Marnetegragene autotemcel Medicine 10 June 2020 Extended indication Marnetegragene autotemcel monotherapy for treatment of severe leukocyte adhesion deficiency type I in infants and toddlers, children, adolescents, adults, and elderly for whom autologous haematopoietic stem cell transplant (allo-HSCT) is appropriate and a human leukocyte antigen (HLA)-identical sibling donor transplant is not available. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Marnetegragene autotemcel Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Marnetegragene autotemcel monotherapy for treatment of severe leukocyte adhesion deficiency type I in infants and toddlers, children, adolescents, adults, and elderly for whom autologous haematopoietic stem cell transplant (allo-HSCT) is appropriate and a human leukocyte antigen (HLA)-identical sibling donor transplant is not available. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Rocket Portfolio holder Mechanism of action Gene therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Hematopoëtische cel gebaseerde gentherapie bestaande uit autologe CD34+ verrijkte cellen getransduceerd met de therapeutische lentivirale vector, Chim-CD18-WPRE, RP-L201. Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date July 2026 Expected Registration August 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Symptomatische behandelingen zijn onder andere hygiënische maatregelen, levenslange antibiotische profylaxe en tijdige en adequate behandeling van infecties. De enige genezende behandeling is een beenmergtransplantatie. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation LAD-1 is een zeer zeldzame aandoening. Op basis van de de data is dit een veelbelovend middel. Echter is de data op dit moment beperkt om een definitieve uitspraak te doen over de plaats in de behandeling. Duration of treatment Frequency of administration Dosage per administration References NCT03812263; NCT03825783; Booth et al. J Clin Imm. 2023; Expertopinie. Additional remarks Expected patient volume per year Patient volume < 5 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://ghr.nlm.nih.gov/condition/leukocyte-adhesion-deficiency-type-1#statistics Additional remarks Er zijn mondiaal tenminste 300 gevallen van deze ziekte bekend. Het patiëntvolume in Nederland zal niet meer dan vijf patiënten bedragen. Expected cost per patient per year Cost References Holovina A. Cost of bone marrow transplantation and where is it performed. MediGlobus. 30 March 2018. Accessed 9 March 2023. https://mediglobus.com/cost-ofbone-marrow-transplantation-and-where-is-itperformed/. Additional remarks In Europa kost een allo-HSCT ongeveer €120.000 tot €200.000. Als het geregistreerd wordt zal RP-L201 substantieel duurder zijn. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks