Laruparetigene zosaparvovec Medicine 06 June 2023 Extended indication Retinitis Pigmentosa Therapeutic value No estimate possible yet Total cost € 161,460,000.00 Registration phase Clinical trials Product Active substance Laruparetigene zosaparvovec Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Eye disorders Extended indication Retinitis Pigmentosa Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Applied Genetic Technologies Portfolio holder Mechanism of action Gene therapy Route of administration Subretinal Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Retinitis pigmentosa (RP) is een erfelijke retinale dystrofie die leidt tot progressief verlies van fotoreceptoren en retinaal pigmentepitheel met blindheid als resultaat na enkele decennia. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date 2025 Expected Registration 2026 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Voretigeen neparvovec Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Laruparetigene zosaparvovec is een recombinant adeno-associated virus vector. Deze therapievorm is bekend en wordt al ingezet (voretigeen). Echter is dat maar in 1% van de gevallen in te zetten. Laruparetigeen zou breder inzetbaar moeten zijn. Duration of treatment Frequency of administration Dosage per administration References NCT04850118 Additional remarks Expected patient volume per year Patient volume 350 - 585 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References orphanet (1); Charters. AGTC-501 gene therapy offers vision gains for X-linked RP. 2022. (2) Additional remarks Prevalentie:1 op de 3.000 tot 1 op de 5.000 (1). In Nederland betreft dat ongeveer 3.500 tot 5.850 personen. Laruperetigeen grijpt in op het RPGR-gen. Dit gen is bij 10% van de RP-patiënten aangedaan (2). Dit zou dus betekenen dat dit middel bij 350 tot 585 personen kan worden ingezet . Expected cost per patient per year Cost € < 345,000.00 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 G-standaard Additional remarks Voretigeen kost €345.000 per behandeling. Omdat dit geneesmiddel iets breder inzetbaar zou zijn, zal het mogelijk iets goedkoper kunnen worden. Potential total cost per year Total cost € 161,460,000.00 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 Adis Insight Additional remarks Other information Additional remarks