Ravulizumab Medicine 08 June 2021 Extended indication Extension of indication to include treatment of paediatric patients with paroxysmal nocturnal haemoglobinuria (PNH) for Ultomiris. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Ravulizumab Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other non-oncological hematological medications Extended indication Extension of indication to include treatment of paediatric patients with paroxysmal nocturnal haemoglobinuria (PNH) for Ultomiris. Current proprietary name Already available biosimilars / generics Proprietary name Ultomiris Manufacturer Alexion Portfolio holder Mechanism of action Complement inhibitor Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Monoclonaal antilichaam gericht tegen het C5 complement eiwit. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date January 2021 Expected Registration November 2021 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks ravulizumab is breed uitgesloten, deze indicatie zal dus ook in de sluis worden geplaatst 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 Additional remarks 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