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

Product

Active substance UM171
Domain Hematology
Reason of inclusion New medicine (specialité)
Main indication Stem cell transplants
Extended indication Allogeneic haematopoietic stem cell transplants
Manufacturer Stem Cell Therapeutics
Mechanism of action Gene therapy
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments Dit gaat om ex-vivo geëxpandeerde stamcellen uit navelstrengbloed.

Registration

Registration route Centralised (EMA)
ATMP Yes
Submission date 2023
Expected Registration 2024
Orphan drug No
Registration phase Clinical trials

Therapeutic value

Therapeutic value No estimate possible yet

Expected patient volume per year

References EBMT database.
Additional comments 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.

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.