Lenadogene nolparvovec Medicine 13 December 2018 Extended indication Leber's hereditary optic neuropathy Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Lenadogene nolparvovec Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Eye disorders Extended indication Leber's hereditary optic neuropathy Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer GenSight Biologics Portfolio holder Mechanism of action Gene therapy Route of administration Intravitreal Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date 2020 Expected Registration August 2021 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Advanced therapy medicinal product (ATMP). Estimated Primary Completion Date fase III REVERSE studie: september 2018. Therapeutic value Current treatment options Geen Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration 9E10 viral genomes References NCT02652767; Healio.com, 15 juni 2018 Additional remarks 3 fase 3 studies, rescue, reverse en reflect. 1e studie net data gepresenteerd. Geen groot verschil in zicht tussen de onbehandelde en behandelde patiënten. Expected patient volume per year Patient volume 3 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.orpha.net/ Additional remarks De wereldwijde prevalentie wordt geschat op 1:15.000 tot 1:50.000. Voor Nederland zou dit 300-1.000 patiënten betekenen. Het gaat hier alleen om patiënten met een mutatie in het ND4 gen. Inschatting is daarom 3 patiënten per jaar. Expected cost per patient per year Cost References Additional remarks Waarschijnlijk duur 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 Additional remarks Other information Additional remarks