Last reviewed 2026-04-20 · How we verify

VORETIGENE NEPARVOVEC

Voretigene neparvovec is a marketed gene therapy with a key composition patent expiring in 2028. Its primary strength lies in being the first approved gene therapy for its indication. The primary risk is the lack of direct competitors, which may lead to increased scrutiny and regulatory pressure.

At a glance

Approved indications

No approved indications tracked.

Common side effects

• Conjunctival hyperemia
• Cataract
• Increased intraocular pressure
• Retinal tear
• Dellen (thinning of the corneal stroma)
• Macular hole
• Subretinal deposits
• Eye inflammation
• Eye irritation
• Eye pain
• Maculopathy (wrinkling on the surface of the macula)

Serious adverse events

• Chorioretinal atrophy (retinal degeneration, retinal depigmentation, injection site atrophy)

Key clinical trials

• A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
• Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 (PHASE1, PHASE2)
• Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy (PHASE3)
• Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (PHASE3)
• Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
• Safety Study in Subjects With Leber Congenital Amaurosis (PHASE1)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• VORETIGENE NEPARVOVEC CI brief — competitive landscape report
• VORETIGENE NEPARVOVEC updates RSS · CI watch RSS