Dorocubicel Medicine 07 December 2022 Extended indication Treatment of adult patients with haematological malignancies requiring an allogeneic hematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. Therapeutic value No estimate possible yet Total cost 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 requiring an allogeneic hematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Excellthera 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. Portfoliohouder: Cordex Biologics International Ltd. 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 July 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Verwachte registratie op basis van IHSI-inschatting. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Dorocubicel (‘UM171’) is een middel wat vanuit cord blood afkomstige hematopoïetische stamcellen kan expanderen zodat ook patiënten die geen goede donor beschikbaar hebben toch in aanmerking kunnen komen voor een stamceltransplantatie. Het huidige gebruik van cord blood donoren in Nederland bij volwassenen ligt niet erg hoog, mogelijk zal het door dit middel wat kunnen toenemen. Duration of treatment Frequency of administration Dosage per administration References NCT02668315; Expertopinie Additional remarks Treatment consists of a single dose for IV infusion containing a dispersion for infusion of expanded CD34+ cells and non-expanded CD34- cells in 1 to 4 infusion bags each. The target dose is 0.4 to 7.5 x 10e6 CD34 cells/kg for the expanded CD34 cell product (dorocubicel) and ≥ 0.52 x 10e6 CD3 cells/kg for the non-expanded CD34- cell product. Expected patient volume per year Patient volume 50 - 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References EBMT database; fabrikant 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 Fabrikant Additional remarks De prijs is nog niet bekend. 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