Voretigene Neparvovec
Voretigene Neparvovec (LUXTURNA) is a gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, including Leber congenital amaurosis and retinitis pigmentosa. It works by delivering a functional copy of the RPE65 gene directly to retinal cells via subretinal injection, allowing them to produce the normal enzyme required for the visual cycle.
A gene therapy that restores functional vision in patients with RPE65 mutation-associated inherited retinal dystrophies by delivering a normal copy of the RPE65 gene to retinal cells.
Key facts at a glance
- ATC
- S01XA27
- RxNorm
- 1995537
- Generic
- Voretigene Neparvovec
- Class
- atc-s01
- Brands
- Luxturna
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
6 groupspregnancy
all trimestersavoid if possible
use only if benefits outweigh potential risks to fetus
regular prenatal monitoring
pediatric_age
children 4 years and olderno adjustment needed
indicated for patients 4 years and older with RPE65 mutation-associated retinal dystrophy
regular ophthalmologic examinations and retinal imaging
geriatric
elderlyno adjustment needed
can be administered to elderly patients; consider overall health status
pre-procedure cardiac and ophthalmologic assessment
renal_impairment
mild to moderateno specific adjustment
use with caution; drug not primarily renally cleared
standard monitoring
hepatic_impairment
mild to moderateno specific adjustment
use with caution; drug not primarily hepatically cleared
standard monitoring
lactation
breastfeedingno adjustment
discontinue breastfeeding or avoid use; insufficient data on excretion in breast milk
no specific monitoring required
Statistics and clinical data
Drug class
Voretigene Neparvovec is an adeno-associated virus (AAV) vector-based gene therapy
FDA approval year
First gene therapy for an inherited retinal disease approved in the United States
Indication
Treatment of patients with confirmed biallelic RPE65 mutations leading to Leber congenital amaurosis type 2
Route of administration
Administered by subretinal injection directly into the retina
Treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy, including Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) in patients with sufficient viable retinal cells.
Delivers a normal copy of the RPE65 gene to retinal pigment epithelial (RPE) cells via a modified adeno-associated virus vector. This enables cells to produce functional retinoid isomerohydrolase (RPE65), an enzyme essential for the visual cycle and normal vision.
Administered via subretinal injection requiring surgical procedure by a qualified surgeon. Potential for immune-mediated responses; corticosteroid prophylaxis recommended. Treatment is unilateral or bilateral; dosing is based on body surface area. Requires genetic confirmation of RPE65 mutations before treatment. Patients should be monitored for intraocular pressure, retinal detachment, and potential cataracts.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy