Glucarpidase Medicine 08 December 2020 Extended indication The treatment of adults and children (from 28 days of age) at risk of methotrexate toxicity due to delayed methotrexate elimination. Therapeutic value Possible added value Total cost € 1,250,000.00 Registration phase Registered Product Active substance Glucarpidase Domain Oncology Reason of inclusion New medicine (specialité) Main indication Oncology other Extended indication The treatment of adults and children (from 28 days of age) at risk of methotrexate toxicity due to delayed methotrexate elimination. Current proprietary name Already available biosimilars / generics Proprietary name Voraxaze Manufacturer Ohara Pharmaceuticals Portfolio holder Mechanism of action Other, see general comments Route of administration Intravenous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Recombinant carboxypeptidase G2 enzyme that hydrolyzes antifolates such as, methotrexate to inactive metabolites. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date August 2020 Expected Registration January 2022 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie ontvangen in november 2021 voor markt autorisatie onder exceptionele omstandigheden. Therapeutic value Current treatment options Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Glucarpidase wordt in Nederland al incidenteel off-label naast leucovorin gegeven, meestal in geval van MTX intoxicaties. Het zorgt voor een sterke daling van de methotrexaat spiegel. Duration of treatment Frequency of administration Dosage per administration References NCT00481559; NCT01305655. Additional remarks Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NCT03684980; NCT03960177. Additional remarks De verwachting is dat dit geneesmiddel in een zeer beperkte populatie ingezet zal worden. Dit zullen op jaarbasis waarschijnlijk niet meer dan 10 patiënten zijn. Expected cost per patient per year Cost € 100,000.00 - 150,000.00 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 Comparable Efficacy With Varying Dosages of Glucarpidase in Pediatric Oncology Patients. Jeffrey R. Scott. 2015., drugs.com Additional remarks De prijs in de Verenigde Staten is $27.000 per 1.000 unit vial. Dit betekent dat een patiënt van 70kg (vier vials voor 3,500 units) meer dan $100.000 kost volgens het toedieningsschema van de FDA. Daarbij zijn er ook andere bronnen die een prijs van $38,599.84 aangeven per 1.000 unit vial. Inschatting gezien bovenstaand tussen de €100.000 en €150.000. Potential total cost per year Total cost € 1,250,000.00 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