| Drug Name |
Ivacaftor |
| Drug ID |
BADD_D01220 |
| Description |
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[L768], and by Health Canada in late 2012.[L6841] Ivacaftor is administered as a monotherapy and also administered in combination with other drugs for the management of CF.[L6814,L6979,L6847]
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.[A20298, A20299] 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.[A20302]
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.[A20297] |
| Indications and Usage |
When used as monotherapy as the product Kalydeco, ivacaftor is indicated for the management of CF in patients age 2 years and older who have a mutation in the CFTR gene that is responsive to ivacaftor potentiation. Ivacaftor received expanded approval in May 2017 for the following 33 CFTR mutations: E56K, P67L, R74W, D110E, D110H, R117C, R117H, G178R, E193K, L206W, R347H, R352Q, A455E, S549N, S549R, G551D, G551S, D579G, S945L, S977F, F1052V, K1060T, A1067T, G1069R, R1070Q, R1070W, F1074L, D1152H, G1244E, S1251N, S1255P, D1270N, and G1349D.[L768,L6979]
When used in combination with the drug [lumacaftor] as the product Orkambi, ivacaftor is indicated for the management of CF patients age 6 years and older who are shown to be homozygous for the F508del mutation in the CFTR gene.
When used in combination with [tezacaftor] in the product Symdeko, it is used to manage CF in patients 12 years and older who have at least one mutation in the CFTR gene or patients aged 12 or older who are shown to be homozygous for the F508del mutation.[L6814]
When used in combination with tezacaftor and [elexacaftor] in the product Trikafta, it is indicated for the treatment of cystic fibrosis in patients 12 years of age and older who have at least one _F508del_ mutation in the CFTR gene.[L9395] |
| Marketing Status |
approved |
| ATC Code |
R07AX02 |
| DrugBank ID |
DB08820
|
| KEGG ID |
D09916
|
| MeSH ID |
C545203
|
| PubChem ID |
16220172
|
| TTD Drug ID |
D0W7WC
|
| NDC Product Code |
65129-1450; 51167-600; 59651-588; 11014-0564; 24538-770; 51167-400; 14501-0019; 51167-770; 66039-900; 51167-200; 51167-300; 67651-0263; 51167-785; 43798-005 |
| UNII |
1Y740ILL1Z
|
| Synonyms |
ivacaftor | N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide | N-(2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide | 3-quinolinecarboxamide, N-(2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl)-1,4-dihydro-4-oxo- | VX-770 | Kalydeco |
|
