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
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.
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 comments 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 Registered
Additional comments Positieve CHMP opinie ontvangen in november 2021 voor markt autorisatie onder exceptionele omstandigheden.

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 comments 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
References Comparable Efficacy With Varying Dosages of Glucarpidase in Pediatric Oncology Patients. Jeffrey R. Scott. 2015., drugs.com
Additional comments 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 100.000-150.000.

Potential total cost per year

Total cost

1,250,000.00

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.

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.