Tezacaftor
Tezacaftor is a CFTR corrector that binds to the misfolded CFTR protein (particularly the F508del mutant) and facilitates its proper folding and trafficking to the cell surface, where it can function as a chloride channel in cystic fibrosis patients. It is used in combination with ivacaftor (a CFTR potentiator) to treat cystic fibrosis in patients with F508del and other responsive mutations. This combinatorial approach of correcting protein folding and enhancing channel gating addresses two distinct molecular defects in CFTR dysfunction.
Peso Molecular
520,5000 g/mol
LogP
2,90
TPSA
113,00 Ų
Regra dos 5 de Lipinski
Aprovado
Pharmacokinetics (PK)
Pharmacodynamics (PD)
Estrutura 2D
Cite this structure
Embed this structure
SMILES
CC(C)(CO)c1cc2cc(NC(=O)C3(c4ccc5c(c4)OC(F)(F)O5)CC3)c(F)cc2n1C[C@@H](O)CO
InChI
InChI=1S/C26H27F3N2O6/c1-24(2,13-33)22-8-14-7-18(17(27)10-19(14)31(22)11-16(34)12-32)30-23(35)25(5-6-25)15-3-4-20-21(9-15)37-26(28,29)36-20/h3-4,7-10,16,32-34H,5-6,11-13H2,1-2H3,(H,30,35)/t16-/m1/s1
Molecular Formula
C26H27F3N2O6
HBD / HBA
4 / 9
Ligações Rotacionáveis
8
Átomos Pesados
37
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.
Perguntas frequentes
Tezacaftor is a CFTR corrector that binds to the misfolded CFTR protein (particularly the F508del mutant) and facilitates its proper folding and trafficking to the cell surface, where it can function as a chloride channel in cystic fibrosis patients. It is used in combination with ivacaftor (a CFTR potentiator) to treat cystic fibrosis in patients with F508del and other responsive mutations. This combinatorial approach of correcting protein folding and enhancing channel gating addresses two distinct molecular defects in CFTR dysfunction.
Yes, Tezacaftor 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). CHEMBL3544914. Open-access bioactivity database.
- PubChem — National Center for Biotechnology Information (NCBI). CID 46199646. Chemical information database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.
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