Expand all sections

Idebenone

Raxone

Extended indication Treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) not using
Therapeutic value No judgement
Total cost 33,000,000.00
Registration phase Registration application pending

Product

Active substance Idebenone
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Duchenne
Extended indication Treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) not using glucocorticoids.
Proprietary name Raxone
Manufacturer Santhera
Mechanism of action Antioxidant
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Extramural (GVS)
Additional remarks Idebenone is a potent antioxidant and inhibitor of lipid peroxidation.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity Indication extension
Submission date June 2019
Expected Registration August 2020
Orphan drug Yes
Registration phase Registration application pending
Additional remarks De CHMP heeft deze indicatie voor Duchenne eerder afgewezen in januari 2018. Dit betreft een nieuwe indiening.

Therapeutic value

Current treatment options Corticosteroïden in de toekomst ook edasalonexent.
Therapeutic value No judgement
Substantiation In de fase 3 studies genaamd DELOS en SYROS werden positieve effecten gezien met betrekking tot de ademhalingsfunctie van patiënten. Er is op dit moment nog niet te zeggen of het geneesmiddel enige meerwaarde zal hebben. Idebenone heeft een ander werkingsmechanisme dan edasalonexent. Mogelijk zullen deze geneesmiddelen gecombineerd worden bij bewezen effectiviteit.
Frequency of administration 3 times a day
Dosage per administration 300 mg
References NCT01027884; NCT03603288

Expected patient volume per year

Patient volume

< 500

Market share is generally not included unless otherwise stated.
References Spierziekten Nederland
Additional remarks Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie.

Expected cost per patient per year

Cost 66,000.00
References Fabrikant, Medicijnkosten.nl, Prescribing Outlook 2017.
Additional remarks Fabrikant: €66.000 per patiënt per jaar. Medicijnkosten.nl: €70.931,68 per patiënt per jaar. Prescribing Outlook 2017: 'At current list price for Raxone, annual cost for 300mg three times daily would be about £77,400.'

Potential total cost per year

Total cost

33,000,000.00

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.