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 Europa 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.