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Tafamidis: A Review in Transthyretin Amyloid Cardiomyopathy

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posted on 20.01.2021, 20:02 by Yvette Lamb
Declarations

Funding The preparation of this review was not supported by any external funding.

Authorship and Conflict of interest Yvette Lamb is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability not applicable

Additional information about this Adis Drug Review can be found here

Abstract

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, life-threatening disease characterized by the aggregation and deposition of amyloidogenic misfolded transthyretin (TTR) in the myocardium. The gradual accumulation of insoluble TTR amyloid fibrils can result in restrictive cardiomyopathy and heart failure. Tafamidis (Vyndaqel®; Vyndamax®), a TTR stabilizer, has been approved for use in the treatment of adults with ATTR-CM in several countries. Tafamidis stabilizes both wild-type and mutant TTR, inhibiting the formation of TTR amyloid fibrils. In the pivotal phase III ATTR-ACT trial, tafamidis significantly reduced all-cause mortality and frequency of cardiovascular-related hospitalizations relative to placebo in patients with ATTR-CM. In addition, tafamidis recipients experienced significantly less deterioration in 6-minute walk test distance and quality of life than placebo recipients over the 30-month treatment period. Treatment benefits were largely consistent between patients with wild-type TTR and patients with a variant TTR genotype. Tafamidis was generally well tolerated in patients with ATTR-CM and, with a safety profile similar to that of placebo, tafamidis is suitable for long-term use. Given that treatment for this condition has in the past been largely limited to symptom management, tafamidis constitutes a valuable disease-modifying therapy for patients with ATTR-CM.

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