Tafamidis Medicine 04 June 2026 Extended indication Tafamidis monotherapy for treatment of hereditary Transthyretin amyloid cardiomyopathy in adults. Therapeutic value Possible equal value Total cost Registration phase Registration application pending Product Active substance Tafamidis Domain Cardiovascular diseases Reason of inclusion Generic G Main indication Other medication for cardiovascular diseases Extended indication Tafamidis monotherapy for treatment of hereditary Transthyretin amyloid cardiomyopathy in adults. Current proprietary name Vyndaqel 61 (Pfizer) Already available biosimilars / generics Proprietary name Manufacturer Portfolio holder Mechanism of action Route of administration Therapeutical formulation Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Generic ATMP No Submission date August 2025 Expected Registration September 2026 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Possible equal value This assessment does not indicate any potential inclusion in the package. Substantiation generiek Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 1,000 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Grogan et al. J Card Fail. 2017 Aug;23(8):S73; GIPdatabank Additional remarks Het is nog lastig in te schatten hoeveel patiënten deze vorm van cardiomyopathie hebben. Er is grote variatie in de geschatte prevalentiecijfers. Afhankelijk van de precieze indicatie is de verwachting dat het patiëntvolume niet boven de 1.000 patiënten per jaar zal komen te liggen. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks