Evinacumab Medicine 04 June 2024 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 Total cost Registration phase Registration application pending Product Active substance Evinacumab Domain Cardiovascular diseases Reason of inclusion Indication extension IND 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 Current proprietary name Already available biosimilars / generics Proprietary name Evkeeza Manufacturer Ultragenyx Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date April 2024 Expected Registration February 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks 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 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. Potential total cost per year Total cost 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