European Commission approves Vyndaqel® (tafamidis) for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP)

We know that, Tafamidis (see structure) or Vyndaqel was discovered in the Jeffery W. Kelly Laboratory at The Scripps Research Institute using a structure-based drug design strategy and was developed at FoldRx pharmaceuticals, a biotechnology company led by Richard Labaudiniere that was acquired by Pfizer in 2010.

Tafamidis or Vyndaqel functions by kinetic stabilization of the correctly folded tetrameric form of the transthyretin (TTR) protein. In patients with FAP, this protein dissociates in a process that is rate limiting for aggregation including amyloid fibril formation, causing neurodegeneration and failure of the autonomic nervous system and/or the peripheral nervous system and/or the heart.

Now, European Commission has approved Vyndaqel^® (tafamidis) for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in adult patients with stage 1 symptomatic polyneuropathy.

More...