Tafamidis

Orphan DrugFDA Approved

Description

Tafamidis is a transthyretin stabilizer approved for transthyretin amyloid cardiomyopathy. It works by stabilizing the tetrameric form of transthyretin protein, preventing its dissociation and subsequent amyloid formation. While specifically approved for ATTR amyloidosis, it represents a therapeutic approach for protein misfolding disorders.

Indications & Therapeutic Use

Transthyretin amyloid cardiomyopathy, transthyretin amyloid polyneuropathy

Global Availability (9 countries)

CountryAccess RouteRegulatory PathwayLead TimeStatus

Available Through

This drug is available for procurement through the following fulfilment partner.

Fulfilment Partner
Tafamidis
Generic NameTafamidis
Brands1 brand available
Active IngredientTafamidis meglumine
Drug ClassTransthyretin amyloid cardiomyopathy
ManufacturerPfizer
Dosage FormsOral capsule 20mg, 61mg
Medical CodeN07XX08
Orphan StatusYes — Orphan Drug
Cold ChainNot Required
Lead Time10 days
Reg. StatusFDA Approved
Clinical TrialNCT01994889
Countries9 countries
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations10 Validated Nodes