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Idebenone

Raxone

Extended indication Duchenne Muscular Dystrophy (DMD).
Therapeutic value No judgement
Total cost 165,000,000.00

Product

Active substance Idebenone
Domain Neurological disorders
Main indication Muscular diseases
Extended indication Duchenne Muscular Dystrophy (DMD).
Proprietary name Raxone
Manufacturer Santhera
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date June 2016
Expected Registration February 2018
Orphan drug Yes
Additional remarks Reeds geregistreerd bij de EMA voor de behandeling van verminderd gezichtsvermogen bij adolescente en volwassen patiënten met opticusneuropathie van Leber (LHON) in 2015. De CHMP heeft de indicatie-uitbreiding voor Duchenne afgewezen in september 2017. Santhera heeft een herbeoordeling aangevraagd per oktober 2017.

Therapeutic value

Therapeutic value No judgement
Frequency of administration 3 times a day
Dosage per administration 300 mg

Expected patient volume per year

Patient volume

< 2,500

Market share is generally not included unless otherwise stated.
References https://mens-en-gezondheid.infonu.nl/ziekten/154842-ziekte-van-duchenne-kenmerken-en-behandeling.html

Expected cost per patient per year

Cost 66,000.00
References Fabrikant, Medicijnkosten.nl, Prescribing Outlook 2017.
Additional remarks Fabrikant: €66.000 p.p.p.j. Medicijnkosten.nl: € 70.931,68 p.p.p.j. 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

165,000,000.00

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.