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<p><b>New page</b></p><div>[[File:Tafamidis.svg|thumb|Tafamidis.svg]] '''Tafamidis''' is a medication used for the treatment of [[transthyretin amyloidosis]], a rare and progressive disease caused by the misfolding of the [[transthyretin]] protein. Tafamidis works by stabilizing the transthyretin protein, thereby preventing its misfolding and the subsequent formation of amyloid fibrils that deposit in various tissues and organs.<br />
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== Medical Uses ==<br />
Tafamidis is primarily indicated for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloid polyneuropathy (ATTR-PN). ATTR-CM is characterized by the deposition of amyloid fibrils in the heart, leading to restrictive cardiomyopathy and heart failure. ATTR-PN involves the deposition of amyloid fibrils in peripheral nerves, causing progressive sensory and motor neuropathy.<br />
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== Mechanism of Action ==<br />
Tafamidis binds selectively to the thyroxine-binding sites of transthyretin, stabilizing the tetrameric form of the protein. This stabilization prevents the dissociation of transthyretin into monomers, which is the rate-limiting step in the formation of amyloid fibrils. By inhibiting this process, tafamidis reduces the deposition of amyloid fibrils in tissues and organs.<br />
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== Dosage and Administration ==<br />
Tafamidis is administered orally, typically in the form of soft gelatin capsules. The recommended dosage for ATTR-CM is 80 mg once daily, while for ATTR-PN, the dosage is 20 mg once daily. The medication should be taken with or without food.<br />
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== Side Effects ==<br />
Common side effects of tafamidis include:<br />
* [[Urinary tract infection]]<br />
* [[Diarrhea]]<br />
* [[Abdominal pain]]<br />
* [[Nausea]]<br />
* [[Fatigue]]<br />
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Serious side effects are rare but may include liver function abnormalities and heart failure exacerbation.<br />
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== History ==<br />
Tafamidis was developed by [[Pfizer]] and received its first approval in the [[European Union]] in 2011 for the treatment of ATTR-PN. It was later approved by the [[United States Food and Drug Administration]] (FDA) in 2019 for the treatment of ATTR-CM.<br />
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== Research ==<br />
Ongoing research is exploring the efficacy of tafamidis in other forms of amyloidosis and its long-term effects on disease progression and patient quality of life. Clinical trials are also investigating combination therapies involving tafamidis and other agents that target amyloid fibril formation.<br />
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== See Also ==<br />
* [[Transthyretin]]<br />
* [[Amyloidosis]]<br />
* [[Cardiomyopathy]]<br />
* [[Peripheral neuropathy]]<br />
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== References ==<br />
{{Reflist}}<br />
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== External Links ==<br />
{{Pfizer}}<br />
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[[Category:Drugs]]<br />
[[Category:Cardiology]]<br />
[[Category:Neurology]]<br />
[[Category:Orphan drugs]]<br />
[[Category:Transthyretin amyloidosis]]<br />
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