Ligand id: 8378

Name: tafamidis

Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 3
Hydrogen bond donors 1
Rotatable bonds 2
Topological polar surface area 63.33
Molecular weight 306.98
XLogP 3.7
No. Lipinski's rules broken 0

Molecular properties generated using the CDK

No information available.
Summary of Clinical Use
In the EU tafamidis is approved to treat transthyretin (TTF) 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. The drug is being evaluated in Phase III clinical trial as a novel treatment for TTR amyloid cardiomyopathy [4]. The drug has been granted orphan designation in the EU (2012) for the treatment of senile systemic amyloidosis (SSA), another TTR amyloid disease [5].
Mechanism Of Action and Pharmacodynamic Effects
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].