L-acetylleucine Medicine 04 June 2026 Extended indication Levacetylleucine monotherapy for treatment of Ataxia telangiectasia in children over 4 years of age, adolescents, adults and elderly Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance L-acetylleucine Domain Neurological disorders Reason of inclusion Indication extension IND Main indication Neurological disorders other Extended indication Levacetylleucine monotherapy for treatment of Ataxia telangiectasia in children over 4 years of age, adolescents, adults and elderly Current proprietary name Already available biosimilars / generics Proprietary name Aqneursa Manufacturer Intra-Bio Portfolio holder Intra-Bio Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Granulate Budgetting framework Extramural (GVS) Centre of expertise Radboudumc Additional remarks Werkingsmechanisme: Calcium channel modulators Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP Submission date June 2026 Expected Registration June 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 is geen behandeling die Ataxia Telangiectasia kan genezen. De behandeling is er op gericht de symptomen van de ziekte zo veel mogelijk te onderdrukken of om het kind er zo goed mogelijk mee te leren om gaan (1) Duration of treatment Frequency of administration Dosage per administration References (1) https://twanfoundation.nl/assets/uploads/files/twanfoundation-brochuredigitaal.pdf (2) NCT03759678, NCT06673056 (IB1001-303) Additional remarks Expected patient volume per year Patient volume 35 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://twanfoundation.nl/assets/uploads/files/twanfoundation-brochuredigitaal.pdf Additional remarks In Nederland zijn er ongeveer 35 patiënten met AT. 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