Ravulizumab Medicine 08 June 2021 Extended indication Extension of indication to include treatment of paediatric patients with paroxysmal nocturnal haemoglobinuria (PNH) for Ultomiris (>10 kg). Therapeutic value No estimate possible yet Total cost € 1,400,000.00 Registration phase Positive CHMP opinion 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 (>10 kg). 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 December 2021 Orphan drug Yes Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in juli 2021. 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 < 5 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie Additional remarks PNH is op de kinderleeftijd erg zeldzaam. De diagnose wordt mogelijk bij minder dan 5 kinderen per jaar in Nederland gesteld. Expected cost per patient per year Cost € < 280,000.00 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 Additional remarks Voor volwassenen is behandeling met ravulizumab €280.000 in het eerste jaar en ongeveer €260.000 in de daar opvolgende jaren. De kosten zullen bij pediatrische patiënten mogelijk lager uitvallen. Potential total cost per year Total cost € 1,400,000.00 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