Extended indication Patients With Sickle-Cell Disease
Therapeutic value Possibly no place in the treatment regimen
Registration phase Clinical trials

Product

Active substance UM171
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialité)
Main indication Other non-oncological hematological medications
Extended indication Patients With Sickle-Cell Disease
Manufacturer Stem Cell Therapeutics
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Submission date 2022
Expected Registration 2023
Orphan drug No
Registration phase Clinical trials

Therapeutic value

Therapeutic value Possibly no place in the treatment regimen
Substantiation Dit gaat om ex-vivo geëxpandeerde stamcellen uit navelstrengbloed. Onverwant navelstrengbloed is geen reguliere bron voor stamcellen bij sikkelceltransplantatie. Met de huidige technieken van HAPLO identieke transplantatie lijkt er op dit moment ook geen behoefte aan deze bron van stamcellen.
References NCT04594031

Expected patient volume per year

References cyberpolisikkelcelziekte;
Additional comments In januari 2007 werd sikkelcelziekte in het hielprikscreeningsprogramma opgenomen. Het totale aantal patiënten met sikkelcelziekte in Nederland wordt geschat op 1.000-1.500; hiervan is twee derde kind.

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.