Narsoplimab

Active substance
Narsoplimab
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Stem cell transplants
Extended indication
Haematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)

1. Product

Manufacturer
Omeros
Mechanism of action
Complement inhibitor
Route of administration
Intravenous
Therapeutical formulation
Intravenous drip
Budgetting framework
Intermural (MSZ)
Additional comments
Humaan monoclonaal antilichaam gericht tegen mannan-binding lectin-associated serine protease-2 (MASP-2), onderdeel van het complementsysteem.

2. Registration

Registration route
Centralised (EMA)
Type of trajectory
Normal trajectory
Submission date
2020
Expected Registration
2021
Orphan drug
Yes
Registration phase
Clinical trials
Additional comments
Verwachte primary completion date NCT03205995 in februari 2020.

3. Therapeutic value

Therapeutic value
No judgement
Duration of treatment
Not found
Frequency of administration
1 times a day
References
NCT03205995
Additional comments
Intraveneuze oplaaddosis gevolgd door dagelijkse subcutane injecties.

4. 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 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.

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

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

9. Other information

There is currently no futher information available.