Rusfertide Medicine 03 December 2024 Extended indication Polycythemia Vera Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Rusfertide Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Polycythemia Vera Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Protagonist Portfolio holder Mechanism of action Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Fabrikant: Protagonist Therapeutics. Hepcidin replacements; Iron modulators. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date December 2025 Expected Registration December 2026 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Indieningsdatum en verwachte registratie op basis van IHSI-inschatting. Therapeutic value Current treatment options Celgroei remmende middelen: hydroxycarbamide (Hydrea®), peginterferon-alfa-2a (Pegasys®) of Ruxolitinib (Jakavi®) (2). Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT05210790 (VERIFY) (1); Achtergronden bij de polycythemia vera (PV)-richtlijn 2019, Nederlands Tijdschrift voor Hematologie (2019,16:123-130) (2) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Polycythemia vera. MPN stichting. 2024 (1). Additional remarks Polycythemia vera is een zeldzame bloedziekte en komt naar schatting voor bij ongeveer 2,3 per 100.000 mensen (1). 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 Indication extensions References Additional remarks Other information Additional remarks