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Lenadogene nolparvovec

Extended indication Leber's hereditary optic neuropathy
Therapeutic value No judgement
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
Manufacturer GenSight Biologics
Route of administration Intravitreal
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)
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)
Submission date 2019
Expected Registration 2020
Orphan drug Yes
Registration phase Clinical trials
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
Duration of treatment one-off
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.
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

Additional remarks Waarschijnlijk duur

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No

Other information

There is currently no futher information available.