What is Tafamidis (Vyndaqel) used for?

From the Guidelines

Tafamidis (Vyndaqel) is used to treat Transthyretin Cardiomyopathy (ATTR-CM), a condition where abnormal proteins accumulate in the heart, leading to heart failure.

Key Points

• Tafamidis binds to the thyroxin-binding site of Transthyretin (TTR) and has been shown to improve cardiovascular outcomes in patients with ATTR-CM 1.
• The medication is available in two formulations: 20-mg capsules (FDA-approved dose: 80 mg once daily) and 61-mg capsules (FDA-approved dose: 61 mg once daily) 1.
• Tafamidis has been shown to reduce all-cause mortality and cardiovascular-related hospitalization in patients with ATTR-CM, particularly those with NYHA class I-III symptoms 1.
• The benefit of tafamidis is expected to be greater when administered early in the disease course, and it is recommended for patients with NYHA functional class I-II symptoms 1.
• However, the use of tafamidis in patients with NYHA functional class III symptoms is still a topic of debate, and a case-by-case decision is needed when determining eligibility for treatment 1.
• Tafamidis is not recommended for patients with severe aortic stenosis, impaired renal function (eGFR <25 mL/min/1.73 m² body surface area), or class IV symptoms 1.
• The high cost of tafamidis may limit its use, and its incremental value is limited in patients with advanced amyloidosis 1.

From the FDA Drug Label

VYNDAQEL and VYNDAMAX are transthyretin stabilizers indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are prescription medicines used to treat adults with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) to reduce death and hospitalization related to heart problems.

Tafamidis (Vyndaqel) is used for:

• Treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults
• Reduction of cardiovascular mortality and cardiovascular-related hospitalization in adults with ATTR-CM 2, 2, 2

From the Research

Tafamidis (Vyndaqel) Uses

• Tafamidis is approved for the treatment of transthyretin amyloidosis with polyneuropathy (ATTR-PN) in adults with stage 1 symptomatic polyneuropathy, to delay peripheral neurological impairment 3
• It is also approved for the treatment of transthyretin amyloid cardiomyopathy 4
• Tafamidis works by kinetically stabilizing the tetramer, slowing monomer formation, misfolding, and amyloidogenesis, thereby slowing the progression of peripheral neurologic impairment in TTR familial amyloid polyneuropathy 5

Administration and Bioequivalence

• Tafamidis is administered orally once daily, as a soft capsule 3
• A bioequivalence study found that tafamidis 61-mg free acid capsules are bioequivalent to tafamidis meglumine 80-mg (4 × 20-mg) capsules 6

Clinical Applications

• Tafamidis has been shown to slow deterioration of neurological function and maintain health-related quality of life in patients with early-stage ATTR-PN and the Val30Met mutation 3
• It has also been shown to reduce mortality in patients with transthyretin amyloid cardiomyopathy 4
• The development of tafamidis represents a major breakthrough in the treatment of transthyretin amyloidosis (ATTR amyloidosis) 7