Ivacaftor
This CFTR potentiator is used to treat cystic fibrosis in patients with specific mutations (such as G551D) that cause the CFTR protein to be present but dysfunctional. It works by binding to the abnormal CFTR protein and keeping its chloride channel open for longer, improving airway hydration and mucus clearance. It was the first therapy to target the underlying genetic defect in cystic fibrosis.
Khối lượng phân tử
392,5000 g/mol
LogP
5,60
TPSA
78,40 Ų
Lipinski RO5
Đạt
Lĩnh vực điều trị
Pharmacokinetics (PK)
Pharmacodynamics (PD)
Cấu trúc 2D
Cite this structure
Embed this structure
SMILES
CC(C)(C)c1cc(C(C)(C)C)c(NC(=O)c2c[nH]c3ccccc3c2=O)cc1O
InChI
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)
Molecular Formula
C24H28N2O3
HBD / HBA
3 / 4
Liên kết có thể quay
4
Nguyên tử nặng
29
No targets recorded
Target interaction data is not yet available for this drug.
No interactions recorded
Drug interaction data is not yet available for this compound.
No side effects recorded
Side effect data is not yet available for this drug.
Câu hỏi thường gặp
This CFTR potentiator is used to treat cystic fibrosis in patients with specific mutations (such as G551D) that cause the CFTR protein to be present but dysfunctional. It works by binding to the abnormal CFTR protein and keeping its chloride channel open for longer, improving airway hydration and mucus clearance. It was the first therapy to target the underlying genetic defect in cystic fibrosis.
Yes, Ivacaftor is an approved drug. It has reached clinical phase 4. It is classified as a Small molecule.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL2010601. Open-access bioactivity database.
- PubChem — National Center for Biotechnology Information (NCBI). CID 16220172. Chemical information database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-04.
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