On 14 October 2016, orphan designation (EU/3/16/1740) was granted by the European Commission to Alacrita LLP, United Kingdom, for adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein for the treatment of retinitis pigmentosa.
The sponsorship was transferred to Allergan Pharmaceuticals International Limited, Ireland, in September 2017.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in June 2021 on request of the Sponsor.
Adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein
Treatment of retinitis pigmentosa
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Documents related to this orphan designation evaluation
EU/3/16/1740: Public summary of positive opinion for orphan designation of adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein for the treatment of retinitis pigmentosa (PDF/215.27 KB)
First published: 15/11/2016
Last updated: 11/06/2021
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: