Tafamidis

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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.

Medical Uses

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.

Mechanism of Action

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.

Dosage and Administration

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.

Side Effects

Common side effects of tafamidis include:

• Urinary tract infection
• Diarrhea
• Abdominal pain
• Nausea
• Fatigue

Serious side effects are rare but may include liver function abnormalities and heart failure exacerbation.

History

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.

Research

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.

See Also

• Transthyretin
• Amyloidosis
• Cardiomyopathy
• Peripheral neuropathy