ARI-0001 Medicine 07 December 2022 Extended indication Patients With CD19 + Acute Lymphoid Leukemia Resistant or Refractory to Therapy. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance ARI-0001 Domain Hematology Reason of inclusion New medicine (specialité) Main indication ALL Extended indication Patients With CD19 + Acute Lymphoid Leukemia Resistant or Refractory to Therapy. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Portfolio holder Mechanism of action CAR-T therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Adult differentiated autologous T-cells from peripheral blood, expanded and transducted with a lentivirus to express a chimeric antigen receptor with anti-CD19 specificity [A3B1] conjugated to the 4-aBB and CD3z co-stimulatory regions. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date 2023 Expected Registration 2024 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Primary completion datum fase 2 studie in maart 2022. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Patiënten worden behandeld met op de zorglocatie geproduceerde CAR T-cellen. Registratie op basis van fase 2 studie (Prime). In de fase 2 studie heeft 79% eerder HSCT gehad. De mediane PFS is 7,83 maanden en de 2-yr PFS is 38%, mediane OS is NR en 2-yr OS is 53% (n=3). Het geneesmiddel lijkt vooralsnog effectief te zijn. Alle patiënten in de klinische studie hebben al een allogene stamceltransplantatie gehad en blinotumumab, er wordt een goede respons waargenomen. Duration of treatment Frequency of administration Dosage per administration 0,4-5 × 10^6 ARI-0001 cells/kg References NCT03144583; NCT04778579 Additional remarks Initially as a single dose and later split into 3 fractions (10%, 30% and 60%) with full administration depending on the absence of cytokine release syndrome (CRS). Expected patient volume per year Patient volume 10 - 15 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR (1); Expert opinie (2); Additional remarks Geschatte aantallen op basis van NKR gegevens over 2017-2018 waren er 180 patiënten met ALL per jaar (1). Er wordt verwacht dat er voor deze behandeling 10 tot 15 patiënten in aanmerking zullen komen afhankelijk van de uiteindelijke lijn en voorbehandeling (2). 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks