Ivacaftor (CAS 873054-44-5) 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. Chemically, Ivacaftor is N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide, featuring a quinolinone core linked via an amide bond to a di-tert-butyl-substituted phenol.
Ivacaftor is a pivotal pharmaceutical API and analytical reference standard in the treatment of cystic fibrosis, functioning as a CFTR potentiator that increases the open probability of the CFTR channel at the cell surface. As a quinolinone-based amide, Ivacaftor binds to the CFTR protein and enhances chloride ion transport in epithelial cells, thereby improving mucociliary clearance and reducing the pulmonary complications associated with cystic fibrosis. Ivacaftor has a molecular weight of 392.49 g/mol and appears as a white to off-white powder. Studies in both G551D- and F508del-CFTR expressing cells have shown that Ivacaftor combined with forskolin significantly increases CFTR-mediated chloride secretion. Ivacaftor is supplied with detailed characterization data compliant with regulatory guidelines and serves as both the API for commercial Kalydeco® production and a reference standard for analytical method development and quality control applications.
Product Parameters
Parameter
Specification
Product Name
Ivacaftor
CAS Number
873054-44-5
Molecular Formula
C₂₄H₂₈N₂O₃
Molecular Weight
392.49 g/mol
Appearance
White to light brown powder
Melting Point
212–215 °C
Boiling Point
550.4 ± 50.0 °C (Predicted)
Density
1.187
Storage Condition
Refrigerator
Mechanism Of Action
Ivacaftor promotes chloride ion transport by enhancing the channel opening probability (or gating) of the FTR protein located on the epithelial cell surface. The overall level of ivacaftor-mediated chloride ion transport depends on the quantity of CFTR proteins present on the cell surface and the extent to which specific mutant CFTR proteins respond to ivacaftor-enhanced effects.
Bioactivity
Ivacaftor (VX-770) is a selective enhancer of CFTR that specifically targets G551D-CFTR and F508del-CFTR, with EC50 values of 100 nM and 25 nM, respectively, in Fisher rat thyroid cells.
Adverse effects
The adverse reactions of evacatide primarily include headache, nausea, diarrhea, and rash, which are typically mild and transient. However, some patients may experience more severe adverse effects such as abnormal liver function or dyspnea.
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