tafamidis   Click here for help

GtoPdb Ligand ID: 8378

Synonyms: FX-1006 | Vyndamax® | Vyndaqel®
Approved drug PDB Ligand
tafamidis is an approved drug (EMA and UK (2011), FDA (2019))
Compound class: Synthetic organic
Comment: Tafamidis binds potently and selectively to transthyretin (TTR), stabilising protein tetramers, thereby inhibiting the formation of amyloid fibrils [2]. The trade name for this drug is Vyndaqel which contains tafamidis meglumine (PubChem CID 24970412). The Vyndamax brand contains tafamidis parent molecule.
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2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 3
Hydrogen bond donors 1
Rotatable bonds 2
Topological polar surface area 63.33
Molecular weight 306.98
XLogP 4.02
No. Lipinski's rules broken 0
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Canonical SMILES Clc1cc(Cl)cc(c1)c1nc2c(o1)cc(cc2)C(=O)O
Isomeric SMILES Clc1cc(Cl)cc(c1)c1nc2c(o1)cc(cc2)C(=O)O
InChI InChI=1S/C14H7Cl2NO3/c15-9-3-8(4-10(16)6-9)13-17-11-2-1-7(14(18)19)5-12(11)20-13/h1-6H,(H,18,19)
No information available.
Summary of Clinical Use Click here for help
Tafamidis is approved to treat transthyretin (TTR)-mediated amyloidosis (aka familial amyloid polyneuropathy or FAP), a rare hereditary disease caused by amyloid build up in tissues around the body including around the nerves. Its use can reduce cardiovascular mortality and cardiovascular-related hospitalisations in patients with cardiomyopathy resulting from TTR-mediated amyloidosis [4,6]. The drug was granted orphan designation in the EU (2012) for the treatment of senile systemic amyloidosis (SSA), another TTR amyloid disease [7].
Mechanism Of Action and Pharmacodynamic Effects Click here for help
Tafamidis stabilises TTR tetramer formation, preventing the dissociation which underlies the mis-folding and aggregation of TTR into amyloid fibrils. The deposition of these fibrils eventually causes degradation of the autonomic nervous system [1].
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