Atidarsagene autotemcel Medicine 03 June 2025 Extended indication Atidarsagene autotemcel monotherapy for treatment of late juvenile metachromatic leukodystrophy in children and adolescents with molecular diagnosis of MLD. Therapeutic value No estimate possible yet Total cost € 18,500,000.00 Registration phase Clinical trials Product Active substance Atidarsagene autotemcel Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Metabolic diseases Extended indication Atidarsagene autotemcel monotherapy for treatment of late juvenile metachromatic leukodystrophy in children and adolescents with molecular diagnosis of MLD. Current proprietary name Already available biosimilars / generics Proprietary name Lenmeldy Manufacturer Kyowa Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Yes Submission date June 2025 Expected Registration January 2026 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. 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 NCT04283227 (OTL-200-07) Additional remarks Expected patient volume per year Patient volume 4 - 5 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References van Rappard, D. F. (2018). Metachromatic leukodystrophy: Natural evolution and treatment effects; Expertopinie Additional remarks Voor Nederland is de geschatte incidentie van MLD bij pasgeborenen 4 tot 5 nieuwe gevallen per jaar. Patiënten dienen behandeld te worden in een vroeg stadium, voordat de cognitieve functie is verminderd. Dit wordt vaak te laat gediagnosticeerd. Het betekent in de praktijk dat alleen juveniele en adulte patiënten voor deze behandeling in aanmerking komen. Er wordt een patiëntvolume van 10 tot 15 patiënten in de komende 3 jaar verwacht. In de toekomst is te verwachten dat er door middel van hielprikscreening mogelijk meer patiënten gevonden zullen worden. Expected cost per patient per year Cost € < 3,700,000.00 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 biospace.com Additional remarks De gentherapie atidarsagene autotemcel (Lenmeldy) heeft een prijs van $4,25 miljoen in de VS. Dit zou omgerekend naar euro €3,7 betekenen. De prijs in Nederland is echter nog niet bekend. Potential total cost per year Total cost € 18,500,000.00 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