Voretigene neparvovec Medicine 09 January 2018 Extended indication RPE65-mediated inherited retinal disease (IRD). Therapeutic value No judgement Total cost Registration phase Unknown Product Active substance Voretigene neparvovec Domain Neurological disorders Reason of inclusion Main indication Eye disorders Extended indication RPE65-mediated inherited retinal disease (IRD). Current proprietary name Already available biosimilars / generics Proprietary name Luxturna Manufacturer Spark Portfolio holder Mechanism of action Unknown Route of administration Intravitreal Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: Voretigene neparvovec is DNA dat codeert voor een functioneel RPE65 eiwit. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date September 2017 Expected Registration July 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 Eenmalige injectie per oog. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References EMA Additional remarks EMA: er zijn twee weesindicaties vastgesteld, samen betreft dat minder dan 4,7 patiënten per 10.000 pesonen. Dat is minder dan 8000 personen in Nederland. Verwachting is dat echte patientenaantal lager ligt en gering is. Expected cost per patient per year Cost € < 1,000,000.00 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. References Scrip Additional remarks Verwachting is dat het meerkosten gaat opleveren. Gaat in Amerika wellicht $1M of meer kosten. 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