GtoPdb is requesting financial support from commercial users. Please see our sustainability page for more information.
Synonyms: compound 48 [PMID: 25441013] | 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 [5,7], the net effect of which is increased chloride channel opening. It is an orally bioavailable small molecule that is effective in cystic fibrosis patients carrying a number of mutant CFTR forms, including F508del (a processing mutation) and CFTRG551D (a gating mutation).
![]() 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 |
|
Selectivity at ion channels | ||||||||||||||||||||||||||||||||||
Key to terms and symbols | Click column headers to sort | |||||||||||||||||||||||||||||||||
|
Targets where the ligand is described in the comment field | |
Target | Comment |
copper metabolism domain containing 1 | COMMD1 is a multi-function protein that is involved in a range of biological processes, including copper homeostasis, regulation of protein localisation, regulation of NF-κB signaling, and it is proposed to be a component of the Cul2-RING ubiquitin ligase complex. A peptide that binds directly to COMMD1 (CIGB-552) has reported anti-tumour activity [3-4], and is also proposed to synergise with cystic fibrosis triple therapy Kaftrio (elexacaftor/tezacaftor/ivacaftor) to futher improve chloride transport via the cystic fibrosis CFTR-F508del protein variant. CIGB-552 is suggested to disrupt COMMD1-associated degradation of the misfolded mutated CFTR [1,6] and to enhance its localisation to the plasma membrane. |