voretigene neparvovec
Luxturna · ATC code S01XA27
Also known as: Voretigen neparvovec, Voretigen neparvovek
- Key facts
- Active substance
- voretigene neparvovec
- ATC code
- S01XA27
- Authorisation
- centrally authorised across the EU (EMA)
- Dosage forms
- Luxturna 5 × 1012 vector genomes/mL concentrate and solvent for solution for injection
- Marketing authorisation holder
- Novartis Europharm Limited
- Information as of
- 2026-10-29
Key facts
Voretigene neparvovec (Luxturna) is an AAV2-based gene therapy injected under the retina, used for vision loss due to inherited retinal dystrophy caused by biallelic RPE65 mutations.
What it is used for
- Treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Dosing
- A single dose of 1.5 × 10¹¹ vector genomes in each eye, delivered into the subretinal space in a total volume of 0.3 mL after vitrectomy; the two eyes are treated on separate days no fewer than 6 days apart.
- Must not be given by intravitreal injection; administered by a retinal surgeon experienced in macular surgery.
- Immunomodulatory regimen for each eye: prednisone (or equivalent) 1 mg/kg/day (maximum 40 mg/day) starting 3 days before and for 4 days including the day of administration, then 0.5 mg/kg/day (maximum 20 mg/day) for 5 days, then 0.5 mg/kg every other day for 5 days.
- Active infection must be excluded before starting. No dose adjustment in elderly, paediatric, hepatic or renal impairment patients.
Contraindications
- Hypersensitivity to the active substance or to any of the excipients.
- Ocular or periocular infection.
- Active intraocular inflammation.
Important warnings
- The administration procedure can cause eye inflammation including endophthalmitis, retinal tear and retinal detachment; symptoms must be reported without delay.
- Foveal thinning or loss of foveal function, macular hole and maculopathy may occur.
- Intraocular pressure should be monitored; air travel or travel to high elevations must be avoided until the air bubble has dissipated (up to one week or more), as a rapid altitude rise can cause irreversible vision loss.
Side effects
- Related to voretigene neparvovec: common retinal deposits; not known: chorioretinal atrophy.
- Related to the procedure (≥5%): conjunctival hyperaemia, cataract, increased intraocular pressure, retinal tear, dellen, macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain and maculopathy.
- Serious procedure-related events included increased intraocular pressure with optic atrophy, loss of foveal function and retinal detachment.
Interactions
- No known clinically significant interactions; no interaction studies have been performed.
Pregnancy and breastfeeding
- As a precaution, use during pregnancy is preferably avoided.
Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.
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For patients
Package leaflet, verbatim
This product has no separate US patient information.
Authorisation and brand names by country
Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.
EU – centrally authorised (all member states)
- LuxturnaNovartis Europharm Limited · EMEA/H/C/004451
Source: European Medicines Agency (EMA), product information
Brand names in 3 more European countries
- Iceland
- Luxturna
- Liechtenstein
- Luxturna
- Norway
- Luxturna
EMA – Article 57 product data (EU/EEA), 2026-10-29
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Other substances in the same group
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Sources
- European Medicines Agency. Luxturna – EPAR, Product Information. 2026-10-29. https://www.ema.europa.eu/en/documents/product-information/luxturna-epar-product-information_en.pdf
Editorial: Medelix editorial team · last updated 2026-10-29 · How these pages are made · Report an error
Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.