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Treatment for TTR amyloidosis (treatment-for-ttr-amyloidosis)
Treatment for TTR amyloidosis (generic name: treatment-for-ttr-amyloidosis) is a The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if n drug developed by Pfizer Inc.. It is currently in preclinical development.
The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if n
Treatment for TTR amyloidosis includes the use of small molecule interventions such as Tafamidis and ALN-TTRSC (revusiran), which are administered subcutaneously. Cardiac Contractility Modulation (CCM) therapy is also being studied as a potential treatment option for patients with TTR amyloidosis and heart failure.
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Baseline preclinical → approval rate
+5.0pp
Industry-wide preclinical drugs reach approval ~5% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Cardiovascular Phase 3 risk
-2.0pp
Modern cardiovascular outcome trials are large + long; many fail to beat aggressive standard-of-care. -
Big-pharma sponsor
+3.0pp
Pfizer Inc. is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2036–2040 | — |
| EMA | EU | 2037–2041 | +0.7 yr |
| MHRA | GB | 2037–2041 | +0.7 yr |
| Health Canada | CA | 2037–2042 | +0.9 yr |
| TGA | AU | 2037–2042 | +1.2 yr |
| PMDA | JP | 2037–2042 | +1.5 yr |
| NMPA | CN | 2038–2043 | +2.3 yr |
| MFDS | KR | 2037–2042 | +1.4 yr |
| CDSCO | IN | 2037–2043 | +1.8 yr |
| ANVISA | BR | 2038–2043 | +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 | treatment-for-ttr-amyloidosis |
|---|---|
| Sponsor | Pfizer Inc. |
| Drug class | The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if n |
| Therapeutic area | Cardiovascular |
| Phase | preclinical |
Approved indications
Pipeline indications
- Transthyretin (TTR) Amyloid Cardiomyopathy — preclinical
Common side effects
Key clinical trials
- Retrospective Study Collecting Neurological Follow-up of Hereditary Transthyretin Amyloidosis (ATTRv (N/A)
- ATTR-CM: A Multi-country, Non-interventional Disease Registry (N/A)
- Thailand ATTR-CM Registry (N/A)
- Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) A (Phase 3)
- Transthyretin Amyloidosis Outcome Survey (THAOS) (N/A)
- Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) (N/A)
- Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy o (N/A)
- Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis (Phase 2/3)
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:
- Treatment for TTR amyloidosis CI brief — competitive landscape report
- Treatment for TTR amyloidosis updates RSS · CI watch RSS
- Pfizer Inc. portfolio CI
Frequently asked questions about Treatment for TTR amyloidosis
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Related
- Drug class: All The usual adult dose is 80 mg of Tafamidis Meglumine orally once daily. The dose may be reduced if n drugs
- Manufacturer: Pfizer Inc. — full pipeline
- Therapeutic area: All drugs in Cardiovascular
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing