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 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; ALXN 2060; ALXN-2060;
NCI Metathesaurus CUI : CL1383184;
Origin ID : C170791;
UMLS CUI : C5418800;
Currated CISMeF NLP mapping
Manual BTNT mappings - CISMeF
Semantic type(s)
concept_is_in_subset
has_salt_form
has_target
is_free_acid_or_base_form_of
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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