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voretigene neparvovec

Phase 3

Biallelic RPE65 Mutation-associated Retinal Dystrophy | Gene therapy | Ophthalmology |Novartis AG|Last Updated: Jun 4, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment4
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04516369Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal DystrophyPHASE3 COMPLETED 4 — — Nov 24, 2020May 19, 2026Jun 4, 20261 Japan
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Study Endpoints
Primary Endpoints
Change from Baseline in full-field light sensitivity threshold
Baseline, Day 30, 90, 180, 270, and Year 1 after second eye injection

Full-field light sensitivity threshold (FST) is evaluated using white light, as averaged over both eyes.

Secondary Endpoints
Change from Baseline in visual field
Baseline, Day 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection
Change from Baseline in macular threshold
Baseline, Day 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection
Change from Baseline in visual acuity
Baseline, Day 1, and 3 after first eye injection; Day 1, 3, 14, 30, 90, 180, 270, and Year 1, 2, 3, 4, 5 after second eye injection
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Voretigene neparvovecEXPERIMENTAL1.5 E11 vg (0.3 mL subretinal injection in each eye, 6-18 days apart)
Interventions
NameTypeDescription
voretigene neparvovecGENETICVoretigene neparvovec is an adeno-associated viral type 2 (AAV2) gene therapy vector driving expression of normal human retinal pigment epithelium 65 kDa protein (hRPE65) gene.
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Eligibility Criteria
Age Range4 Years — 100 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Japanese participants with biallelic RPE65 mutation-associated retinal dystrophy; molecular diagnosis of RPE65 mutation must be confirmed by a Novartis designated laboratory in Japan. * Age four years or older. * Visual acuity worse than 20/60 (both eyes) and/or visual field l...

Countries:Japan
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Recent Changes (Last 90 Days)
HIGHJun 4, 2026NCT04516369Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJun 4, 2026NCT04516369Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJun 4, 2026NCT04516369Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJun 4, 2026NCT04516369Status: ACTIVE_NOT_RECRUITING → COMPLETED
LOWMay 26, 2026NCT04516369primaryCompletionDate: changed
LOWMay 24, 2026NCT04516369studyFirstPostDate: changed