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.

Key facts

Active substance
9-cis-Retinyl acetate
Disease / condition
Treatment of Leber's congenital amaurosis
Date of first decision
EU designation 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

Granzer Regulatory Consulting & Services
Kistlerhofstrasse 172c
81379 Munich
Tel: + 49 89-780 68 98 55

Patients' organisations

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;

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