Viltolarsen Medicine 07 December 2022 Extended indication Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping Therapeutic value No estimate possible yet Total cost € 20,700,000.00 Registration phase Clinical trials Product Active substance Viltolarsen Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Duchenne Extended indication Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Nippon Shinyaku Portfolio holder Mechanism of action Antisense oligonucleotide Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Viltrolarsen induceert exon 53 skipping waardoor er een functioneel dystrofine eiwit wordt geproduceerd. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2024 Expected Registration 2025 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Goedgekeurd door de FDA in augustus 2020. Therapeutic value Current treatment options Corticosteroïden Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Fase 2 studie gepubliceerd in 2020. Fase 3 studie (racer53, n=74, follow up tot 48 weken) loopt nog tot 2024. Duration of treatment Frequency of administration 1 times a week Dosage per administration 80 mg/kg References NCT04060199 Additional remarks Expected patient volume per year Patient volume 40 - 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Spierziekten Nederland; Clemens et al. JAMA Neurol. 2020;77(8):982-991. Additional remarks Er zijn in Nederland rond de 500 patiënten met Duchenne spierdystrofie. Echter, alleen patiënten met mutaties in exon 53 komen in aanmerking. Dit is ongeveer 8 tot 10% van de patiënten en dus is het mogelijke aantal patiënten dat in aanmerking komt 40 tot 50. Expected cost per patient per year Cost € 300,000.00 - 620,000.00 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.biopharmadive.com/news/viltolarsen-duchenne-fda-approval-ns-pharma/583410/ Additional remarks Mogelijk gelijk aan de eerdere schattingen voor eteplirsen: zo'n $300,000 per jaar. In de Verenigde Staten heeft viltolarsen een lijstprijs van $733.000 per jaar voor een patiënt van 30kg. Dit komt neer op ongeveer €620.000. De prijs in Nederland zal naar verwachting lager liggen. Potential total cost per year Total cost € 20,700,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks