Active Ingredient History
Tafamidis meglumine (Vyndaqel®, Pfizer) is a novel, first-in-class drug for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP), a rare neurodegenerative disorder characterized by progressive sensory, motor and autonomic impairment that is ultimately fatal. Pathogenic mutations in the transthyretin (TTR) protein lead to destabilization of its tetrameric structure and subsequent formation of amyloid aggregates. Tafamidis is a small-molecule inhibitor that binds selectively to TTR in human plasma and kinetically stabilizes the tetrameric structure of both wild-type TTR and a number of different mutants. Clinical trials indicate that tafamidis slows disease progression in patients with TTR-FAP and reduces the burden of disease, demonstrating improvement in small and large nerve fiber function, modified body mass index and lower extremity neurological examination. Tafamidis meglumine has been launched for TTR FAP in the EU, Japan, Argentina, Malta and Mexico, and is preregistration in the US for this indication. NCATS
Drug Pricing (per unit)
Note: This drug pricing data is preliminary, incomplete, and may contain errors.
| Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
|---|
| Organization | Org Type | FDA approvals | Clinical Trials involvement | Org ID | Force Sort |
|---|
Amyloid Neuropathies, Familial (approved 2011)
Amyloid (Phase 3)
Amyloid Neuropathies, Familial (Phase 2/Phase 3)
Amyloidosis (Phase 4)
Cardiomyopathies (Phase 4)
Healthy Volunteers (Phase 1)
Liver Diseases (Phase 3)
Osteoarthritis, Knee (Phase 1/Phase 2)
Polyradiculoneuropathy (Phase 4)
| Trial | Phase | Start Date | Organizations | Indications |
|---|
Feedback
Data collection and curation is an ongoing process for CDEK - if you notice any information here to be missing or incorrect, please let us know! When possible, please include a source URL (we verify all data prior to inclusion).
Report issue
