Idebenone Medicine 09 January 2018 Extended indication Treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) not using glucocorticoids. Therapeutic value No judgement Total cost € 33,000,000.00 Registration phase Registration application pending Product Active substance Idebenone Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Duchenne Extended indication Treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) not using glucocorticoids. Current proprietary name Already available biosimilars / generics Proprietary name Raxone Manufacturer Santhera Portfolio holder Mechanism of action Antioxidant Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Idebenone is a potent antioxidant and inhibitor of lipid peroxidation. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Indication extension ATMP Unknown Submission date June 2019 Expected Registration August 2020 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks De CHMP heeft deze indicatie voor Duchenne eerder afgewezen in januari 2018. Dit betreft een nieuwe indiening. Therapeutic value Current treatment options Corticosteroïden in de toekomst ook edasalonexent. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. 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 Frequency of administration 3 times a day Dosage per administration 300 mg References NCT01027884; NCT03603288 Additional remarks Expected patient volume per year Patient volume < 500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Spierziekten Nederland Additional remarks Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie. 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 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.' Potential total cost per year Total cost € 33,000,000.00 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