This medicine is now known as voretigene neparvovec.
On 28 July 2015, orphan designation (EU/3/15/1518) was granted by the European Commission to Alan Boyd Consultants Ltd, United Kingdom, for adenovirus-associated viral vector serotype 2 containing the human RPE65 gene for the treatment of retinitis pigmentosa.
The sponsorship was transferred to Spark Therapeutics Ireland Ltd, in March 2017.
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene in the amended indication treatment of inherited retinal dystrophies has been authorised in the EU as Luxturna since 22 November 2018.
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec)
|Disease / condition||
Treatment of retinitis pigmentosa
|Date of decision||
|Orphan decision number||
Review of designation
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;