lumacaftor

Lumacaftor is a small molecule drug classified as a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It is primarily used in combination with ivacaftor for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the CFTR gene. Lumacaftor acts by improving the conformational stability of F508del-mutated CFTR proteins, increasing their processing and trafficking to the cell surface, which results in enhanced chloride ion transport across epithelial cell membranes. This mechanism helps reduce thick mucus buildup and improves lung function and other symptoms associated with CF[4][9]. Lumacaftor was developed by Vertex Pharmaceuticals.

Brand names

LYRIC-HF

Other names

LYRIC-HF