voretigene neparvovec

This medicine is authorised for use in the European Union.


Luxturna is a medicine that is used to treat adults and children with loss of vision due to inherited retinal dystrophy, a rare genetic disorder of the retina (the light sensitive membrane at the back of the eye).

Luxturna can only be used while patients still have enough functioning cells left in the retina and when the disease is caused by mutations (changes) in the gene RPE65. RPE65 is responsible for the production of an enzyme called all-trans retinyl isomerase, which is necessary for the normal functioning of retinal cells.

Luxturna contains the active substance voretigene neparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

Inherited retinal dystrophy is rare, and Luxturna was designated an ‘orphan medicine’ (a medicine used in rare diseases) for two forms of the disease on various dates (retinitis pigmentosa: 28 July 2015; Leber's congenital amaurosis: 2 April 2012).

This EPAR was last updated on 05/01/2021

Authorisation details

Product details
Agency product number
Active substance
voretigene neparvovec
International non-proprietary name (INN) or common name
voretigene neparvovec
Therapeutic area (MeSH)
  • Leber Congenital Amaurosis
  • Retinitis Pigmentosa
Anatomical therapeutic chemical (ATC) code
Not yet assigned
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Novartis Europharm Limited 
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Vista Building
Elm Park
Merrion Road
Dublin 4

Product information

20/11/2020 Luxturna - EMEA/H/C/004451 - PSUSA/00010742/202001


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Pharmacotherapeutic group

Not yet assigned

Therapeutic indication

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.

Assessment history

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