Preferred Label : Acoramidis;

NCIt synonyms : TTR Stabilizer AG10; Transthyretin Stabilizer AG10;

NCIt definition : A potent, highly selective, orally bioavailable transthyretin (TTR) stabilizer with potential disease-modifying activity. Upon oral administration, acoramidis binds to and stabilizes transthyretin (TTR), thereby preventing tetramer dissociation into monomers. This prevents misfolding of the TTR protein and inhibits the formation of TTR amyloid fibrils and the subsequent deposition of these insoluble protein clusters in the heart and peripheral nerves. TTR is a 127 amino acid transport protein for thyroxine and retinol and is secreted by the liver into the blood. The accumulation of TTR amyloid fibrils may result in thickening and stiffening of the ventricular wall, leading to heart failure.;

UNII : T12B44A1OE;

InChIKey : WBFUHHBPNXWNCC-UHFFFAOYSA-N;

CAS number : 1446711-81-4;

Molecule name : AG-10; AG 10;

NCI Metathesaurus CUI : CL1383184;

Details


Main resources

You can consult :


https://www.ema.europa.eu/en/medicines/human/EPAR/beyonttra
2025
false
false
false
Netherlands
French
English
syndication feed
summary of product characteristics
package leaflet
treatment outcome
Acoramidis
Acoramidis
Acoramidis Hydrochloride
Acoramidis Hydrochloride
acoramidis
drug approval
europe
product surveillance, postmarketing
administration, oral
amyloidosis, hereditary, Transthyretin-Related
Hereditary ATTR amyloidosis
Wild type ATTR amyloidosis
adult
amyloidosis
cardiomyopathies
drug interactions
Pharmacological Chaperone
drug evaluation, preclinical

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11/05/2025


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