Valiltramiprosate Medicine 03 June 2025 Extended indication Valiltramiprosate monotherapy for treatment of homozygous APOE4/4 genotype Early onset Alzheimerʼs disease in adults over 50 years of age and elderly. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Valiltramiprosate Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Dementia Extended indication Valiltramiprosate monotherapy for treatment of homozygous APOE4/4 genotype Early onset Alzheimerʼs disease in adults over 50 years of age and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Alzheon Portfolio holder Mechanism of action Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date 2026 Expected Registration 2027 Orphan drug No Registration phase Clinical trials 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 265mg tweemaal daags References NCT04770220; NCT06304883 Additional remarks Expected patient volume per year Patient volume 11,400 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References 1-http://www.levenmetalzheimer.nl/content/alzheimer/feiten-cijfers.asp; 2- Declaratiedata 2020 (geëxtrapoleerd naar 2022); 3-Yuan J, et al. (2021) J Alzheimers Dis. 2021; doi: 4- APOE4 homozygosity represents a distinct genetic form of Alzheimer’s disease. Juan Fortea et all . 3 November 2023. Additional remarks In Nederland hebben 270.000 mensen dementie. Hiervan hebben 160.000 tot 190.000 mensen de ziekte van Alzheimer (maximaal 70%) (1). Hiervan is 80% tussen 50 en 90 jaar (2). Er zijn ongeveer net zoveel mensen met MCI (mild cognitive impairment)(3). 50% van de mensen met alzheimer heeft bij diagnose lichte alzheimer (76.000) (3). Van de algemene populatie is 15% homozygoot voor het APOE4 gen, maar bij mensen met Alzheimer komt dit in 15% van de gevallen voor(4). Als we uitgaan van 15% van de patiënten met lichte alzheimer zullen er mogelijk 11.400 patiënten in aanmerking komen. Expected cost per patient per year Cost References Additional remarks 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