FDA approved two tafamidis preparations, Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), for the treatment of transthyretin mediated cardiomyopathy (ATT-CM).
An oral transthretin stabilizer, in two different formulations for adult transthretin mediated amyloid cardiomyopathy.
It selectively binds to an stabilizes transthretin, preventing dissociation of the tetramers and slowing the formation of amyloid.
A drug used to delay loss of peripheral nerve function in adults with familial amyloid polyneuropathy (FAP).
It stabilizes the correctly folded tetrameric form of the transthyretin (TTR) protein by binding in one of the two thyroxine-binding sites of the tetramer.
In people with FAP, the individual monomers fall away from the tetomer, misfold, and aggregate; the aggregates harm nerves.
Also used for the treatment of the heart disease caused by transthyretin mediated amyloidosis (ATTR-CM).
Works by stabilizing the protein, transthyretin, which is normally made up of four strands.
In FAP these strands separate and form clumps that harm nerves and invades other tissues.
Oral agent: 2 formulations
Two preparations, tafamidis meglumine and tafamidis, were approved for the treatment of transthyretin mediated cardiomyopathy (ATT-CM).
The two preparations have the same active moiety, tafamidis, but they are not substitutable on a milligram to milligram basis.
It is used to delay impairment of peripheral nerve function in adults with familial amyloid polyneuropathy (FAP).
It is used to delay impairment of peripheral nerve function in adults with familial amyloid polyneuropathy.
Not used in pregnancy, while breast feeding and after liver transplant.
Adverse events: 10% or more have urinary tract infections, vaginal infections, upper abdominal pain, or diarrhea.
Tafamidis does not interact with cytochrome P450 enzymes.
Inhibits BCRP-likely to affect availability of drugs including methotrexate, rosuvastatin, and imatinib.
Inhibits OAT1 and OAT3 so is likely to interact with NSAIDs and other drugs that rely on those transporters.
It functions as a chaperone that stabilizes the folded tetrameric form of the transthyretin (TTR) protein by binding in one of the two thyroxine-binding sites of the tetramer.
In FAP, monomers fall away from the tetomer, misfold, and aggregate; and the aggregates harm nerves.
The maximum plasma concentration is achieved around 2 hours after dosing.
It is almost completely bound to proteins.
It is metabolized by glucuronidation and excreted via bile; in humans.
Around 59% of a dose is recovered in feces, and approximately 22% in urine.