Evinacumab

Extended indication	Extension of indication for EVKEEZA to include the treatment of paediatric patients with homozygous familial hypercholesterolaemia aged 6 months to less than 5 years
Therapeutic value	No estimate possible yet
Registration phase	Registration application pending

Product

Active substance	Evinacumab
Domain	Cardiovascular diseases
Reason of inclusion	Indication extension
Main indication	Lipid-lowering medications
Extended indication	Extension of indication for EVKEEZA to include the treatment of paediatric patients with homozygous familial hypercholesterolaemia aged 6 months to less than 5 years
Proprietary name	Evkeeza
Manufacturer	Ultragenyx
Route of administration	Intravenous
Therapeutical formulation	Intravenous drip
Budgetting framework	Intermural (MSZ)

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

Therapeutic value	No estimate possible yet

There is currently nothing known about the expected patient volume.

Cost	< 100,000.00
References	https://www.forbes.com/sites/johnlamattina/2019/08/15/regenerons-great-science-led-to-an-important-drug-but-will-it-flop-commercially/#70369e62653b
Additional remarks	De exacte prijs is nog niet bekend gemaakt door de fabrikant. In de Verenigde Staten kost LDL-aferese $8,000 per maand ($96,000 per jaar). De verwachting is dan ook dat de prijs minimaal $100,000 op jaarbasis zal gaan bedragen. Dit middel is in de sluis geplaatst tot er een financieel arrangement is overeengekomen.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.