Etuvetidigene autotemcel Medicine 07 June 2022 Extended indication Waskyra is indicated for the treatment of patients aged 6 months and older with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom haematopoietic stem cell (HSC) transplantation is appropriate and no suitable human leukocyte antigen (HLA)-matched related haematopoietic stem cell donor is available. Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Etuvetidigene autotemcel Domain Hematology Reason of inclusion New medicine (specialité) Main indication Stem cell transplants Extended indication Waskyra is indicated for the treatment of patients aged 6 months and older with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom haematopoietic stem cell (HSC) transplantation is appropriate and no suitable human leukocyte antigen (HLA)-matched related haematopoietic stem cell donor is available. Current proprietary name Already available biosimilars / generics Proprietary name Waskyra Manufacturer Fondazione Telethon Portfolio holder Fondazione Telethon Mechanism of action Autologous modified cell therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Erasmus MC Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP Yes Submission date January 2025 Expected Registration January 2026 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Rejected Additional remarks Positieve CHMP-opinie in november 2025. Therapeutic value Current treatment options Antibiotica en immunoglobuline, bloedtransfusies, immunosuppressiva, beenmergtransplantatie. 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 NCT03837483 (Fase III) https://doi.org/10.1016/S2352-3026(19)30066-3 (fase I/II) Additional remarks Expected patient volume per year Patient volume < 45 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.erasmusmc.nl/nl-nl/patientenzorg/aandoeningen/wiskott-aldrich-syndroom Additional remarks Wiskott-Aldrich syndrome (WAS) is extreem zeldzaam en komt voor in ongeveer vijf op de 1.000.000 personen (voornamelijk jongens). Expected cost per patient per year Cost References Additional remarks In verband met kleine aantal patiëntengroepen, unmet need en aard van de therapie worden mogelijk hoge kosten verwacht. 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