Extended indication Patients With CD19 + Acute Lymphoid Leukemia Resistant or Refractory to Therapy.
Therapeutic value No estimate possible yet
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.
Mechanism of action CAR-T therapy
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments 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)
ATMP Yes
Submission date 2023
Expected Registration 2024
Orphan drug No
Registration phase Clinical trials
Additional comments Primary completion datum fase 2 studie in maart 2022.

Therapeutic value

Therapeutic value No estimate possible yet
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.
Dosage per administration 0,4-5 × 10^6 ARI-0001 cells/kg
References NCT03144583; NCT04778579
Additional comments 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.

References NKR (1); Expert opinie (2);
Additional comments 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.