WARNING: This product is for research use only, not for human or veterinary use.
MedKoo CAT#: 507210
Description: Ivacaftor, also known as VX-770, is a drug used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, who account for 4–5% cases of cystic fibrosis, and is included in a combination drug, lumacaftor/ivacaftor, which is used to treat people with cystic fibrosis who have the F508del mutation in CFTR. Ivacaftor is a "potentiator" of CFTR, meaning it increases the probability that the defective channel will be open and allow chloride ions pass through the channel pore.
MedKoo Cat#: 507210
Chemical Formula: C24H28N2O3
Exact Mass: 392.20999
Molecular Weight: 392.49072
Elemental Analysis: C, 73.44; H, 7.19; N, 7.14; O, 12.23
Synonym: VX770; VX 770; VX-770; Ivacaftor; brand name: KALYDECO.
IUPAC/Chemical Name: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide
InChi Key: PURKAOJPTOLRMP-UHFFFAOYSA-N
InChi Code: InChI=1S/C24H28N2O3/c1-23(2,3)16-11-17(24(4,5)6)20(27)12-19(16)26-22(29)15-13-25-18-10-8-7-9-14(18)21(15)28/h7-13,27H,1-6H3,(H,25,28)(H,26,29)
SMILES Code: O=C(C1=CNC2=C(C=CC=C2)C1=O)NC3=CC(O)=C(C(C)(C)C)C=C3C(C)(C)C
KALYDECO (ivacaftor, VX-770) is Vertex's lead medicine in development for the treatment of people with cystic fibrosis. Known as a CFTR potentiator, this oral medicine in development aims to help CFTR protein function more normally once it reaches the cell surface, which is believed to help hydrate and clear mucus from the airways. Vertex retains worldwide rights to develop and commercialize KALYDECO (kuh-LYE-deh-koh). The brand name KALYDECO has been approved by the EMA and provisionally approved by the FDA, but KALYDECO has not been granted marketing authorization or approval from any regulatory authority.
About Cystic Fibrosis : CF is a life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 38 years. According to the 2010 Cystic Fibrosis Foundation Patient Registry Annual Data Report, approximately 4 percent of the total CF patient population in the United States have at least one copy of the G551D mutation. The most common form of CF is caused by the F508del mutation, which is present in nearly 90 percent of people with the disease.
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