Preferred Label : Acoramidis Hydrochloride;
NCIt synonyms : AG10 Hydrochloride; ALXN2060 Hydrochloride;
NCIt related terms : Attruby;
NCIt definition : The hydrochloride salt form of acoramidis, 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 : VY9C88C2NV;
CAS number : 2242751-53-5;
Drug name : Attruby;
NCI Metathesaurus CUI : CL1648537;
Origin ID : C179070;
UMLS CUI : C5555442;
Currated CISMeF NLP mapping
Semantic type(s)
concept_is_in_subset
has_free_acid_or_base_form
is_salt_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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