Fordadistrogene movaparvovec Medicine 07 December 2022 Extended indication Treatment of ambulatory Duchenne Muscular Dystrophy patients Therapeutic value No estimate possible yet Total cost € 975,000,000.00 Registration phase Clinical trials Product Active substance Fordadistrogene movaparvovec Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Duchenne Extended indication Treatment of ambulatory Duchenne Muscular Dystrophy patients Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Pfizer Portfolio holder Mechanism of action Gene therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date April 2023 Expected Registration February 2024 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Dit geneesmiddel is geclassificeerd als een ATMP. De Fabrikant geeft aan indiening in het tweede kwartaal van 2023 te verwachten. Therapeutic value Current treatment options Steroïden. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT04281485 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 (1); Additional remarks Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie (1). Expected cost per patient per year Cost € < 1,950,000.00 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 https://www.thepharmaletter.com/article/duchenne-market-to-be-an-expensive-place-to-be Additional remarks De prijs is nog niet vastgesteld. De prijsstelling zal niet vóór het 2de kwartaal van 2024 beschikbaar zijn. In de VS wordt verwacht dat de prijs per behandeling rond de 2 miljoen dollar zal liggen. Potential total cost per year Total cost € 975,000,000.00 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