Tafamidis is a specific transthyretin stabiliser designed to prevent the formation of the misfolded proteins and the subsequent amyloid deposits that induce neurodegeneration and decline of neurological function. The original compound Fx-1006 has been modified as the tafamidis meglumine salt in pharmacological development. It has recently been shown to benefit TTR-FAP cardiac amyloidosis, with symptom improvement soon after commencement and hard outcome improvement after 18 months.
Cautions and Interactions
- Upper abdominal pain
- Urinary tract infection
- Vaginal infection
Intrinsically disordered proteins (IDPs, natively unfolded proteins) lack stable tertiary and/or secondary structure under physiological conditions in vitro and are often involved in cell regulation, signaling and control. Misfolding of such proteins can lead to amyloid production, which is what this drug tries to prevent. Occupies vacant thyroxine binding sites in transthyretin, stablilising the transthyretin and rendering less likely to aggregate into amyloid. Seems more effective in wild-type form and in some mutations (e.g. Val30Met) of the hereditary form.
- ↑ Coelho T, Maia LF, Martins da Silva A, Waddington Cruz M, Planté-Bordeneuve V, Lozeron P, Suhr OB, Campistol JM, Conceição IM, Schmidt HH, Trigo P, Kelly JW, Labaudinière R, Chan J, Packman J, Wilson A, Grogan DR. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012 Aug 21; 79(8):785-92.(Link to article – subscription may be required.)