhuis PD, Burton B, Cao D, Neuberger T, Turnbull A, Singh A, Joubran J, Hazlewood A et al.. (2009)
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci USA, 106 (44): 18825-30. [PMID:19846789] |