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 No registration expected 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 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 Normal trajectory Particularity ATMP No Submission date Expected Registration Orphan drug No Registration phase No registration expected Reimbursement Medicine sluice Additional remarks In juli 2025 kondigde het bedrijf aan dat het de ontwikkeling op het gebied van ernstige gecombineerde immuundeficiëntie (SCID) stopzet. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De huidige conditionering protocollen voor beenmerg (BM) die nodig zijn voor hematopoëtische stamceltransplantatie (HSCT), maken gebruik van bestraling en/of chemotherapie en brengen ernstige toxiciteit met zich mee, wat resulteert in aanzienlijke morbiditeit, mortaliteit en aanzienlijke complicaties op de lange termijn. Duration of treatment Frequency of administration Dosage per administration References NCT02963064; Kwon et al. Blood. 2019; Long et al. Transplant Cell Ther. 2024; Salerno et al. CPT Pharmacometrics Syst Pharmacol. 2022. Additional remarks Expected patient volume per year Patient volume < 3 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet; Expertopinie Additional remarks De incidentie van SCID is volgens orphanet 1 op de 50.000 geboortes. Dit betekent dat er gemiddeld drie 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 Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks