Timrepigene emparvovec Medicine 08 December 2020 Extended indication Patients with Choroideremia aged 18 years and older Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Timrepigene emparvovec Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Eye disorders Extended indication Patients with Choroideremia aged 18 years and older Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Biogen Portfolio holder Mechanism of action Gene therapy Route of administration Subretinal Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Gene therapy vector made from adeno-associated virus (AAV) called AAV2-REP1. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Yes Submission date 2021 Expected Registration 2022 Orphan drug Unknown Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Primary completion datum fase 3 studie in november 2020. Therapeutic value Current treatment options Op dit moment geen behandeling. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation De fase1/2 studies lieten zien dat na 24 maanden ruim 90% van de patiënten zijn zicht behield in plaats van de verwachte achteruitgang. Bij patiënten die achteruit gaan kan verbetering worden gezien. De fase 3 studie (STAR) loopt op dit moment nog. Duration of treatment Frequency of administration Dosage per administration References NCT02407678 (REGENERATE); NCT03507686 (GEMINI); NCT03584165 (SOLSTICE); NCT03496012 (STAR) Additional remarks Expected patient volume per year Patient volume < 136 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References CBS; Nelson LB, Spaeth GL, Nowinski TS, Margot CE, Jackson L.Aniridia. A review. Surv Ophthalmol 1984; 28: 621-4. Additional remarks De prevalentie wordt geschat op 1 op de 100.000. Dit zou voor Nederland betekenen dat er mogelijk 170 patiënten in aanmerking zullen komen voor deze behandeling. Hiervan zijn er, bij een gelijke distributie, 136 patiënten van 18 jaar of ouder. Expected cost per patient per year Cost References Additional remarks De kosten van deze behandeling zullen mogelijk in dezelfde lijn liggen als die van andere gentherapieën. 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 No Indication extensions References adisinsght Additional remarks Other information Additional remarks