ECT-001-CB Medicine 07 December 2022 Extended indication Allogeneic haematopoietic stem cell transplants Therapeutic value No estimate possible yet Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Stem Cell Therapeutics Portfolio holder Mechanism of action Gene therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Dit gaat om ex-vivo geëxpandeerde stamcellen uit navelstrengbloed. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date 2023 Expected Registration 2024 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration 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. Maximum patient volume for sluice 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 Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions 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 Additional remarks Other information Additional remarks