General
Preferred name
LUMACAFTOR
Synonyms
VRT 826809 ()
VX-809 ()
VX-809 (Lumacaftor) ()
Lumacaftor (VX-809) ()
VRT-826809 ()
VX 809 ()
P&D ID
PD008912
CAS
936727-05-8
1160221-26-0
Tags
available
drug
Approved by
FDA
First approval
2015
Drug Status
approved
Drug indication
Cystic fibrosis
Max Phase
Phase 4
Structure
Probe scores
P&D probe-likeness score
[[ v.score ]]%
Structure formats
[[ format ]]
[[ compound[format === 'MOL' ? 'molblock' : format.toLowerCase()] ]]
Description
(extracted from source data)
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)
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].
Compound Sets
19
AdooQ Bioactive Compound Library
A10986
Axon Medchem Screening Library
3234
Cayman Chemical Bioactives
22196
ChEMBL Approved Drugs
CHEMBL2103870
ChEMBL Drugs
CHEMBL2103870
Drug Repurposing Hub
DrugBank
DB09280
DrugBank Approved Drugs
DB09280
DrugCentral
5010
DrugCentral Approved Drugs
5010
DrugMAP
DMCLWDJ
Guide to Pharmacology
7481
Mcule NIBR MoA Box Subset
MCULE-7817076462
MedChem Express Bioactive Compound Library
HY-13262
NCATS Inxight Approved Drugs
EGP8L81APK
Novartis Chemogenetic Library (NIBR MoA Box)
ReFrame library
Selleckchem Bioactive Compound Library
VX-809
TargetMol Bioactive Compound Library
T2595
External IDs
34
Properties
(calculated by RDKit )
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)
Pathway
Membrane Transporter/Ion Channel
Autophagy
Target
F508del-CFTR
CFTR corrector
MOA
CFTR
Specific stabilization of CFTR MSD1, specifically TMD1.
CFTR channel agonist
Member status
member
Indication
cystic fibrosis
Source data