Ivacaftor
Encyclopedia
Ivacaftor is an experimental drug candidate for the treatment of cystic fibrosis
, currently under development by Vertex Pharmaceuticals
and the Cystic Fibrosis Foundation
. The drug is designed to be effective in patients that have the G551D mutation in the defective protein that causes the disease. G551D, meaning that the amino acid glycine
in position 551 is substituted with aspartic acid
, is only found in a small percentage of cystic fibrosis patients. The drug is currently in Phase 3 clinical trials and has showed promising results for this subset of cystic fibrosis patients.
Cystic fibrosis is caused by a defect in a protein, cystic fibrosis transmembrane conductance regulator
, which regulates fluid flow within cells and affects the components of sweat, digestive fluids, and mucus. VX-770 potentiates the effect of the defective protein to restore a more normal function.
Cystic fibrosis
Cystic fibrosis is a recessive genetic disease affecting most critically the lungs, and also the pancreas, liver, and intestine...
, currently under development by Vertex Pharmaceuticals
Vertex Pharmaceuticals
Vertex Pharmaceuticals is a biotechnology company with activities spanning the length of the pharmaceutical product pipeline, from target identification through to clinical trials and marketing. Most of its activity has been in collaboration with much larger pharmaceutical firms, though some of its...
and the Cystic Fibrosis Foundation
Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is a non-profit organization in the United States established to provide the means to cure and control cystic fibrosis . The Foundation provides information about cystic fibrosis and finances CF research that aims to improve the quality of life for people with the...
. The drug is designed to be effective in patients that have the G551D mutation in the defective protein that causes the disease. G551D, meaning that the amino acid glycine
Glycine
Glycine is an organic compound with the formula NH2CH2COOH. Having a hydrogen substituent as its 'side chain', glycine is the smallest of the 20 amino acids commonly found in proteins. Its codons are GGU, GGC, GGA, GGG cf. the genetic code.Glycine is a colourless, sweet-tasting crystalline solid...
in position 551 is substituted with aspartic acid
Aspartic acid
Aspartic acid is an α-amino acid with the chemical formula HOOCCHCH2COOH. The carboxylate anion, salt, or ester of aspartic acid is known as aspartate. The L-isomer of aspartate is one of the 20 proteinogenic amino acids, i.e., the building blocks of proteins...
, is only found in a small percentage of cystic fibrosis patients. The drug is currently in Phase 3 clinical trials and has showed promising results for this subset of cystic fibrosis patients.
Cystic fibrosis is caused by a defect in a protein, cystic fibrosis transmembrane conductance regulator
Cystic fibrosis transmembrane conductance regulator
Cystic fibrosis transmembrane conductance regulator is a protein that in humans is encoded by the CFTR gene.CFTR is a ABC transporter-class ion channel that transports chloride and thiocyanate ions across epithelial cell membranes...
, which regulates fluid flow within cells and affects the components of sweat, digestive fluids, and mucus. VX-770 potentiates the effect of the defective protein to restore a more normal function.
External links
- FAQs About VX-770 from the Cystic Fibrosis Foundation