Drug intelligence / Profile preview

acoramidis

Development stage
Approved
Lead developer
BridgeBio Pharma
Modality
Small Molecules
Administration
Oral
01

Overview

Acoramidis is an orally administered small molecule drug used to treat cardiomyopathy associated with wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults, aiming to reduce cardiovascular death and hospitalization[1][2][5]. It acts as a selective stabilizer of the transthyretin (TTR) protein by binding at thyroxine binding sites, slowing the dissociation of TTR tetramers into monomers—the rate-limiting step in amyloid formation[3][4]. By stabilizing TTR, acoramidis prevents misfolding and aggregation of the protein into amyloid fibrils that deposit in organs such as the heart. The drug was developed to mimic the protective properties of a naturally occurring T119M mutation in TTR[5][6]. Acoramidis is marketed under the brand name Attruby and was developed by BridgeBio for oral use as a tablet formulation[3][6].

Brand names
Attruby
Other names
acoramidisALXN2060ALXN-2060ALXN 2060AG10AG-10AG 10
02

Targets

TTR (Transthyretin)

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