Eteplirsen Medicine 09 January 2018 Extended indication Duchenne Muscular Dystrophy (DMD). Therapeutic value No judgement Total cost Registration phase Unknown Product Active substance Eteplirsen Domain Neurological disorders Reason of inclusion Main indication Muscular diseases Extended indication Duchenne Muscular Dystrophy (DMD). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Sarepta Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise 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 Particularity Unknown ATMP Unknown Submission date January 2017 Expected Registration February 2018 Orphan drug No Registration phase Unknown Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume < 2,500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://mens-en-gezondheid.infonu.nl/ziekten/154842-ziekte-van-duchenne-kenmerken-en-behandeling.html Additional remarks Expected cost per patient per year Cost 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 Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks