Tafamidis for Patients with Transthyretin Amyloid Cardiomyopathy: Promising Results

Transthyretin amyloid cardiomyopathy, a life-threatening condition, occurs when misfolded transthyretin protein accumulates as amyloid fibril deposits in the heart, leading to heart failure (HF) and conduction disorders. It occurs primarily in men aged ≥60 and can be inherited as an autosomal dominant trait or by deposition of wild-type transthyretin protein. Among people with HF and preserved systolic function, the condition's prevalence may exceed 10%; treatment has been limited to supportive care.

Tafamidis binds to transthyretin and stabilizes it, inhibiting its deposition as amyloid. In the manufacturer-funded, phase III ATTR-ACT study (NCT01994889), researchers examined the efficacy and safety of tafamidis in 441 patients with heredita…