Pozelimab Medicine 05 December 2023 Extended indication Treatment of patients with paroxysmal nocturnal hemoglobinuria who switch from eculizumab therapy Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Pozelimab Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Treatment of patients with paroxysmal nocturnal hemoglobinuria who switch from eculizumab therapy Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Regeneron Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Complement C5 inhibitors Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date 2024 Expected Registration 2025 Orphan drug Unknown Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks NCT05131204 Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er lopen nog diverse studies waarbij pozelimab gecombineerd wordt met eem siRNA cemdisiran. Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 80 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie (1) Additional remarks Op basis van de gebruikersaantallen van eculizumab bij PNH patiënten wordt er verwacht dat er maximaal 80 patiënten in aanmerking komen indien er substitutie plaatsvindt. Expected cost per patient per year Cost References drugs.com Additional remarks De Amerikaanse prijs voor pozelimab is (bbfg 200mg/mL) $36,460 voor 2 milliliter. De prijs in de Verenigde Staten ligt vaak hoger dan de Nederlandse prijs. 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