Lumacaftor (USAN, codenamed VX-809) is an experimental drug for the treatment of cystic fibrosis being developed by Vertex Pharmaceuticals. The drug is designed to be effective in patients that have the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), the defective protein that causes the disease. F508del, meaning that the amino acid phenylalanine in position 508 is missing, is found in about 60% of cystic fibrosis patients in Europe, and in about 90% of persons with some mutation in the CFTR gene.
Lumacaftor acts on F508 CFTR during protein folding and suppresses misfolding.
Results from a Phase II clinical trial indicate that patients with the most common form of genetic mutation causing cystic fibrosishomozygous F508delhad a mean increase of 7.4% in lung function (FEV1) on a combination of lumacaftor and ivacaftor.However these results did not show a significant improvement in lung function.
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