Luxturna
What Luxturna is used for
Luxturna is indicated for the 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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
Key facts
- Brand
- Luxturna
- Active substance
- voretigene neparvovec
- Disease area
- Leber Congenital Amaurosis; Retinitis Pigmentosa
- Medicine group
- Other ophthalmologicals
- ATC code
- S01XA27
- Marketing authorisation holder
- Novartis Europharm Limited
- Authorised in the EU since
- 2018
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Luxturna used for?
Luxturna is indicated for the 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.
What is the active substance in Luxturna?
Luxturna contains voretigene neparvovec.
Who makes Luxturna?
The EU marketing authorisation holder is Novartis Europharm Limited.
Is Luxturna an orphan medicine?
Yes. Luxturna is designated an orphan medicine in the EU, for a rare disease (Leber Congenital Amaurosis; Retinitis Pigmentosa).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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