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Glucarpidase

Voraxaze

Extended indication The treatment of adults and children (from 28 days of age) at risk of methotrexate toxicity due to d
Therapeutic value Possible added value
Registration phase Positive CHMP opinion

Product

Active substance Glucarpidase
Domain Oncology and Hematology
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.
Proprietary name Voraxaze
Manufacturer Ohara Pharmaceuticals
Mechanism of action Other, see general comments
Route of administration Intravenous
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)
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
Submission date August 2020
Expected Registration January 2022
Orphan drug Yes
Registration phase Positive CHMP opinion
Additional remarks Door de FDA goedgekeurd in 2012. Positieve CHMP opinie ontvangen in november 2021 voor a marketing authorisation under exceptional circumstances.

Therapeutic value

Therapeutic value Possible added value
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.
References NCT00481559; NCT01305655.

Expected patient volume per year

Patient volume

< 10

Market share is generally not included unless otherwise stated.
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

References Comparable Efficacy With Varying Dosages of Glucarpidase in Pediatric Oncology Patients. Jeffrey R. Scott. 2015.
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.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.