Acoramidis

Transthyretin Stabilizer — CARDIOVASCULAR SYSTEM

Key facts

Acoramidis is an onp drug in the Transthyretin Stabilizer class catalogued in DrugCentral. It was first approved by the FDA in 2024. Its mechanism of action is classified as Cytochrome P450 2C9 Inhibitors and Transthyretin Stabilizers. DrugCentral lists 1 molecular target, including TTR. Recorded indications include Transthyretin-mediated amyloidosis.

• Approval status: onp (first approved 2024 by the FDA).
• Drug class: Transthyretin Stabilizer.
• Mechanism of action: Cytochrome P450 2C9 Inhibitors; Transthyretin Stabilizers.
• Molecular targets: TTR (1 listed in DrugCentral).
• Indications: Transthyretin-mediated amyloidosis.
• ATC code: C01EB25 (Other cardiac preparations).
• Molecular formula: C15H17FN2O3; molecular weight 292.31 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

• Transthyretin-mediated amyloidosis

Mechanism & targets

• Mechanism of action (MoA): Cytochrome P450 2C9 Inhibitors; Transthyretin Stabilizers
• Established pharmacologic class: Transthyretin Stabilizer

Molecular targets (1)

Target	Gene	Action	Activity
Transthyretin	TTR	other	Kapp 7.79

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2025-03-27	PMDA	—	Alexion Pharma GK	Yes
2025-02-10	EMA	—	Bayer AG	Yes
2024-11-22	FDA	—	BridgeBio Pharma	Yes

ATC classification

ATC code	Therapeutic hierarchy
C01EB25	CARDIOVASCULAR SYSTEM › CARDIAC THERAPY › OTHER CARDIAC PREPARATIONS › Other cardiac preparations

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Cardiac failure	64.2
Renal impairment	57.5
Acute kidney injury	56.7
Atrioventricular block	56.0

Chemical structure

Full physicochemical, ADMET and 3D-structure analysis of Acoramidis is on its molecule page.

• Molecular formula: C₁₅H₁₇FN₂O₃
• Molecular weight: 292.31 g/mol
• CAS number: —
• InChIKey: WBFUHHBPNXWNCC-UHFFFAOYSA-N
• PubChem CID: 71464713

CC1=C(C(=NN1)C)CCCOC2=C(C=CC(=C2)C(=O)O)F

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Acoramidis used for?

DrugCentral lists the following indications for Acoramidis: Transthyretin-mediated amyloidosis.

How does Acoramidis work?

Acoramidis is classified as Cytochrome P450 2C9 Inhibitors and Transthyretin Stabilizers; its molecular targets in DrugCentral include TTR.

When was Acoramidis first approved?

Acoramidis was first approved by the FDA in 2024.

What drug class does Acoramidis belong to?

Acoramidis is in the Transthyretin Stabilizer class.

What is the chemical formula of Acoramidis?

Acoramidis has the molecular formula C15H17FN2O3 and a molecular weight of 292.31 g/mol.

Sources

• DrugCentral drug card 5842 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3940890 — compound record and bioactivity
• PubChem CID 71464713 — chemical identifiers
• FDA UNII T12B44A1OE — substance identity
• DailyMed: FDA labels for Acoramidis — current prescribing information (label search)
• UniProt P02766 (TTR) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral

How this page is built: methodology · editorial policy & corrections.