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Nomacopan

Extended indication Paediatric (aged 6 months-18 years) Haematopoietic Stem-Cell Transplant Associated Thrombotic Microa
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Nomacopan
Domain Hematology
Reason of inclusion New medicine (specialité)
Main indication Stem cell transplants
Extended indication Paediatric (aged 6 months-18 years) Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
Manufacturer Akari
Budgetting framework Intermural (MSZ)
Additional remarks Fabrikant AKARI Therapeutics

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date February 2024
Expected Registration March 2025
Orphan drug Yes
Registration phase Clinical trials
Additional remarks NCT04784455

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation De verwachting volgens experts is dat nomacopan potentieel waardevol kan zijn.

Expected patient volume per year

Patient volume

15

Market share is generally not included unless otherwise stated.
References Record: Narsoplimab;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%. Er zijn onder kinderen ongeveer 80 allogene stamceltransplantaties per jaar. Dit betekent dat er ongeveer 15 patiënten in aanmerking zullen komen.

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.