Pegozafermine Medicine 04 December 2025 Extended indication Pegozafermin monotherapy for treatment of severe Hypertriglyceridemia in adults over 22 years of age and elderly. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Pegozafermine Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Lipid-lowering medications Extended indication Pegozafermin monotherapy for treatment of severe Hypertriglyceridemia in adults over 22 years of age and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer 89bio Portfolio holder 89bio Mechanism of action Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Fabrikant: 89bio Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP Submission date May 2026 Expected Registration June 2027 Orphan drug Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Statinen (atorvastatin, pitavastatin, rosuvastatin). Adjunctbehandeling: omega-3 meervoudig onverzadigde vetzuren (icosapent-ethyl); fibraten (bezafibraat, fenofibraat) Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De ENTRUST fase 3 studie loopt nog. Duration of treatment Average 8 week / weeks Frequency of administration Dosage per administration References NCT04541186; NCT05852431 (ENTRUST) Additional remarks Expected patient volume per year Patient volume < 2,800 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Baass et al. Cardiometabolic Health. 2024 (1); Expertopinie (2). Additional remarks De Deense incidentie van ernstige hypertriglyceridemie (TG ≥886mg/dL) is 390 per miljoen (1). In Nederland komt dit neer op ongeveer 7.000 nieuwe gevallen per jaar. Veel patiënten kunnen worden behandeld met dieet of bestaande therapieën (fibraten, omega-3). Omdat olezarsen alleen geregistreerd is voor volwassen patiënten (niet-zwangere) en er contra-indicaties zijn, zoals leverfunctiestoornissen en mogelijke trombopenie, wordt het effectieve behandelbare aantal patiënten geschat op ongeveer 40% van de 7.000 (n=2.800) (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 Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions MASH References IHSI Additional remarks Dit geneesmiddel is ook in ontwikkeling voor MASH (ENLIGHTEN trials). Registratie hiervan wordt in 2028-2029 verwacht. Other information Additional remarks