UM171 Medicine 07 December 2021 Extended indication Patients With Sickle-Cell Disease Therapeutic value Possibly no place in the treatment regimen Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Stem Cell Therapeutics Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date 2024 Expected Registration 2025 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Possibly no place in the treatment regimen This assessment does not indicate any potential inclusion in the package. 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. Duration of treatment Frequency of administration Dosage per administration References NCT04594031 Additional remarks Expected patient volume per year Patient volume 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Cyberpolisikkelcelziekte (1); Expertopinie (2) Additional remarks 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 (1). De inschatting is dat dit geneesmiddel in Nederland voor maximaal circa 10 patiënten per jaar een optie zou kunnen zijn. Dit ook omdat het gebruik van HAPLO donoren toeneemt (2). 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 Unknown Indication extensions References Additional remarks Other information Additional remarks