|
gptkbp:instanceOf
|
gptkb:gene_therapy
|
|
gptkbp:administeredBy
|
gptkb:ophthalmologist
single dose per eye
|
|
gptkbp:age
|
patients aged 1 year and older
|
|
gptkbp:approvalYear
|
2017
|
|
gptkbp:approvedBy
|
gptkb:FDA
gptkb:EMA
|
|
gptkbp:ATCCode
|
gptkb:S01XA27
|
|
gptkbp:countryOfOrigin
|
gptkb:United_States
|
|
gptkbp:developedBy
|
gptkb:Spark_Therapeutics
|
|
gptkbp:firstBook
|
yes
|
|
gptkbp:genericName
|
gptkb:voretigene_neparvovec
|
|
gptkbp:indication
|
gptkb:Leber_congenital_amaurosis
retinal dystrophy due to RPE65 mutation
|
|
gptkbp:mechanismOfAction
|
delivers functional RPE65 gene
|
|
gptkbp:orphanDrugStatus
|
yes
|
|
gptkbp:prescriptionStatus
|
prescription only
|
|
gptkbp:priceRange
|
$850,000 per treatment
|
|
gptkbp:routeOfAdministration
|
subretinal injection
|
|
gptkbp:sideEffect
|
gptkb:glaucoma
ocular inflammation
increased intraocular pressure
endophthalmitis
macular hole
retinal tear
|
|
gptkbp:storage
|
refrigerated
|
|
gptkbp:targetGene
|
gptkb:RPE65
|
|
gptkbp:type
|
AAV2-based gene therapy
|
|
gptkbp:yearOfEMAApproval
|
2018
|
|
gptkbp:bfsParent
|
gptkb:Spark_Therapeutics
gptkb:voretigene_neparvovec
|
|
gptkbp:bfsLayer
|
7
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
Luxturna
|