Alirocumab Medicine 06 June 2023 Extended indication Extension of indication to include treatment of paediatric patients 8 years of age and older with heterozygous familial hypercholesterolemia (HeFH) as an adjunct to diet, alone or in combination with other LDL -C lowering therapies. Therapeutic value Possible added value Total cost € 733,375.00 Registration phase Registered and reimbursed Product Active substance Alirocumab Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Lipid-lowering medications Extended indication Extension of indication to include treatment of paediatric patients 8 years of age and older with heterozygous familial hypercholesterolemia (HeFH) as an adjunct to diet, alone or in combination with other LDL -C lowering therapies. Current proprietary name Already available biosimilars / generics Proprietary name Praluent Manufacturer Sanofi Portfolio holder Sanofi Mechanism of action Enzyme inhibitor Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks PCSK9 remmer Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date March 2023 Expected Registration November 2023 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Registratie november 2023 Therapeutic value Current treatment options Evolocumab is geregistreerd voor kinderen met HeFH vanaf 10 jaar. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Alirocumab heeft therapeutische meerwaarde voor kinderen met HeFH die met maximale orale lipide verlagende medicatie zijn behandeld. Dat alirocumab in twee doseringen beschikbaar is kan voordelig zijn. Duration of treatment Frequency of administration 1 times every 2 weeks Dosage per administration 75 mg or 150 mg (bodyweight <50 kg/≥50 kg) References Bruckert E, Caprio S, Wiegman A, Charng MJ, Zárate-Morales CA, Baccara-Dinet MT, Manvelian G, Ourliac A, Scemama M, Daniels SR. Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study. Arterioscler Thromb Vasc Biol. 2022 Dec;42(12):1447-1457. doi: 10.1161/ATVBAHA.122.317793. Epub 2022 Nov 3. PMID: 36325897; PMCID: PMC9750107. NCT03510715 Additional remarks Expected patient volume per year Patient volume 50 - 200 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Mach F, Baigent C, Catapano AL, Koskinas KC, Casula M, Badimon L, Chapman MJ, De Backer GG, Delgado V, Ference BA, et al.; ESC Scientific Document Group. 2019 esc/eas guidelines for the management of dyslipidaemias: Lipid modification to reduce cardiovascular risk. Eur Heart J. 2020;41:111–188. doi: 10.1093/eurheartj/ehz455 Additional remarks De prevalentie ligt tussen de 1 op 160.000 tot 1 op 320.000. Het gaat om een klein aantal patiënten die niet effectief behandeld kunnen worden met orale middelen. Aangezien ook evolocumab al beschikbaar is vanaf 10 jaar zal het uiteindelijke patiëntvolume waarschijnlijk dichter bij de 50 patiënten liggen. Expected cost per patient per year Cost € < 5,867.00 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 medicijnkosten.nl Additional remarks Voor alirocumab is een financieel arrangement van toepassing tot 1 januari 2027. Gemiddelde prijs per 1 wegwerpspuit Praluent, 1ml injectievloeistof 150 mg/ml €225,66 inclusief btw. Als uitgegaan wordt van een patiënt van boven de 75kg elke twee weken leidt dit tot een bedrag van € 5.867 per jaar per patiënt. Potential total cost per year Total cost € 733,375.00 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