Key result
Transthyretin-directed therapies gain FDA approval for ATTR-CM, but real-world prescribing data remain limited.
Why the study?
Despite FDA approvals and guideline recommendations for transthyretin-directed therapies including tafamidis, acoramidis, and vutrisiran for ATTR-CM, real-world prescribing data remain limited.
Observational
Persistent underdiagnosis of ATTR-CM limits uptake of approved therapies; leaves open optimal screening strategies in older HF patients.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed and undertreated cause of heart failure in older adults. Tafamidis received Food and Drug Administration (FDA) approval in 2019 following the ATTR-ACT trial, [1] and a Class I recommendation in the 2022 AHA/ACC/HFSA heart failure guideline. In 2024, acoramidis (Attruby) received FDA approval following the ATTRibute-CM trial. In 2025, vutrisiran (Amvuttra) received FDA approval following the HELIOS-B trial [2]. Despite these advances, real-world prescribing data remains limited [3].
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Menda et al. (2026) conducted an observational in Transthyretin amyloid cardiomyopathy (ATTR-CM). Transthyretin-directed therapy (tafamidis, acoramidis, vutrisiran) was evaluated. Transthyretin-directed therapies (tafamidis, acoramidis, vutrisiran) have received FDA approval for ATTR-CM since 2019, though real-world prescribing data remain limited.
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