Narsoplimab Medicine 10 December 2019 Extended indication Haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Therapeutic value No estimate possible yet 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 Injection / infusion Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Narsoplimab is a human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system. The lectin pathway is one of the principal pathways of complement and is activated primarily by tissue damage and microbial infection. Importantly, inhibition of MASP-2 does not appear to interfere with the classical complement pathway, a critical component of the acquired immune response to infection. Registration Registration route Unknown Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date 2021 Expected Registration 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks In de Verenigde Staten heeft narsoplimab een FDA Breakthrough designation ontvangen. In de EU heeft narsoplimab een weesgeneesmiddelstatus ontvangen. 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 1 times a week 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 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