From the Guidelines
Tafamidis is the only therapy that improves cardiovascular outcomes in transthyretin amyloid cardiomyopathy (ATTR-CM) and should be initiated as soon as the disease is diagnosed, regardless of disease stage, to maximize its benefits on morbidity, mortality, and quality of life. The medication is available in two formulations: tafamidis meglumine 80 mg daily or tafamidis 61 mg daily, which are therapeutically equivalent 1. Tafamidis works by binding to the thyroxin-binding site of transthyretin (TTR), preventing the formation of amyloid fibrils that damage the heart. Key benefits of tafamidis include:
- Reduced all-cause mortality by 29.5% compared to 42.9% in the placebo group 1
- Lower cardiovascular-related hospitalization rates, with 0.48 versus 0.70 per year after 30 months 1
- Improved quality of life, with an estimated increase in average survival by 1.97 years and QALY by 1.29 1 However, it's essential to note that tafamidis is most effective when administered early in the disease course and has not shown benefit in patients with class IV symptoms, severe aortic stenosis, or impaired renal function (eGFR <25 mL/min/1.73 m² body surface area) 1. Regular follow-up with a cardiologist is necessary to monitor disease progression and medication effectiveness. Despite its high cost, with an annual cost of $225,000, tafamidis has been shown to provide significant clinical benefits, and its use should be considered in patients with ATTR-CM who are likely to benefit from treatment 1.
From the FDA Drug Label
Tafamidis is a selective stabilizer of TTR. Tafamidis binds to TTR at the thyroxine binding sites, stabilizing the tetramer and slowing dissociation into monomers, the rate-limiting step in the amyloidogenic process. The main use of Tifamidis, also known as Tafamidis, is as a selective stabilizer of transthyretin (TTR), which helps to prevent the formation of amyloid deposits.
- Key points about Tifamidis include:
- It binds to TTR at the thyroxine binding sites
- It stabilizes the tetramer and slows dissociation into monomers
- It is used to treat transthyretin-related hereditary amyloidosis 2
From the Research
Tifamidis Overview
- Tifamidis, also known as tafamidis, is a medication used to treat transthyretin-related amyloidosis (ATTR) [(3,4,5,6,7)].
- It works by selectively binding to transthyretin (TTR) and stabilizing its tetrameric structure, thereby preventing the formation of amyloid fibrils [(3,4,5,6,7)].
Efficacy and Safety
- Clinical studies have shown that tafamidis is effective in slowing down the progression of neuropathy and maintaining nutritional status and quality of life in patients with early-stage ATTR [(3,4,6)].
- Tafamidis has also been shown to be safe and well-tolerated, with no significant safety or tolerability issues reported in long-term studies [(3,4,6)].
- The medication has been approved for use in several countries, including Europe, Japan, Mexico, and Argentina, for the treatment of early-stage TTR-related amyloidosis [(3,4)].
Clinical Applications
- Tafamidis is used to treat transthyretin amyloidosis with polyneuropathy (ATTR-PN), a rare and progressive hereditary disorder [(4,5)].
- It is also used to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a condition characterized by the deposition of amyloid fibrils in the heart 7.
- The medication is administered orally once daily, and its long-term use has been shown to be effective in delaying disease progression and conferring survival benefits in patients with stage 1 ATTR-PN [(4,6)].