Extended indication Hyperargininaemia
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Pegzilarginase
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Other metabolism and Endocrinology
Extended indication Hyperargininaemia
Manufacturer Aeglea
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments Arginase replacement. Intraveneuze of subcutane toediening.

Registration

Registration route Centralised (EMA)
ATMP No
Submission date August 2022
Expected Registration September 2023
Orphan drug Yes
Registration phase Registration application pending
Additional comments Estimated primary completion datum fase 3 studie in augustus 2024.

Therapeutic value

Current treatment options Bloedtransfusie kan de arginaseactiviteit beïnvloeden.
Therapeutic value No estimate possible yet
Substantiation Het lijkt effectief in het verminderen van de arginine plasmawaarden (in ieder geval de eerste weken). Het is echter nog onzeker of er ook toegevoegde waarde is klinisch (en of het kosteneffectief zal zijn). Ook is het nog onbekend wat de langere termijn effecten zijn. Het lijkt vooralsnog dat er relatief veel bijwerkingen zijn.
References NCT03921541
Additional comments After completion of 24 weeks DB treatment, weekly IV infusions of pegzilarginase plus individualized disease management for an additional 150 weeks, with the option to receive treatment by SC after 8 weeks of the LTE study.

Expected patient volume per year

Patient volume

< 17

Market share is generally not included unless otherwise stated.

References Orphanet (1);
Additional comments Arginase deficiëntie is een zeldzame aandoening in Nederland. De ziekte komt voor bij ongeveer 1 op de miljoen personen (1). Dit betekent dat er in theorie 17 patiënten in aanmerking 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.