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ECT-001-CB

Extended indication Allogeneic haematopoietic stem cell transplants
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance ECT-001-CB
Domain Hematology
Reason of inclusion New medicine (specialité)
Main indication Stem cell transplants
Extended indication Allogeneic haematopoietic stem cell transplants
Manufacturer ExCellThera
Mechanism of action Gene therapy
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks Dit gaat om ex-vivo geëxpandeerde stamcellen uit navelstrengbloed.

Registration

Registration route Centralised (EMA)
ATMP Yes
Submission date 2024
Expected Registration 2025
Orphan drug Yes
Registration phase Clinical trials
Additional remarks tijdslijn op basis van IHSI data

Therapeutic value

Therapeutic value No estimate possible yet
References NCT02668315
Additional remarks Transplantation of cord blood expanded with UM171 Prethaw CB cell count prior to manipulation: CD34+ cell count 1.0-4.9 x 10E5/kg and TNC superior or equal to 2.0 x 10E7/kg.

Expected patient volume per year

Patient volume

< 30

Market share is generally not included unless otherwise stated.
References EBMT database.
Additional remarks Jaarlijks worden in Nederland circa 600 allogene stamceltransplantaties uitgevoerd. De indicatie betreft allogene stamceltransplantatie specifiek uit navelstrengbloed, dit is maar een klein deel (enkele tientallen) van het totale aantal allogene stamceltransplantaties. Of al deze patiënten dan in aanmerking komen voor expansie is nog niet in te schatten.

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

Indication extension Yes
Indication extensions Phase II: Acute myeloid leukaemia; Haematological malignancies; Lymphoid leukaemia; Myelodysplastic syndromes Phase I/II: Multiple myeloma Phase I: Sickle cell anaemia
References Adis Insight

Other information

There is currently no futher information available.