Lumacaftor

CFTR corrector

Phase of research

FDA-/EMA-approved for Cystic Fibrosis

How it helps

Restore CFTR Function

General information

Lumacaftor is a drug developed specifically for cystic fibrosis treatment by Vertex Pharmaceuticals. It is a CFTR corrector, which helps the conformational stability of the mutated CFTR protein, prevents its misfolding and increases its presence at the cell surface. This CFTR modulator was approved by the FDA in 2015 in combination with the CFTR potentiator ivacaftor as Orkambi®. Orkambi® is indicated for patients with two copies of F508del. Lumacaftor has no medical use on its own.  In relation to cystic fibrosis, our AIM tool found the data that lumacaftor can help correct multiple CFTR mutations, usually in combination with other substances.

Lumacaftor on PubChem
Lumacaftor on DrugBank
Lumacaftor on Wikipedia

Synonyms

VX-809

Marketed as

Dietary sources

N/A

Structure not available

C24H18F2N2O5

Drug-Mutation Relation

Treats

Mutation Number of sources Average impact factor
F508del
10 6.12
Link Tested on Impact factor Notes
MicroRNA-145 Antagonism Reverses TGF-β Inhibition of F508del CFTR Correction in Airway Epithelia human cell cultures 16.49 In combination with ivacaftor. Better in combination with ivacaftor and a miR-145 antagonist.
Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation. yeast cell cultures 4.01 In combination with ivacaftor restores only a subpopulation of F508del-CFTR Cl− channels. 
Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis human cell cultures 2.81 Better in combination with ivacaftor and Corr-4a.
Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression Defect. human cell cultures 9.80 Better in combination with knockdown of RPL12.
Potentiation of ΔF508- and G551D-CFTR-Mediated Cl- Current by Novel Hydroxypyrazolines. human cell cultures 2.81 Better in combination with CP7q.
Simple image-based no-wash method for quantitative detection of surface expressed CFTR human cell cultures 3.80
Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis human cell cultures N/A
Pseudomonas aeruginosa Reduces VX-809 Stimulated F508del-CFTR Chloride Secretion by Airway Epithelial Cells human cell cultures 3.06 Better on its own than in combination with ivacaftor.
Discovery of novel potent ΔF508-CFTR correctors that target the nucleotide binding domain. human cell cultures 8.25 Comparable to NCI407882.
A novel triple combination of pharmacological chaperones improves F508del-CFTR correction. human cell cultures 4.01 Better in combination with RDR1. Most effective in combination with RDR1 and MCG1516A.
E1418X
2 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with tezacaftor.
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with tezacaftor. Better in combination with ivacaftor.
A561E/A561E
1 0.00
Link Tested on Impact factor Notes
Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis human cell cultures N/A
S1435Gfs14X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with tezacaftor.
E831X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with tezacaftor. Even better when followed by treatment with ivacaftor.
L997F/R258G
1 4.12
Link Tested on Impact factor Notes
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. human cell cultures 4.12
G1244E/F508del
1 32.62
Link Tested on Impact factor Notes
Thymosin α1 represents a potential potent single molecule-based therapy for cystic fibrosis human cell cultures 32.62 In combination with ivacaftor.
Y1092X
1 0.00
Link Tested on Impact factor Notes
Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis human cell cultures N/A
p.Ile1234_Arg1239del
1 7.33
Link Tested on Impact factor Notes
Genetic, cell biological, and clinical interrogation of the CFTR mutation c.3700 A>G (p.Ile1234Val) informs strategies for future medical intervention. humans 7.33
S1455X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with tezacaftor.
LXX8
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 In combination with G418.
E1418Rfs14X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with tezacaftor. Followed by treatment with ivacaftor.
F508del/D1152H
1 4.12
Link Tested on Impact factor Notes
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. human cell cultures 4.12
R347P
1 3.45
Link Tested on Impact factor Notes
Folding–function relationship of the most common cystic fibrosis–causing CFTR conductance mutants human cell cultures 3.45
F508del/1717-1G->A
1 4.12
Link Tested on Impact factor Notes
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. human cell cultures 4.12
F508del/394delTT
1 4.12
Link Tested on Impact factor Notes
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. human cell cultures 4.12
F508del/F508del
10 23.85
Link Tested on Impact factor Notes
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study. humans 21.47 In combination with ivacaftor as Orkambi®.
Vertex Lumacaftor (VX-809) and Ivacaftor in Children with CF aged 6 to 11 years and have two copies of the delF508 CFTR mutation (Vertex VX13-809-011b) humans 15.24 In combination with ivacaftor as Orkambi®.
Phase 3 study of lumacaftor/ivacaftor in children with CF (Vertex VX15-809-115) humans N/A In combination with ivacaftor as Orkambi®.
Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. humans 21.47 In combination with ivacaftor as Orkambi®.
Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. humans 4.29 In combination with ivacaftor as Orkambi®.
A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. humans 9.63 In combination with ivacaftor as Orkambi®.
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. humans 59.56 In combination with ivacaftor as Orkambi®.
Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup humans 19.29 In combination with ivacaftor.
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. human cell cultures 4.12
Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR humans 59.56 Tested in combination with ivacaftor.
Q1476X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Alone or in combination with tezacaftor.

Does not treat

Mutation Number of sources Average impact factor
F508del/711+1G->T
1 4.12
Link Tested on Impact factor Notes
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. human cell cultures 4.12
F508del/E730X
1 3.45
Link Tested on Impact factor Notes
Folding–function relationship of the most common cystic fibrosis–causing CFTR conductance mutants human cell cultures 3.45
F508del/N1303K
1 4.12
Link Tested on Impact factor Notes
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators. human cell cultures 4.12
N1303K
1 0.00
Link Tested on Impact factor Notes
Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis human cell cultures N/A
Q1390X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Not even in combination with tezacaftor.
Q1412X
1 5.22
Link Tested on Impact factor Notes
Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis human cell cultures 5.22 Not even in combination with tezacaftor.