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Synonyms: compound 48 [PMID: 25441013] | Kalydeco® | VX-770
ivacaftor is an approved drug
Compound class:
Synthetic organic
Comment: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator [6,8], it increases channel open probability. It is an orally bioavailable small molecule that is effective in cystic fibrosis patients carrying carrying gating CFTR variants (e.g. G551D) and in combination therapies with corrector(s) for patients carrying other variants, including F508del.
![]() Ligand Activity Visualisation ChartsThese are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts. ✖![]() View more information in the IUPHAR Pharmacology Education Project: ivacaftor |
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References |
1. Csanády L, Töröcsik B. (2019)
Cystic fibrosis drug ivacaftor stimulates CFTR channels at picomolar concentrations. Elife, 8. [PMID:31205003] |
2. Drévillon L, Tanguy G, Hinzpeter A, Arous N, de Becdelièvre A, Aissat A, Tarze A, Goossens M, Fanen P. (2011)
COMMD1-mediated ubiquitination regulates CFTR trafficking. PLoS One, 6 (3): e18334. [PMID:21483833] |
3. 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] |
4. Fernández Massó JR, Oliva Argüelles B, Tejeda Y, Astrada S, Garay H, Reyes O, Delgado-Roche L, Bollati-Fogolín M, Vallespí MG. (2013)
The Antitumor Peptide CIGB-552 Increases COMMD1 and Inhibits Growth of Human Lung Cancer Cells. J Amino Acids, 2013: 251398. [PMID:23401744] |
5. Gomez Rodriguez Y, Oliva Arguelles B, Riera-Romo M, Fernandez-De-Cossio J, Garay HE, Fernandez Masso J, Guerra Vallespi M. (2022)
Synergic effect of anticancer peptide CIGB-552 and Cisplatin in lung cancer models. Mol Biol Rep, 49 (4): 3197-3212. [PMID:35094208] |
6. Hadida S, Van Goor F, Zhou J, Arumugam V, McCartney J, Hazlewood A, Decker C, Negulescu P, Grootenhuis PD. (2014)
Discovery of N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, ivacaftor), a potent and orally bioavailable CFTR potentiator. J Med Chem, 57 (23): 9776-95. [PMID:25441013] |
7. Tanguy G, Drévillon L, Arous N, Hasnain A, Hinzpeter A, Fritsch J, Goossens M, Fanen P. (2008)
CSN5 binds to misfolded CFTR and promotes its degradation. Biochim Biophys Acta, 1783 (6): 1189-99. [PMID:18267124] |
8. 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] |