Extended indication

Extension of indication to include treatment of adult patients with Paroxysmal Nocturnal Hemoglobinu

Therapeutic value

No estimate possible yet

Registration phase

Registration application pending

Product

Active substance

Pegcetacoplan

Domain

Cardiovascular diseases

Reason of inclusion

Indication extension

Main indication

Other non-oncological hematological medications

Extended indication

Extension of indication to include treatment of adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not previously treated with a complement inhibitor

Proprietary name

Aspaveli

Manufacturer

Sobi

Mechanism of action

Complement inhibitor

Budgetting framework

Extramural (GVS)

Registration

Registration route

Centralised (EMA)

Type of trajectory

Normal trajectory

ATMP

No

Submission date

April 2023

Expected Registration

February 2024

Orphan drug

Yes

Registration phase

Registration application pending

Therapeutic value

Therapeutic value

No estimate possible yet

Expected patient volume per year

There is currently nothing known about the expected patient volume.

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.