Idebenone Medicine 09 January 2018 Extended indication Duchenne Muscular Dystrophy (DMD). Therapeutic value No judgement Total cost € 165,000,000.00 Registration phase Unknown Product Active substance Idebenone 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 Raxone Manufacturer Santhera Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date June 2016 Expected Registration February 2018 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice 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 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 3 times a day Dosage per administration 300 mg 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 € 66,000.00 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. 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 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