Idebenone

Active substance
Idebenone
Domain
Neurological disorders
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Duchenne
Extended indication
Treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) not using glucocorticoids.

1. Product

Proprietary name
Raxone
Manufacturer
Santhera
Mechanism of action
Antioxidant
Route of administration
Oral
Therapeutical formulation
Tablet
Budgetting framework
Extramural (GVS)
Additional comments
Idebenone is a potent antioxidant and inhibitor of lipid peroxidation.

2. 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 comments
De CHMP heeft deze indicatie voor Duchenne eerder afgewezen in januari 2018. Dit betreft een nieuwe indiening.

3. 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.
Duration of treatment
Not found
Frequency of administration
3 times a day
Dosage per administration
300 mg
References
NCT01027884; NCT03603288

4. Expected patient volume per year

Patient volume

< 500

Market share is generally not included unless otherwise stated.

References
Spierziekten Nederland
Additional comments
Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie.

5. Expected cost per patient per year

Cost
66,000
References
Fabrikant, Medicijnkosten.nl, Prescribing Outlook 2017.
Additional comments
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.'

6. Potential total cost per year

Total cost

33,000,000

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.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

There is currently nothing known about indication extensions.

9. Other information

There is currently no futher information available.