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Vyndaqel (Tafamidis Meglumine)
Tafamidis selectively stabilizes TTR tetramer by binding at thyroxine sites, slowing monomer dissociation.
Tafamidis meglumine (Vyndaqel) is a transthyretin stabilizer indicated for treating cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults. The drug demonstrates strong protein binding (>99%) with a half-life of approximately 49 hours and primarily hepatic clearance via glucuronidation. Key risk is BCRP inhibition, which may increase exposure to BCRP substrate drugs requiring monitoring and potential dose adjustment. Clinical efficacy is supported by reduction in cardiovascular mortality and cardiovascular-related hospitalization in ATTR-CM patients.
At a glance
| Generic name | Tafamidis Meglumine |
|---|---|
| Sponsor | Foldrx Pharms |
| Drug class | Transthyretin stabilizer |
| Target | Transthyretin (TTR) |
| Modality | Small molecule |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2019 |
Mechanism of action
Tafamidis is a selective stabilizer of transthyretin (TTR) that binds to TTR at the thyroxine binding sites. By binding at these sites, tafamidis stabilizes the TTR tetramer and slows its dissociation into monomers. This mechanism is significant because monomer dissociation is the rate-limiting step in the amyloidogenic process, the pathological cascade that leads to transthyretin-mediated amyloidosis.
Approved indications
- Amyloid polyneuropathy type I
- Amyloidosis
- Transthyretin related familial amyloid cardiomyopathy
- Wild-type transthyretin cardiac amyloidosis
Common side effects
- Fall
- Dyspnoea
- Cough
- Atrial fibrillation
- Constipation
- Oedema peripheral
- Cardiac failure
- Dizziness
- Diarrhoea
- Arthralgia
- Insomnia
- Fatigue
Drug interactions
- BCRP substrates (e.g., methotrexate, rosuvastatin, imatinib)
- CYP3A4 substrates (e.g., midazolam)
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 |
| 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