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Dorocubicel

Extended indication

Treatment of adult patients with haematological malignancies
Therapeutic value

No estimate possible yet
Registration phase

Registration application pending

Product

Active substance

Dorocubicel
Domain

Hematology
Reason of inclusion

New medicine (specialité)
Main indication

Stem cell transplants
Extended indication

Treatment of adult patients with haematological malignancies
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)
Type of trajectory

Accelerated assessment
Particularity

New medicine with Priority Medicines (PRIME)
ATMP

Yes
Submission date

June 2024
Expected Registration

April 2025
Orphan drug

Yes
Registration phase

Registration application pending
Additional remarks 
Verwachte registratie op basis van IHSI-inschatting.

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.