Extended indication Haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).
Therapeutic value No estimate possible yet
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).
Manufacturer Omeros
Mechanism of action Complement inhibitor
Route of administration Intravenous
Therapeutical formulation Injection / infusion
Budgetting framework Intermural (MSZ)
Additional comments 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

Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials
Additional comments In de Verenigde Staten heeft narsoplimab een FDA Breakthrough designation ontvangen. In de EU heeft narsoplimab een weesgeneesmiddelstatus ontvangen.

Therapeutic value

Therapeutic value No estimate possible yet
Frequency of administration 1 times a week
References NCT03205995.
Additional comments Intraveneuze oplaaddosis gevolgd door dagelijkse subcutane injecties.

Expected patient volume per year

Patient volume

< 25

Market share is generally not included unless otherwise stated.

References Expertopinie; Gavriilaki et al. Clin Transplant. 2018 Sep;32(9):e13371
Additional comments 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions aHUS; IgA Nephropathy (IgAN); Lupus Nephritis & Other Renal Diseases.
References Omeros pipeline.

Other information

There is currently no futher information available.