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.
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec)
|Disease / condition||
Treatment of leber's congenital amaurosis
|Date of decision||
|Orphan decision number||
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Luxturna at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the orphan medicine assessment report
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;