Skip to main content
Free research Free research Free compound review with research home and workspace preview
Quick search ChEMBL 36
Compound Detail

CHEMBL2010601

IVACAFTOR
💊 Approved Drug
Enter ChEMBL ID:
Free Research Context This compound will appear in recent viewed items on the research home for this browser.
Research home View demo workspace
💊 Approved Drug
CC(C)(C)c1cc(C(C)(C)C)c(NC(=O)c2c[nH]c3ccccc3c2=O)cc1O
ChEMBL ↗ Properties Compare Identifier

Basic Information

ChEMBL ID CHEMBL2010601
Name IVACAFTOR
Phase Approved
Structure Type MOL
InChI Key PURKAOJPTOLRMP-UHFFFAOYSA-N
Therapeutic ✓ Yes

Known Names

Ivacaftor Ivacaftor component of orkambi Ivacaftor component of symkevi Ivacaftor component of trikafta Kalydeco VX-770
4
Targets
22
Activities
3
Assay Types
12
PK Parameters
20
Publications
6
Known Names
7
Indications
1
Mechanisms

Molecular Properties

392.5
MW (Da)
5.08
ALogP
3
HBA
3
HBD
82.2
PSA (Ų)
0.57
QED
1
Ro5 Violations
2
Rot. Bonds

Drug Information

Molecule Type Small molecule
First Approval 2012
Availability Prescription
Chirality Achiral

Routes of Administration

Oral

Flags

Natural Product First in Class
USAN Stem -caftor
USAN Year 2010

Extended Properties

Molecular Formula C24H28N2O3
MW 392.50
Aromatic Rings 3
Heavy Atoms 29
NP-Likeness -0.22

Mechanism of Action

Mechanism Action Type Target Direct Efficacy
Cystic fibrosis transmembrane conductance regulator positive modulator POSITIVE MODULATOR Cystic fibrosis transmembrane conductance regulator ✓ ✓

Indications

Cystic Fibrosis Respiratory Tract Diseases Bronchitis, Chronic Ciliary Motility Disorders Pulmonary Disease, Chronic Obstructive Liver Diseases Sinusitis

ATC Classification

R07AX02
ivacaftor
Level 1: RESPIRATORY SYSTEM
Level 2: OTHER RESPIRATORY SYSTEM PRODUCTS

Activity Summary

EC50 19 meas. best 8.52
IC50 2 meas. best 5.4
Kd 1 meas. best 4.4

Target Activity Profile

Target Type Best pChEMBL Mean pChEMBL Best (nM) # Data
Cystic fibrosis transmembrane conductance regulator
CHEMBL4051
 EC50 8.52 7.3539 3.0 18
Unchecked
CHEMBL612545
 EC50 6.63 6.63 236.0 1
NRK-49F
CHEMBL614203
 IC50 5.4 5.4 4000.0 1
SARS-CoV-2
CHEMBL4303835
 IC50 5.18 5.18 6570.0 1
Unchecked
CHEMBL612545
 Kd 4.4 4.4 40000.0 1

Pharmacokinetic Data

Parameter Value Units Species
AUC 4800.0 ng.hr.mL-1 Rattus norvegicus
AUC 13500.0 ng.hr.mL-1 Canis lupus familiaris
CL 20.0 mL.min-1.kg-1 Mus musculus
CL 0.7 mL.min-1.kg-1 Canis lupus familiaris
CL 7.4 mL.min-1.kg-1 Macaca fascicularis
F 55.0 % Rattus norvegicus
F 43.0 % Canis lupus familiaris
F 55.0 % Rattus norvegicus
T1/2 1.3 hr Mus musculus
T1/2 13.0 hr Canis lupus familiaris
T1/2 6.7 hr Macaca fascicularis
T1/2 9.5 hr Rattus norvegicus

Activity Data Samples

Top measurements by pChEMBL value

Target Type Rel. Value Units pChEMBL Assay PubMed
Cystic fibrosis transmembrane conductance regulator EC50 = 3.0 nM 8.52 Corrector activity at CFTR mutant (unknown origin) by phenotypic screening assay 25384240
Cystic fibrosis transmembrane conductance regulator EC50 = 3.0 nM 8.52 Potentiation of human CFTR F508del mutant expressed in mouse NIH-3T3 cells after... 25441013
Cystic fibrosis transmembrane conductance regulator EC50 = 11.7 nM 7.93 Potentiator activity at CFTR F508del mutant (unknown origin) expressed in CFBE41... 32078860
Cystic fibrosis transmembrane conductance regulator EC50 = 12.59 nM 7.9 Corrector activity at CFTR F508del mutant in forskolin-stimulated human bronchia... 38527911
Cystic fibrosis transmembrane conductance regulator EC50 = 13.0 nM 7.89 Corrector activity at CFTR F508del mutant in forskolin-stimulated human bronchia... 38527911
Cystic fibrosis transmembrane conductance regulator EC50 = 22.0 nM 7.66 Potentiation of human CFTR F508del/G551D mutant in human bronchial epithelial ce... 25441013
Cystic fibrosis transmembrane conductance regulator EC50 = 25.0 nM 7.6 Inhibition of CFTR F508 deletion mutant (unknown origin) 36155354
Cystic fibrosis transmembrane conductance regulator EC50 = 34.0 nM 7.47 Potentiation of wild type CFTR (unknown origin) expressed in HBE cells assessed ... 34028270
Cystic fibrosis transmembrane conductance regulator EC50 = 49.0 nM 7.31 Potentiation of CFTR F508del mutant (unknown origin) expressed in FRT cells asse... 34028270
Cystic fibrosis transmembrane conductance regulator EC50 = 49.0 nM 7.31 Potentiation of CFTR F508del mutant (unknown origin) 38194776
Cystic fibrosis transmembrane conductance regulator EC50 = 68.0 nM 7.17 Potentiation of CFTR F508 deletion mutant (unknown origin) expressed in HBE cell... 34028270
Cystic fibrosis transmembrane conductance regulator EC50 = 70.0 nM 7.16 Potentiation of wild-type CFTR (unknown origin) expressed in FRT cells assessed ... 34028270
Cystic fibrosis transmembrane conductance regulator EC50 = 100.0 nM 7.0 Inhibition of CFTR G551D mutant (unknown origin) 36155354
Cystic fibrosis transmembrane conductance regulator EC50 = 126.0 nM 6.9 Potentiation of CFTR F508del mutant (unknown origin) expressed in human CFBE41o ... 29148763
Cystic fibrosis transmembrane conductance regulator EC50 = 131.0 nM 6.88 Potentiation of CFTR G551D mutant (unknown origin) expressed in FRT cells assess... 34028270
Cystic fibrosis transmembrane conductance regulator EC50 = 236.0 nM 6.63 Potentiation of human CFTR F508del mutant in human bronchial epithelial cells by... 25441013
Cystic fibrosis transmembrane conductance regulator EC50 = 236.0 nM 6.63 Corrector activity at human bronchial epithelium CFTR F508del and G551D mutant a... 25384240
Unchecked EC50 = 236.0 nM 6.63 Cytotoxicity against human HBE cells assessed as inhibition of cell growth 36155354
Cystic fibrosis transmembrane conductance regulator EC50 = 1300.0 nM 5.89 Potentiation of CFTR G551D mutant (unknown origin) expressed in HEK293 cells inc... 29148763
NRK-49F IC50 = 4000.0 nM 5.4 Inhibition of TGF-beta1-induced total collagen accumulation in rat NRK-49F cells 25467157

Literature References

PubMed DOI Target Context # Activities
- 10.6019/CHEMBL5724801 HEK-293T 320
- 10.6019/CHEMBL5724801 U2OS 320
- 10.6019/CHEMBL5724801 Fibroblast 320
32078860 10.1016/j.ejmech.2020.112116 Cystic fibrosis transmembrane conductance regulator 11
34028270 10.1021/acs.jmedchem.1c00343 Cystic fibrosis transmembrane conductance regulator 10
- 10.6019/CHEMBL5303304 HEK-293T 9
- 10.6019/CHEMBL4495565 SARS-CoV-2 6
- 10.6019/CHEMBL5303304 U2OS 5
25441013 10.1021/jm5012808 Canis familiaris 5
- 10.6019/CHEMBL5303304 Fibroblast 5
- 10.6019/CHEMBL4651402 SARS-CoV-2 4
29148763 10.1021/acs.jmedchem.7b01288 Cystic fibrosis transmembrane conductance regulator 4
37468498 10.1038/s41467-023-40064-9 Alpha-2A adrenergic receptor 3
37468498 10.1038/s41467-023-40064-9 5-hydroxytryptamine receptor 2B 3
25467157 10.1016/j.bmcl.2014.10.071 Mus musculus 3
25441013 10.1021/jm5012808 Cystic fibrosis transmembrane conductance regulator 3
25441013 10.1021/jm5012808 Mus musculus 3
25441013 10.1021/jm5012808 Cynomolgus monkey 3
32946228 10.1021/acs.jmedchem.0c01050 Cystic fibrosis transmembrane conductance regulator 3
- 10.6019/CHEMBL4495564 Replicase polyprotein 1ab 3

Data from ChEMBL 36 via Core Engine