Lumacaftor (VX-809) is a pharmaceutical drug that acts as a chaperone during

protein folding Protein folding is the physical process by which a protein chain is translated to its native three-dimensional structure, typically a "folded" conformation by which the protein becomes biologically functional. Via an expeditious and reproduc ...

and increases the number of CFTR proteins that are trafficked to the cell surface. It is available in a single pill with

ivacaftor Ivacaftor is a medication used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (primarily the G551D mutation), who account for 4–5% cases of cystic fibrosis. It ...

; the combination, lumacaftor/ivacaftor (brand name ''Orkambi''), is used to treat people with cystic fibrosis who are homozygous for the F508del mutation in the

cystic fibrosis transmembrane conductance regulator Cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel in vertebrates that is encoded by the ''CFTR'' gene. Geneticist Lap-Chee Tsui and his team identified the CFTR gene in 1989 as the gene linked wi ...

(CFTR) gene, the defective protein that causes the disease.Lumacaftor label
Last updated July 2015. Check index pag
here
for label updates It was developed by

Vertex Pharmaceuticals Vertex Pharmaceuticals is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headqu ...

and the combination was approved by the FDA in 2015. As of 2015, lumacaftor had no medical use on its own.

References

CYP3A4 inducers Organofluorides Carboxylic acids Cystic fibrosis Cyclopropanes Orphan drugs {{respiratory-system-drug-stub

See also

References