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AG10 oral tablet
AG10 oral tablet is a Transthyretin stabilizer Small molecule drug developed by Eidos Therapeutics, a BridgeBio company. It is currently in Phase 3 development for Transthyretin cardiac amyloidosis (ATTR-CM), wild-type and hereditary forms. Also known as: Eidos Therapeutics AG10, TTR Stabilizer, Oral AG10.
AG10 stabilizes transthyretin (TTR) protein to prevent its misfolding and aggregation, which causes amyloid deposition in cardiac and neurological tissues.
AG10 is an oral tablet used in clinical trials for conditions such as Familial ATTR-CM, Wild-type ATTR-CM, and Amyloid Cardiomyopathy. AG10 is a biological vaccine that induces an immune response, according to its mechanism of action.
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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 | AG10 oral tablet |
|---|---|
| Also known as | Eidos Therapeutics AG10, TTR Stabilizer, Oral AG10 |
| Sponsor | Eidos Therapeutics, a BridgeBio company |
| Drug class | Transthyretin stabilizer |
| Target | Transthyretin (TTR) |
| Modality | Small molecule |
| Therapeutic area | Cardiovascular / Rare Disease |
| Phase | Phase 3 |
Mechanism of action
AG10 is a transthyretin stabilizer that binds to the TTR protein and increases its thermodynamic stability, preventing dissociation into monomers that would otherwise misfold and aggregate into amyloid fibrils. By stabilizing the native TTR tetramer, AG10 halts the pathological cascade underlying transthyretin amyloidosis, particularly in the cardiac form of the disease.
Approved indications
- Transthyretin cardiac amyloidosis (ATTR-CM), wild-type and hereditary forms
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)
- Single and Multiple Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of AG10 (PHASE1)
- Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy (PHASE3)
- 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)
- Study of AG10 in Amyloid Cardiomyopathy (PHASE2)
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:
- AG10 oral tablet CI brief — competitive landscape report
- AG10 oral tablet updates RSS · CI watch RSS
- Eidos Therapeutics, a BridgeBio company portfolio CI
Frequently asked questions about AG10 oral tablet
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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 Cardiovascular / Rare Disease
- Indication: Drugs for Transthyretin cardiac amyloidosis (ATTR-CM), wild-type and hereditary forms
- Also known as: Eidos Therapeutics AG10, TTR Stabilizer, Oral AG10
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing