1Department of Radio-Diagnosis, Meenakshi Medical College Hospital & Research Institute, Meenakshi Academy of Higher Education and Research
2Department of Oral Medicine and Radiology, Meenakshi Ammal Dental College and Hospital
3Arulmigu Meenakshi College of Nursing, Meenakshi Academy of Higher Education and Research
4Meenakshi College of Physiotherapy, Meenakshi Academy of Higher Education and Research
Background: Transthyretin amyloid cardiomyopathy (ATTR-CM), which has been observed in wild-type, as well as in hereditary forms, is a disease whereby progressive myocardial infiltration is observed, resulting in heart failure and high mortality. Existing treatment only lowers the rates of disease progression, and it does not always lead to any significant stabilization in phenotypes.
Objective: This paper assesses the efficacy and safety of a next-generation transthyretin stabilizer named acoramidis when used in patients with wild-type and variant ATTR-CM.
Method: An ATTR-CM case of confirmed patients treated at random was utilized in a randomized, double-blind, placebo-controlled trial of adults. Acoramidis or placebo matched controls were used to treat the participants (30 months). Outcomes of primary concerns were the change of the levels of NT-proBNP, the six-minute walk distance, and all-cause mortality. Secondary outcomes were quality of life, hospitalization rates and safety profiles.
Results: Acoramidis found that there was pronounced biochemical stabilization of transthyretin and that there were NT-proBNP and delayed functional deterioration as compared to placebo. In the wild-type subgroups, as well as variant ones, improvements were noticed. The treatment was well-tolerated generally, and no difference was observed between the treatment and the placebo groups in the occurrence and severity of adverse events.
Conclusion: Acoramidis demonstrates promising efficacy and good safety profile in the management of ATTR-CM, which presents a possible disease-modifying therapy against wild-type and hereditary ATTR-CM. More long-term outcomes should also be done to ensure benefit sustainability.
Keywords: Acoramidis, ATTR-CM, TTR stabilizer, ATTR-CM, wild type ATTR, NT-proBNP
How to cite this article: Keerthi BHP, Sasidharan S, Biswas PG, Rani GA, Uma S, Kodi M. Evaluating the Efficacy and Safety of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy. Int J Drug Deliv Technol. 2026;16(10s): 145-149; DOI: 10.25258/ijddt.16.10s.21
Source of support: Nil.
Conflict of interest: None