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Vyndaqel (Tafamidis)
Vyndaqel stabilizes the Transthyretin protein, preventing its misfolding and aggregation into amyloid fibrils.
Vyndaqel (Tafamidis) is a small molecule drug developed by Foldrx Pharms, targeting Transthyretin, a protein associated with amyloidosis. It was FDA-approved in 2019 for the treatment of amyloid polyneuropathy type I, amyloidosis, transthyretin-related familial amyloid cardiomyopathy, and wild-type transthyretin cardiac amyloidosis. Vyndaqel works by stabilizing the Transthyretin protein, preventing its misfolding and aggregation into amyloid fibrils. The drug has a half-life of 49 hours and is currently patented, with no generic manufacturers available. Key safety considerations include its potential effects on the liver and kidneys.
At a glance
| Generic name | Tafamidis |
|---|---|
| Sponsor | Foldrx Pharms |
| Drug class | tafamidis |
| Target | Transthyretin |
| Modality | Small molecule |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2019 |
Mechanism of action
Tafamidis is selective stabilizer of TTR. Tafamidis binds to TTR at the thyroxine binding sites, stabilizing the tetramer and slowing dissociation into monomers, the rate-limiting step in the amyloidogenic process.
Approved indications
- Amyloid polyneuropathy type I
- Amyloidosis
- Transthyretin related familial amyloid cardiomyopathy
- Wild-type transthyretin cardiac amyloidosis
Common side effects
- Cardiac failure
- Cardiac amyloidosis
- Atrial fibrillation
- Disease progression
- Left ventricular hypertrophy
- Dysphagia
- Dyspnoea exertional
- Essential hypertension
- Chronic left ventricular failure
Key clinical trials
- A Retrospective Observational Study of Epidemiology and Outcomes in Transthyretin Amyloid Cardiomyopathy in Germany
- Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy
- A Study to Compare the Amount of Two Forms of Tafamidis in the Blood of Healthy Adults Under Fed Conditions (PHASE1)
- Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
- A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis (PHASE4)
- Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study
- Amyloidosis TTR Flow Reserve Evaluation (NA)
- A Study to Learn About Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in India
Patents
| Patent | Expiry | Type |
|---|---|---|
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 |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Vyndaqel CI brief — competitive landscape report
- Vyndaqel updates RSS · CI watch RSS
- Foldrx Pharms portfolio CI