Tafamidis: Transthyretin Stabilizer Therapy

Tafamidis (Vyndaqel/Vyndamax) stabilizes transthyretin (TTR) tetramer, preventing amyloidogenesis. It is the only approved disease-modifying therapy in ATTR cardiomyopathy (ATTR-CM). ATTR-ACT trial showed 30% mortality reduction and 32% cardiovascular hospitalization reduction at 30 months. Effective in both wild-type (wt-ATTR) and hereditary (h-ATTR) variants. Maximum benefit in NYHA Class I-III patients. Dose: tafamidis meglumine 80 mg or tafamidis free base 61 mg daily. Well tolerated with mild GI side effects. Acoramidis (Attruby) FDA-approved 2024 as new alternative. ESC 2023 Class I recommendation.