This medicine is now known as voretigene neparvovec.
On 2 April 2012, orphan designation (EU/3/12/981) 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 Leber's congenital amaurosis.
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.
The sponsorship was transferred to Novartis Europharm Limited, Ireland, in February 2019.
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec)
Treatment of Leber's congenital amaurosis
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
Documents related to this orphan designation evaluation
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: