Proteïne C Medicine 07 June 2022 Extended indication Extension of indication to include long-term prophylaxis (deletion of wording ‘short-term’ and currently listed conditions) of purpura fulminans and coumarin induced skin necrosis in patients with severe congenital protein C deficiency. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Proteïne C Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Antithrombotic medications Extended indication Extension of indication to include long-term prophylaxis (deletion of wording ‘short-term’ and currently listed conditions) of purpura fulminans and coumarin induced skin necrosis 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 February 2023 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks 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 References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References medicijnkosten Additional remarks De gemiddelde prijs per verpakking ex. btw is € 1.024. De behandelduur is nog niet bekend. 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