Tafamidis Meglumine is a medication primarily used to treat transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin familial amyloid polyneuropathy (ATTR-PN). It stabilizes the transthyretin (TTR) protein, which in its mutated form can dissociate and aggregate into amyloid fibrils that deposit in tissues, causing organ dysfunction.
Mechanism of Action:
1.Stabilizes TTR Tetramer: Tafamidis selectively binds to the thyroxine (T4) binding sites on the tetramer form of TTR. This binding prevents the dissociation of the TTR tetramer into monomers, which is the rate-limiting step in the formation of toxic amyloid fibrils.
2.Prevents Amyloid Fibril Formation: By stabilizing the tetrameric form, Tafamidis inhibits the formation of amyloid fibrils and thus prevents their deposition in tissues and subsequent organ damage.
