On 13 May 2011, orphan designation (EU/3/11/861) was granted by the European Commission to ORS Oxford Ltd, United Kingdom, for 9-cis-retinyl acetate for the treatment of Leber's congenital amaurosis. The sponsorship was transferred to QLT Ophthalmics (UK), Ltd, United Kingdom, in November 2011.
The sponsorship was transferred to Granzer Regulatory Consulting & Services., Germany in May 2019.
|Disease / condition||
Treatment of leber's congenital amaurosis
|Date of decision||
|Orphan decision number||
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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;