OTL 103 Medicine 07 June 2022 Extended indication Patients with wiskott-aldrich syndrome Therapeutic value No estimate possible yet Total cost Registration phase No registration expected Product Active substance OTL 103 Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Patients with wiskott-aldrich syndrome Current proprietary name Already available biosimilars / generics Proprietary name Onbekend Manufacturer Orchard Portfolio holder Mechanism of action Autologous modified cell therapy Route of administration Unknown Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Erasmus MC Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date Expected Registration Orphan drug Unknown Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Indicatiegebied betreft een weesziekte. Kans dat het middel een weesstatus krijgt. Orchard zal de registratie niet doorzetten en het is de vraag of er een andere fabrikant geïnteresseerd zal zijn om het programma over te nemen. Er zal dit jaar geen registratie ingediend gaan worden. 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 De fase 3 trial loopt op dit moment in Milaan met 10 patiënten, open label behandeling. 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 < 85 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 5 op de 1.000.000 personen. 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks