Proteïne C Medicine 07 June 2022 Extended indication Ceprotin is indicated for prophylaxis and treatment of purpura fulminans, coumarin‑induced skin necrosis and venous thrombotic events in patients with severe congenital protein C deficiency. Therapeutic value No estimate possible yet Total cost € 80,380.00 Registration phase Registered and reimbursed Product Active substance Proteïne C Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Antithrombotic medications Extended indication Ceprotin is indicated for prophylaxis and treatment of purpura fulminans, coumarin‑induced skin necrosis and venous thrombotic events in patients with severe congenital protein C deficiency. Current proprietary name Already available biosimilars / generics Proprietary name Ceprotin Manufacturer Takeda Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date April 2022 Expected Registration December 2022 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in november 2022 Therapeutic value Current treatment options 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 200 ie/kg References Additional remarks Expected patient volume per year Patient volume < 5 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References A Minford et al. Diagnosis and management of severe congenital protein C deficiency (SCPCD): Communication from the SSC of the ISTH. Thromb Haemost. 2022 (1); Additional remarks Ernstige proteïne C deficiëntie komt voor bij 1 op de 4 miljoen(1), maar mogelijk ligt het aantal nog lager vanwege vroege vruchtdood in de algehele populatie. Deze patiënten met ernstige proteïne C deficiëntie hebben risico op purpura fulminans. Expected cost per patient per year Cost € 16,076.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References medicijnkosten.nl, CBS Additional remarks De gemiddelde prijs per verpakking exclusief btw (500IE + SOLV 5ML) is €1.024. Uitgaande van een behandelduur van een jaar 200 IE/kg per 48 uur zou dat bij een gemiddeld gewicht van 78,5 neerkomen op €16.076,80 Potential total cost per year Total cost € 80,380.00 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