Tafamidis: is it truly a game changer for transthyretin amyloid cardiomyopathy?

Transthyretin cardiac amyloidosis (ATTR-CM) is an increasingly recognized condition caused by the misfolding of transthyretin (TTR) protein, leading to amyloid deposits in various tissues and organs. In 2019, tafamidis was the first FDA-approved disease-modifying therapy to address an unmet need for patients with ATTR-CM, stabilizing TTR proteins and preventing dissociation. Tafamidis has been shown to reduce all-cause mortality and slow decline in functional capacity and quality of life and is generally considered a safe treatment because of its minimal major drug interactions. However, tafamidis has several well-recognized limitations, including high cost, reduced effectiveness in advanced disease stages, limited ability to address established amyloid burden, and limited evidence for advanced polyneuropathy. As new therapies and advancements emerge, further research is needed to compare their relative effectiveness. In this review, we explore the development of tafamidis, pivotal clinical trial results, clinical applications, safety considerations, and future therapeutic directions for ATTR-CM.