Acoramidis Medicine 04 June 2024 Extended indication Transthyretin amyloid cardiomyopathy Therapeutic value No estimate possible yet Total cost € 30,000,000.00 Registration phase Registration application pending Product Active substance Acoramidis Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other medication for cardiovascular diseases Extended indication Transthyretin amyloid cardiomyopathy Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer BridgeBio Pharma Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Werkingsmechanisme: prealbumin modulators Registration Registration route Centralised (EMA) Type of trajectory Adaptive pathways Particularity Unknown ATMP No Submission date February 2024 Expected Registration March 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Tafamidis (hoge dosering) Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation In patiënten met transthyretine amyloïde cardiomyopathie, werden significant betere uitkomsten gevonden op mortaliteit, morbiditeit en functie vergeleken met placebo. Duration of treatment Frequency of administration 2 times a day Dosage per administration mg References NCT03860935 Additional remarks Expected patient volume per year Patient volume 300 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expert opinie Additional remarks Er wordt verwacht dat er mogelijk rond de 300 patiënten in aanmerking kunnen komen. Acoramidis zal de concurrentie aangaan met tafamidis dat op dit moment ook voor dezelfde indicatie wordt gegeven. Expected cost per patient per year Cost € > 100,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References GIPdatabank Additional remarks Tafamidis kostte in 2023 €104.195 per patiënt. De verwachting is dat acoramidis vergelijkbaar geprijsd zal worden al zal dat afhangen van de uiteindelijke studieresultaten. Potential total cost per year Total cost € 30,000,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks