Acoramidis
Chemical compound
From Wikipedia, the free encyclopedia
Acoramidis, sold under the brand name Attruby, is a medication used for the treatment of cardiomyopathy.[1] It is a near-complete (>90%) transthyretin stabilizer, developed to mimic the protective properties of the naturally occurring T119M mutation,[4][5] to treat transthyretin amyloid cardiomyopathy. It is taken by mouth.[1]
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| Pronunciation | ə-corAM-i-dis |
| Trade names | Attruby, others |
| Other names | AG10 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a625013 |
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| Routes of administration | By mouth |
| Drug class | Amyloidogenesis suppressant |
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| Formula | C15H17FN2O3 |
| Molar mass | 292.310 g·mol−1 |
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The most common adverse reactions include diarrhea and upper abdominal pain.[6]
Acoramidis was approved for medical use in the United States in November 2024,[6][7][8] and in the European Union in February 2025.[2][3]
Medical uses
Side effects
The most common side effects are diarrhea and abdominal pain.[10]
History
The efficacy and safety of acoramidis were evaluated in a multicenter, international, randomized, double-blind, placebo-controlled study in 611 adult participants with wild-type or hereditary (variant) ATTR-CM (NCT03860935).[6]
Clinical trials
Phase I data indicated acoramidis achieved near-complete (>90%) TTR stabilization across the entire dosing interval at steady state.[11]
Phase II and the Open-Label Extension (OLE) data indicated after a median of 38 months, long-term treatment with acoramidis was generally well tolerated and resulted in a median decline in NT-proBNP levels, normalization of serum TTR, and sustained stabilization of TTR in individuals with ATTR-CM.[12]
Phase III data from ATTRibute-CM indicated acoramidis resulted in a significantly better four-step primary hierarchical outcome containing components of mortality, morbidity, and function than placebo at 30 months in participants with ATTR-CM. Adverse events were similar in the two groups.[13]
Other analyses from ATTRibute-CM indicated a 50% reduction in cumulative cardiovascular hospitalizations (CVH), a 42% reduction in all-cause mortality (ACM) and recurrent CVH, and a 3-month time-to-separation of the Kaplan Meier curves for ACM or CVH. No other treatment has demonstrated this degree of treatment effect this quickly in participants with ATTR-CM.[14][15][16]
In vitro data indicated acoramidis exhibits near-complete (>90%) TTR stabilization at therapeutic trough concentrations, and its TTR stabilization exceeds that of tafamidis' across a range of destabilizing TTR mutations.[17]
Society and culture
Legal status
Acoramidis was approved for medical use in the United States in November 2024.[6][7][18] The approval was granted to BridgeBio Pharma.[9]
In December 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Beyonttra, intended for the treatment of transthyretin amyloidosis in adults with cardiomyopathy.[2] The applicant for this medicinal product is BridgeBio Europe B.V.[2] Acoramidis was designated an orphan medicine by the EMA.[2] Acoramidis was authorized for medical use in the European Union in February 2025.[2][3]
Names
During development, acoramidis was known as AG10 (the Alhamadsheh-Graef molecule 10).[19]
Acoramidis is the international nonproprietary name.[20]
Acoramidis is sold under the brand names Attruby[1][6] and Beyonttra.[2][3]
