Zilganersen Medicine 04 December 2025 Extended indication Zilganersen monotherapy for treatment of Alexander disease in children, adolescents, adults. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Zilganersen Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Neurological disorders other Extended indication Zilganersen monotherapy for treatment of Alexander disease in children, adolescents, adults. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Ionis Portfolio holder Mechanism of action Antisense oligonucleotide Route of administration Intrathecal Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP Submission date June 2026 Expected Registration July 2027 Orphan drug Yes 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 Er zijn op dit moment nog geen resultaten bekend. Duration of treatment Frequency of administration Dosage per administration References NCT04849741 Additional remarks Expected patient volume per year Patient volume 6 - 7 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Messing et al. J Neurosci. 2012. Additional remarks Met een prevalentie van 1 op de 2,7 miljoen zijn er zes à zeven mensen in Nederland die de ziekte van Alexander hebben. Expected cost per patient per year Cost References Additional remarks Er is nog niets bekend over de mogelijke prijs. 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