On 19 June 2013, orphan designation (EU/3/13/1142) was granted by the European Commission to Centre Hospitalier Universitaire de Nantes, France, for adenovirus associated viral vector serotype 5 containing the human pde6β gene for the treatment of retinitis pigmentosa.
The sponsorship was transferred to Horma SAS, France, in December 2014.
In November 2016 Horama SAS changed name to Horama SA.
The sponsor’s name was changed from Horama SAS to Coave Therapeutics and the address was updated in August 2022.
adenovirus associated viral vector serotype 5 containing the human pde6β gene
Treatment of retinitis pigmentosa
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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: