General
Preferred name
LUMACAFTOR
Synonyms
VX-809 ()
VRT 826809 ()
VX-809 (Lumacaftor) ()
Lumacaftor (VX-809) ()
Lumacaftor component of orkambi ()
VX809 ()
VRT-826809 ()
VRT826809 ()
VX 809 ()
P&D ID
PD008912
CAS
936727-05-8
1160221-26-0
Tags
available
drug
Approved by
FDA
First approval
2015
Drug indication
Liver disease
Cystic fibrosis
Drug Status
approved
Max Phase
4.0
Probe control
Probe control not defined
Orthogonal probes
0
No orthogonal probes found
Similar probes
0
No structurally similar probes found
Structure
P&D probe-likeness score
Structure formats
[[ format ]]
[[ compound[format === 'MOL' ? 'molblock' : format.toLowerCase()] ]]
Description
(extracted from source data)
PHARMACODYNAMICS
Results from clinical trials indicated that treatment with Orkambi (lumacaftor/ [DB08820]) results in improved lung function, reduced chance of experiencing a pulmonary exacerbation, reduced sweat chloride, increased weight gain, and improvements in CF symptoms and quality of life [FDA Label].; ; Orkambi was not found to increase the QTc interval to any clinically relevant extent [FDA Label].
DESCRIPTION
Lumacaftor is termed a cystic fibrosis transmembrane conductance regulator (CFTR) corrector, acting to reduce mis-folding of the defective F508del mutant CFTR protein, in comparison to which is termed a CFTR potentiator drug. The F508del mutation is the most common cause of cystic fibrosis and accounts for approximately 60% of CF patients in Europe. Cryo-electron microscopy of CFTR-lumacaftor complexes provided insight into how the correctors operate at the molecular level, with the findings published in early 2022 by Fiedorczuk and Chen .
(GtoPdb)
DESCRIPTION
Lumacaftor (VX-809; VRT 826809) is a CFTR modulator that corrects the folding and trafficking of CFTR protein.
PRICE
57
DESCRIPTION
Lumacaftor is a protein chaperone. It is used for the treatment of cystic fibrosis. It is an allosteric modulator of cystic fibrosis transmembrane conductance regulator.
(Enamine Bioactive Compounds)
DESCRIPTION
Lumacaftor (VRT 826809) is a CFTR modulator that corrects the folding and trafficking of CFTR protein. It enhances F508del-CFTR protein maturation in FRT cells (EC50: 100 nM).
(TargetMol Bioactive Compound Library)
[[ p.pathway_name ]]
[[ compound.targets[tid].gene_name ]]
Compound Sets
20
AdooQ Bioactive Compound Library
Axon Medchem Screening Library
Cayman Chemical Bioactives
ChEMBL Approved Drugs
ChEMBL Drugs
Drug Repurposing Hub
DrugBank
DrugBank Approved Drugs
DrugCentral
DrugCentral Approved Drugs
DrugMAP
Enamine Bioactive Compounds
Guide to Pharmacology
Mcule NIBR MoA Box Subset
MedChem Express Bioactive Compound Library
NCATS Inxight Approved Drugs
Novartis Chemogenetic Library (NIBR MoA Box)
ReFrame library
Selleckchem Bioactive Compound Library
TargetMol Bioactive Compound Library
[[ a.name ]]
[[ ligand_id ]]
free of charge
External IDs
37
Molecular Weight
452.12
Hydrogen Bond Acceptors
5
Hydrogen Bond Donors
2
Rotatable Bonds
5
Ring Count
5
Aromatic Ring Count
3
cLogP
4.75
TPSA
97.75
Fraction CSP3
0.21
Chiral centers
0.0
Largest ring
6.0
QED
0.58
Structural alerts
0
No structural alerts detected
Related compounds
Custom attributes
(extracted from source data)
MOA
CFTR
Specific stabilization of CFTR MSD1, specifically TMD1.
CFTR channel agonist
Member status
member
Indication
cystic fibrosis
Pathway
Autophagy
Membrane Transporter/Ion Channel
Target
CFTR corrector
F508del-CFTR
Source data
