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Acoramidis (AG10)
Acoramidis (AG10) is a Transthyretin stabilizer Small molecule drug developed by Eidos Therapeutics, a BridgeBio company. It is currently in Phase 3 development for Transthyretin amyloid cardiomyopathy (ATTR-CM), Hereditary transthyretin amyloidosis (hATTR). Also known as: ALXN2060, AG10, ALXN 2060.
Acoramidis stabilizes transthyretin (TTR) protein to prevent its misfolding and aggregation, which causes amyloid disease.
Acoramidis (AG10) is a small molecule drug that stabilizes transthyretin, a protein associated with amyloidosis. It is being studied for the treatment of conditions such as amyloid cardiomyopathy and familial amyloidosis.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Rare-disease pathway favourability
+5.0pp
Rare-disease drugs benefit from FDA Orphan Drug Act, smaller pivotal trials, and more flexible endpoints. Approval rates run ~5pp above baseline.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Acoramidis (AG10) |
|---|---|
| Also known as | ALXN2060, AG10, ALXN 2060 |
| Sponsor | Eidos Therapeutics, a BridgeBio company |
| Drug class | Transthyretin stabilizer |
| Target | Transthyretin (TTR) |
| Modality | Small molecule |
| Therapeutic area | Rare disease / Cardiology |
| Phase | Phase 3 |
Mechanism of action
Acoramidis is a selective transthyretin stabilizer that binds to the TTR protein and increases its thermodynamic stability, preventing dissociation and subsequent misfolding into amyloid fibrils. By stabilizing the native TTR tetramer, the drug reduces amyloid formation and deposition in tissues, thereby slowing or halting disease progression in transthyretin amyloidosis.
Approved indications
- Transthyretin amyloid cardiomyopathy (ATTR-CM)
- Hereditary transthyretin amyloidosis (hATTR)
Common side effects
- Peripheral edema
- Dyspnea
- Fatigue
- Diarrhea
Key clinical trials
- Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants (PHASE1)
- Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant (PHASE3)
- A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM (PHASE3)
- Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants (PHASE3)
- To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE) (PHASE3)
- A Single Dose Study to Evaluate the Pharmacokinetics of Acoramidis Modified Release Formulations in Healthy Subjects (PHASE1)
- Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN) (PHASE3)
- Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Acoramidis (AG10) CI brief — competitive landscape report
- Acoramidis (AG10) updates RSS · CI watch RSS
- Eidos Therapeutics, a BridgeBio company portfolio CI
Frequently asked questions about Acoramidis (AG10)
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Related
- Drug class: All Transthyretin stabilizer drugs
- Target: All drugs targeting Transthyretin (TTR)
- Manufacturer: Eidos Therapeutics, a BridgeBio company — full pipeline
- Therapeutic area: All drugs in Rare disease / Cardiology
- Indication: Drugs for Transthyretin amyloid cardiomyopathy (ATTR-CM)
- Indication: Drugs for Hereditary transthyretin amyloidosis (hATTR)
- Also known as: ALXN2060, AG10, ALXN 2060
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing