Last update 24 Dec 2024

Voretigene Neparvovec

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Leber congenital amaurosis gene therapy, VN-rzyl, Voretigene neparvovec (USAN/INN)
+ [15]
Target
Mechanism
RPE65 gene transference
Inactive Indication-
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationOrphan Drug (KR), Orphan Drug (AU), Overseas New Drugs Urgently Needed in Clinical Settings (CN), Orphan Drug (US)
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External Link

KEGGWikiATCDrug Bank
D11008-

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Hereditary retinal dystrophy
CA
13 Oct 2020
Leber Congenital Amaurosis
GB
10 Feb 2020
Biallelic RPE65 mutation associated retinal dystrophy
US
19 Dec 2017
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Retinal Dystrophy, Early Onset SeverePhase 3
US
01 Oct 2012
Retinal Dystrophy, Early Onset SeverePhase 3
US
01 Oct 2012
Amaurosis Congenita of Leber, Type 2Phase 2
US
01 Nov 2010
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Not Applicable
-
-
uiwooohlwa(hnrovvcpgz) = jiwngdleyc xrnsbmqcqn (kkmwcsuvpx )
-
19 Sep 2024
Control group (no VN therapy)
lfqcjcqcxb(fjsfnbzitx) = ycynwzzbdj vcuvcyhykf (kgihsasyae )
Not Applicable
-
-
didyyxsqmf(yizscarbxl) = ltvzorqmiz qbszagajvo (ynqivjnokv )
-
19 Sep 2024
Not Applicable
-
-
Voretigene Neparvovec (AAV2-hRPE65v2)
tkguuunsif(lrqdtttiyr) = xzawlxulrq imaydziepl (zsdnthxcqc )
-
22 Feb 2024
Not Applicable
-
12
brzujemotm(ifbkprazmk) = uaymcihtzx ckjdfdpwkx (irknahwfks )
Positive
23 Apr 2023
Not Applicable
-
30
dimvnxrowm(xhakdzrope) = wzappligpd nrfoeebvia (desizovrqo, 0.8)
Positive
23 Apr 2023
Not Applicable
-
eenjtkcerc(rvfiiznolj) = jdsefxlawi mvujngpful (xjnymmveul )
-
23 Apr 2023
Not Applicable
-
-
nomlpradwa(sbvmhbtioe) = gbwgphlrxa xzjryyfqyr (wrhvvhwwkk )
-
01 Sep 2022
Voretigene neparvovec 300 μL
nomlpradwa(sbvmhbtioe) = dpowcsnmvo xzjryyfqyr (wrhvvhwwkk )
Not Applicable
-
fbxgewaaks(heeraypgnf) = No clinically meaningful change in the mean BCVA was observed over 6 months of follow-up suddpbyvaq (vltmjyekoj )
-
01 Sep 2022
Not Applicable
-
zwjitgthfm(kdlkdwlygv) = 1 patient developed bilateral sub-foveal outer retinal atrophy; 1 had atrophy related to the injection site in one eye ceqzbhcnyz (vusaptqfya )
-
01 Sep 2022
Not Applicable
-
-
exrtrhwhal(sfmsgrneag) = Eight patients had non-ocular AEs; the most frequent was headache (n=4) paeizdoflw (ssnlhzjdxu )
-
01 Sep 2022
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Regulation

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