Pegzilarginase Medicine 07 December 2022 Extended indication Hyperargininaemia Therapeutic value No estimate possible yet Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Aeglea Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Arginase replacement. Intraveneuze of subcutane toediening. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date August 2022 Expected Registration September 2023 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks 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 This assessment does not indicate any potential inclusion in the package. 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. Duration of treatment Frequency of administration Dosage per administration References NCT03921541 Additional remarks 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 1 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet (1); expertise centrum (2) Additional remarks 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. In de praktijk is er slechts 1 patiënt in Nederland bekend (2). Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks