tezacaftor   Click here for help

GtoPdb Ligand ID: 10199

Synonyms: example 315 [US20090131492A1] | VX-661 | VX661
Approved drug
tezacaftor is an approved drug (EMA & FDA (2018))
Compound class: Synthetic organic
Comment: Tezacaftor is a small molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulator that was developed by Vertex Pharmaceuticals [1].
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2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 5
Hydrogen bond donors 4
Rotatable bonds 9
Topological polar surface area 113.18
Molecular weight 520.18
XLogP 2.67
No. Lipinski's rules broken 0
SMILES / InChI / InChIKey
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Canonical SMILES OCC(Cn1c2cc(F)c(cc2cc1C(CO)(C)C)NC(=O)C1(CC1)c1ccc2c(c1)OC(O2)(F)F)O
Isomeric SMILES OC[C@@H](Cn1c2cc(F)c(cc2cc1C(CO)(C)C)NC(=O)C1(CC1)c1ccc2c(c1)OC(O2)(F)F)O
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
InChI Key MJUVRTYWUMPBTR-MRXNPFEDSA-N
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Summary of Clinical Use Click here for help
Tezacaftor was approved in 2018 in a fixed-dose combination with ivacaftor with the trade name Symdeko® as a therapy for patients with cystic fibrosis with specific gene mutations (see Table 1 in the drugs.com monograph using the link below for a full list of indicated mutations).
Mechanism Of Action and Pharmacodynamic Effects Click here for help
The combination of tezacaftor and ivacaftor improves the delivery of CFTR protein to the cell surface and increases the transporter's ability to move anions across the membrane to help restore ion and fluid balance.
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