Nomacopan Medicine 05 December 2023 Extended indication Paediatric (aged 6 months till18 years) Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy. Therapeutic value No estimate possible yet Total cost € 4,500,000.00 Registration phase Clinical trials Product Active substance Nomacopan Domain Hematology Reason of inclusion New medicine (specialité) Main indication Stem cell transplants Extended indication Paediatric (aged 6 months till18 years) Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Akari Portfolio holder Mechanism of action Complement inhibitor Route of administration Unknown Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Fabrikant AKARI Therapeutics Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date 2025 Expected Registration 2026 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks NCT04784455 Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De verwachting volgens experts is dat nomacopan potentieel waardevol kan zijn. Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume 15 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Record: Narsoplimab;Expertopinie; Gavriilaki et al. Clin Transplant. 2018 Sep;32(9):e13371 Additional remarks Jaarlijks worden in Nederland circa 600 allogene stamceltransplantaties uitgevoerd. De geschatte incidentie van TMA bij deze patiënten ligt rond de 15%. Er zijn onder kinderen ongeveer 80 allogene stamceltransplantaties per jaar. Dit betekent dat er ongeveer 15 patiënten in aanmerking zullen komen. Expected cost per patient per year Cost € 250,000.00 - 350,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Horizonscan (1) Additional remarks Er is nog niks bekend over de prijs van nomacopan. De prijs van andere complementsysteemremmers (pegcetacoplan, zilucoplan) zit in de range van €250.000 tot €350.000 per patiënt per jaar (1). Potential total cost per year Total cost € 4,500,000.00 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