Briquilimab Medicine 03 June 2025 Extended indication Briquilimab monotherapy for treatment of Severe combined immunodeficiency syndrome in infants, toddlers, children, adolescents, adults and elderly undergoing blood stem cell transplantation. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Briquilimab Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Briquilimab monotherapy for treatment of Severe combined immunodeficiency syndrome in infants, toddlers, children, adolescents, adults and elderly undergoing blood stem cell transplantation. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Jasper Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Proto oncogene protein c-kit inhibitor. Fabrikant: Jasper Therapeutics Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date September 2025 Expected Registration October 2026 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn 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 Duration of treatment Frequency of administration Dosage per administration References NCT02963064 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet Additional remarks De incidentie van SCID is volgens orphanet 1 op de 50.000 geboortes. Dit betekent dat er gemiddeld 3 kinderen per jaar geboren zullen worden. 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