About voretigene neparvovec
Adeno-associated viral gene therapy
Used for
- Treats inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations, when viable retinal cells remain.
Usual adult dose
A single subretinal dose of 1.5 × 10¹¹ vector genomes per eye; eyes treated separately at least six days apart.
Usual dose in children
Same dose as adults; used in eligible children with confirmed RPE65 mutations and viable retinal cells.
Common side effects
- Eye redness, cataract, increased eye pressure, or inflammation
- Retinal tears, detachment, or thinning
- Eye pain or light sensitivity
- Headache or nausea
Important warnings
- Requires genetic confirmation of biallelic RPE65 mutations and assessment of viable retinal cells.
- Administered by subretinal injection by an experienced retinal surgeon.
- Risks include retinal damage, infection, and increased eye pressure.
- Avoid pregnancy during treatment and for four months afterward.
Pregnancy
Human pregnancy data are limited; treatment is generally avoided during pregnancy. Avoid pregnancy for four months after treatment.
Breastfeeding
It is unknown whether the therapy passes into breast milk; discuss breastfeeding with the treating specialist.
Interactions
- No established clinically important medicine interactions; medicines around surgery may affect care.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.