Lenzumestrocel Medicine 03 December 2024 Extended indication Lenzumestrocel in combination with riluzole for treatment of familial or sporadic amyotrophic lateral sclerosis in adults over 25 years of age and elderly. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Lenzumestrocel Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication ALS Extended indication Lenzumestrocel in combination with riluzole for treatment of familial or sporadic amyotrophic lateral sclerosis in adults over 25 years of age and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Neuronata-R Manufacturer Corestemchemon Portfolio holder Mechanism of action Route of administration Intrathecal Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Neuronata-R® (lenzumestrocel) is een autoloog beenmerg-afgeleid mesenchymale stamcel (BM-MSC) product. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP Yes Submission date August 2026 Expected Registration September 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Riluzol verlengt de overleving van mensen met ALS, maar geneest het niet. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er zijn nog geen fase 3-resultaten bekend. Duration of treatment Frequency of administration Dosage per administration References NCT01363401 (1); NCT04745299 (ALSummit) (2) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Stichting ALS Nederland. 2024 (1). Additional remarks In Nederland leven zo'n 1.500 mensen met amyotrofische laterale sclerose (ALS) (1). Expected cost per patient per year Cost € 1,000,000.00 - 3,000,000.00 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. References Additional remarks De prijs is nog niet bekend. Mogelijk zal lenzumestrocel in dezelfde categorie vallen als Libmeldy en Zolgensma (ATMP + weesgeneesmiddel). Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks