Narsoplimab Medicine 10 December 2019 Extended indication Haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Narsoplimab Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Stem cell transplants Extended indication Haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Omeros Portfolio holder Mechanism of action Complement inhibitor Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Humaan monoclonaal antilichaam gericht tegen mannan-binding lectin-associated serine protease-2 (MASP-2), onderdeel van het complementsysteem. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date 2020 Expected Registration 2021 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Verwachte primary completion date NCT03205995 in februari 2020. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration References NCT03205995 Additional remarks Intraveneuze oplaaddosis gevolgd door dagelijkse subcutane injecties. Expected patient volume per year Patient volume < 25 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie; Gavriilaki et al. Clin Transplant. 2018 Sep;32(9):e13371; Sakellari et al. Blood 2016 128:982; Additional remarks Jaarlijks worden in Nederland circa 600 allogene stamceltransplantaties uitgevoerd. De geschatte incidentie van TMA bij deze patiënten ligt rond de 15% (90 patiënten). Het inclusiecriterium van de trial is vanaf 12 jaar. Heel beperkte groep die uiteindelijk in aanmerking zal komen, verwachting is minder dan 25 patiënten op jaarbasis. 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 Yes Indication extensions aHUS; IgA Nephropathy (IgAN); Lupus Nephritis & Other Renal Diseases References Omeros pipeline Additional remarks Other information Additional remarks