Nomacopan

Extended indication	Paediatric (aged 6 months till18 years) Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
Therapeutic value	No estimate possible yet
Total cost	4,500,000.00
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 till18 years) Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
Manufacturer	Akari
Mechanism of action	Complement inhibitor
Budgetting framework	Intermural (MSZ)
Additional remarks	Fabrikant AKARI Therapeutics

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	2025
Expected Registration	2026
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.

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

Cost	250,000.00 - 350,000.00
References	Horizonscan (1)
Additional remarks	Er is nog niks bekend over de prijs van nomacopan. De prijs van andere complementsysteemremmers (pegcetacoplan, zilucoplan) zit in de range van €250.000-€350.000 p.p.p.j. (1).

Potential total cost per year

Total cost	4,500,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Total cost

4,500,000.00

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

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.