voretigene neparvovec at a glance
Medicine class
AAV2-based retinal gene augmentation therapy
Who it is for
Adults and children with vision loss from confirmed biallelic RPE65 mutations and sufficient viable retinal cells
Administration
Injected beneath the retina during specialist vitreoretinal surgery under anaesthesia
Key aftercare
Follow positioning instructions and avoid flying or high-altitude travel until the eye's air bubble has disappeared
What is voretigene neparvovec used for?
Licensed use:
Vision loss in adults and children with inherited retinal dystrophy caused by confirmed mutations in both copies of the RPE65 gene, provided sufficient viable retinal cells remain.
How does voretigene neparvovec work?
RPE65 protein is needed to regenerate a light-sensitive molecule used in the visual cycle. Voretigene neparvovec uses a modified adeno-associated virus vector to carry a functioning RPE65 gene into retinal pigment epithelial cells, allowing them to produce RPE65 protein and potentially restore part of the visual cycle.
How to take voretigene neparvovec
This treatment is not self-administered. A specialist retinal surgeon injects the prepared solution beneath the retina during eye surgery under anaesthesia.
Take any prescribed immune-suppressing corticosteroid exactly as directed; do not stop it without specialist advice.
Maintain the postoperative head position specified by the surgical team.
Do not fly or travel to high altitude until an eye examination confirms that the air bubble has disappeared.
Ask the eye team before swimming, strenuous activity, driving or using machinery.
Place dressings and waste contaminated with tears or nasal secretions in sealed bags as instructed by the treatment centre.
voretigene neparvovec side effects
Common or expected effects
- Eye redness
- Cataract
- Raised pressure inside the eye
- Eye pain, irritation or swelling
- Retinal tear or retinal deposits
- Eye inflammation
- Headache, dizziness, nausea or vomiting
Get urgent medical advice
- Infection inside the eye
- Retinal detachment
- Retinal or choroidal bleeding
- Macular hole or other macular damage
- Chorioretinal atrophy, which can progress and may affect central vision
- A rapid pressure rise and irreversible sight loss if altitude increases while an air bubble remains in the eye
voretigene neparvovec in pregnancy
The effects during pregnancy and on an unborn baby are unknown. As a precaution, voretigene neparvovec should not be given during pregnancy; pregnancy or plans to conceive should be discussed with the specialist team before treatment.
voretigene neparvovec while breastfeeding
It is unknown whether voretigene neparvovec passes into breast milk. A specialist should help decide whether to pause breastfeeding or defer treatment, considering the benefits of breastfeeding and treatment.
voretigene neparvovec interactions
No clinically significant medicine interactions are known, although formal interaction studies have not been performed. The specialist team still needs a complete list of medicines, including non-prescription and herbal products.
Common questions about voretigene neparvovec
Answers are fully visible for fast scanning and source review.
Who is eligible for voretigene neparvovec?
Eligibility requires vision loss from inherited retinal dystrophy, confirmed harmful mutations in both RPE65 gene copies and enough viable retinal cells to treat. Assessment is undertaken by a specialist inherited-retinal-disease team.
Is genetic testing required before voretigene neparvovec?
Yes. The licensed indication requires confirmation of biallelic RPE65 mutations.
Can voretigene neparvovec restore normal vision?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.