OCU400 Medicine 03 December 2024 Extended indication Patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance OCU400 Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Eye disorders Extended indication Patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Ocugen Portfolio holder Mechanism of action Gene therapy Route of administration Ocular Therapeutical formulation Concentrate for solution for injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date June 2027 Expected Registration July 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Primary completion (estimated): 30 juni 2025. Therapeutic value Current treatment options 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 NCT06388200 Additional remarks Expected patient volume per year Patient volume 2,000 - 3,100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References 1: https://www.orpha.net/ ; 2: https://www.lumc.nl/actueel/2023/gentherapie-voor-retinitis-pigmentosa-werkt-op-gekweekt-netvlies/; 3: Diverse Genetic Landscape of Suspected Retinitis Pigmentosa in a Large Korean Cohort Yoon-Jeon et al. Genes (Basel). 2021 May; 12(5): 675.; Additional remarks De wereldwijde prevalentie van retinitis pigmentosa wordt geschat op 1:3.000 tot 1:5000 (1). In Nederland wordt het patiëntvolume geschat op 4.000 (2). De RHO mutatie is de meest voorkomende 5 tot 10% van de gevallen. Het percentage van genetische mutaties dat gediagnosticeerd wordt in patiënten met een familie historie van de ziekte (70,7%) ligt hoger dat dat van sporadische gevallen van de ziekte (51,3%) (3). Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks