On 4 July 2014, orphan designation (EU/3/14/1290) was granted by the European Commission to NightstaRx Ltd., United Kingdom, for adeno-associated viral vector serotype 2 containing the human REP1 gene for the treatment of choroideremia.

The sponsor’s address was updated in September 2018.

The sponsorship was transferred to Nightstar Europa Limited, Ireland, in February 2019.

Key facts

Active substance
Adeno-associated viral vector serotype 2 containing the human REP1 gene
Disease / condition
Treatment of choroideraemia
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

Nightstar Europa Limited
88 Harcourt Street
Dublin 2, D02 DK18
Tel. +353 1 691 5700

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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