Extended indication Haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)
Therapeutic value No judgement
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 Intravenous drip
Budgetting framework Intermural (MSZ)
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
Submission date 2020
Expected Registration 2021
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Verwachte primary completion date NCT03205995 in februari 2020.

Therapeutic value

Therapeutic value No judgement
Frequency of administration 1 times a day
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.

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

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.