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Year : 2021  |  Volume : 2  |  Issue : 2  |  Page : 32-37

Transthyretin amyloid cardiomyopathy: Treatment pipeline, clinical trials, and challenges

1 Department of Pharmacology, Delhi Institute of Pharmaceutical Sciences and Research, Delhi, India
2 Department of Nursing, Rajkumari Amrit Kaur College of Nursing, Delhi, India

Correspondence Address:
Dr. Sweety Sharma
Department of Pharmacology, Delhi Institute of Pharmaceutical Sciences and Research, Delhi
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Source of Support: None, Conflict of Interest: None

DOI: 10.4103/jphpc.jphpc_8_21

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Transthyretin amyloid cardiomyopathy (ATTR-CM) is potentially a fatal disease characterized by abnormal buildup of amyloid fibrils primarily in the heart causing progressive heart failure. It is categorized into two subtypes-hereditary ATTR and wild type ATTR. Previously, no treatment is available, due to which liver transplantation, multi-organ transplantation, and symptomatic treatment were the only therapies at that time. Approval of Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules in 2019, acts like a kick in the research fields due to which other therapeutics are now emerging. Several clinical trials are going on to evaluate the efficacy of different drugs in ATTR-CM. Most of the clinical trials demonstrated positive outcomes which leads to further evaluation for confirmation. In this review treatment pipeline, ongoing clinical trials and challenges related to ATTR-CM are described.

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