Giroctocogene fitelparvovec

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
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
Additional remarks	Werkingsmechanisme: prealbumin modulators

Registration

Registration route	Centralised (EMA)
Type of trajectory	Adaptive pathways
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	mg
References	NCT03860935

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.

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.

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.

Acoramidis