VX-770

Ivacaftor is a potent and orally bioavailable CFTR potentiator, targeting G551D-CFTR and F508del-CFTR with EC50s of 100 nM and 25 nM, respectively.
Solid
Ivacaftor is an aromatic amide obtained by formal condensation of the carboxy group of 4-oxo-1,4-dihydroquinoline-3-carboxylic acid with the amino group of 5-amino-2,4-di-tert-butylphenol. Used for the treatment of cystic fibrosis. It has a role as a CFTR potentiator and an orphan drug. It is a quinolone, a member of phenols, an aromatic amide and a monocarboxylic acid amide.|Ivacaftor (also known as Kalydeco or VX-770) is a drug used for the management of Cystic Fibrosis (CF). It is manufactured and distributed by Vertex Pharmaceuticals. It was approved by the Food and Drug Administration on January 31, 2012, and by Health Canada in late 2012. Ivacaftor is administered as a monotherapy and also administered in combination with other drugs for the management of CF. Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, an ion channel involved in the transport of chloride and sodium ions across cell membranes. CFTR is active in epithelial cells of organs such as of the lungs, pancreas, liver, digestive system, and reproductive tract. Alterations in the CFTR gene result in altered production, misfolding, or function of the protein and consequently abnormal fluid and ion transport across cell membranes. As a result, CF patients produce thick, sticky mucus that clogs the ducts of organs where it is produced making patients more susceptible to complications such as infections, lung damage, pancreatic insufficiency, and malnutrition. Prior to the development of ivacaftor, management of CF primarily involved therapies for the control of infections, nutritional support, clearance of mucus, and management of symptoms rather than improvements in the underlying disease process or lung function (FEV1). Notably, ivacaftor was the first medication approved for the management of the underlying causes of CF (abnormalities in CFTR protein function) rather than control of symptoms.|Ivacaftor is a Cystic Fibrosis Transmembrane Conductance Regulator Potentiator. The mechanism of action of ivacaftor is as a Chloride Channel Activation Potentiator, and Cytochrome P450 3A4 Inhibitor, and Cytochrome P450 2C9 Inhibitor, and P-Glycoprotein Inhibitor, and Cytochrome P450 3A Inhibitor.|Ivacaftor, lumacaftor, tezacaftor and elexacaftor are orally available potentiators or correctors of the cystic fibrosis transmembrane conductance regulator (CFTR) that are used to treat patients with cystic fibrosis with specific mutations of the CFTR. Ivacaftor alone or in combination with lumacaftor or tezacaftor has been associated with low rate of transient serum enzyme elevations during treatment, but not with clinically apparent acute liver injury with jaundice, while the addition of elexacaftor to ivacaftor and tezacaftor has been implicated in a higher rate of serum enzyme elevations (up to 10% of patients) and to rare instances of clinically apparent liver injury with jaundice.