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

Orkambi - prescribing information

Dietary sources

N/A

C₂₄H₁₈F₂N₂O₅

Drug-Mutation Relation

results for 3737delA / Y1092X

See all data on Lumacaftor

Treats

Mutation	Link	Tested on	Impact factor	Notes
Y1092X	Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis	human cell cultures	N/A

Does not treat

Mutation	Link	Tested on	Impact factor	Notes