Expand all sections

Voretigene neparvovec

Luxturna

Extended indication RPE65-mediated inherited retinal disease (IRD).
Therapeutic value No judgement

Product

Active substance Voretigene neparvovec
Domain Neurological disorders
Main indication Eye disorders
Extended indication RPE65-mediated inherited retinal disease (IRD).
Proprietary name Luxturna
Manufacturer Spark
Route of administration Intravitreal
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)
Additional remarks Werkingsmechanisme: Voretigene neparvovec is DNA dat codeert voor een functioneel RPE65 eiwit.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date September 2017
Expected Registration July 2018
Orphan drug No

Therapeutic value

Therapeutic value No judgement
Duration of treatment Not found
Additional remarks Eenmalige injectie per oog.

Expected patient volume per year

References EMA
Additional remarks EMA: er zijn twee weesindicaties vastgesteld, samen betreft dat minder dan 4,7 patiënten per 10.000 pesonen. Dat is minder dan 8000 personen in Nederland. Verwachting is dat echte patientenaantal lager ligt en gering is.

Expected cost per patient per year

Cost < 1,000,000.00
References Scrip
Additional remarks Verwachting is dat het meerkosten gaat opleveren. Gaat in Amerika wellicht $1M of meer kosten.

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.