On 14 October 2016, orphan designation (EU/3/16/1741) was granted by the European Commission to Horama SA, France, for adeno-associated viral vector serotype 5 containing the human RLBP1 gene for the treatment of retinitis pigmentosa.
The sponsor’s name was changed from Horama SAS to Coave Therapeutics and the address was updated in August 2022.
Adeno-associated viral vector serotype 5 containing the human RLBP1 gene
Treatment of retinitis pigmentosa
|Orphan designation status||
|EU designation number||
|Date 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: