Tafamidis (Vyndaqel): a light for FAP patients

ChemMedChem. 2013 Oct;8(10):1617-9. doi: 10.1002/cmdc.201300245. Epub 2013 Sep 2.

Abstract

Slowing FAP progression: Tafamidis meglumine is a small molecule capable of stabilizing the transthyretin (TTR) tetramer. Tafamidis acts in a similar way to the natural hormone T4, prevents TTR amyloid fibril formation, and offers a potential alternative to liver transplantation for the treatment of patients with TTR familial amyloid polyneuropathies (TTR-FAP).

Keywords: drug discovery; familial amyloid polyneuropathy; ligand design; tafamidis meglumine; transthyretin.

MeSH terms

  • Amyloid Neuropathies, Familial / drug therapy*
  • Benzoxazoles / chemistry
  • Benzoxazoles / metabolism
  • Benzoxazoles / therapeutic use*
  • Binding Sites
  • Drug Design
  • Humans
  • Mutation
  • Prealbumin / chemistry
  • Prealbumin / genetics
  • Prealbumin / metabolism
  • Protein Binding
  • Protein Structure, Tertiary

Substances

  • Benzoxazoles
  • Prealbumin
  • tafamidis