Assessing Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy
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Reviewed by Dat Tien Nguyen, B.A, ScM.
Translated by Nhi Phuong Quynh Le, B.A |
Posted on March 6th, 2024
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Transthyretin (TTR) is a crucial transport hormone that functions in a tetramer arrangement. Mutations can lead to the dissociation of the monomer from the tetramer, resulting in its deposition in the myocardium and causing transthyretin amyloid cardiomyopathy. A study was undertaken to evaluate the efficacy of Acoramidis, a TTR stabilizer with a high binding affinity, in treating transthyretin amyloid cardiomyopathy.
The clinical trial involved 632 patients who were diagnosed with transthyretin amyloid cardiomyopathy via endomyocardial biopsy. The average age of the patients was 77 years, with a mean NT-proBNP level of 2872 ng/L. Participants were randomly allocated to receive either 800 mg of Acoramidis orally or a placebo. Following a 30-month follow-up period, the study concluded that Acoramidis effectively reduced serum transthyretin levels by 7.01 mg per deciliter and NT-proBNP levels. Clinically, Acoramidis decreased the risk of mortality and enhanced patients' physical capacity. Additionally, the use of Acoramidis did not result in a significant increase in adverse effects.
The clinical trial involved 632 patients who were diagnosed with transthyretin amyloid cardiomyopathy via endomyocardial biopsy. The average age of the patients was 77 years, with a mean NT-proBNP level of 2872 ng/L. Participants were randomly allocated to receive either 800 mg of Acoramidis orally or a placebo. Following a 30-month follow-up period, the study concluded that Acoramidis effectively reduced serum transthyretin levels by 7.01 mg per deciliter and NT-proBNP levels. Clinically, Acoramidis decreased the risk of mortality and enhanced patients' physical capacity. Additionally, the use of Acoramidis did not result in a significant increase in adverse effects.