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Eteplirsen

Extended indication Duchenne Muscular Dystrophy (DMD).
Therapeutic value No judgement

Product

Active substance Eteplirsen
Domain Neurological disorders
Main indication Muscular diseases
Extended indication Duchenne Muscular Dystrophy (DMD).
Manufacturer Sarepta
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks Toedieningsvorm: voor kinderen komt er ook een vorm van dit geneesmiddel beschikbaar dat subcutaan kan worden gegeven (bron EMA).

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date January 2017
Expected Registration February 2018
Orphan drug No

Therapeutic value

Therapeutic value No judgement

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

References Prescribing Outlook 2017
Additional remarks Prescribing Outlook 2017: 'Expected to be very expensive. Average US cost is estimated at $300,000 per year.'

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.