Expand all sections

Acoramidis

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
Manufacturer BridgeBio Pharma
Portfolio holder Bayer
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Extramural (GVS)
Centre of expertise UMCG, MUMC, Erasmus MC, UMCU
Additional remarks Werkingsmechanisme: transthyretine (TTR) stabilisator

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date February 2024
Expected Registration March 2025
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Tafamidis (hoge dosering)
Therapeutic value No estimate possible yet
Substantiation In patiënten met transthyretine amyloïde cardiomyopathie, werden significant betere uitkomsten gevonden op mortaliteit, morbiditeit en functie vergeleken met placebo.
Duration of treatment continuous
Frequency of administration 2 times a day
Dosage per administration 800 mg
References NCT03860935, https://pubmed.ncbi.nlm.nih.gov/38197816/

Expected patient volume per year

Patient volume

300

Market share is generally not included unless otherwise stated.
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
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

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.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.