On 24 April 2015, orphan designation (EU/3/15/1482) was granted by the European Commission to Horama SAS, France, for adeno-associated viral vector serotype 5 containing the human CHM gene for the treatment of choroideremia.

In November 2016 Horama SAS changed name to Horama SA.

The sponsorship was transferred to Inserm-Transfert SA, France, in October 2017.

Key facts

Active substance
Adeno-associated viral vector serotype 5 containing the human CHM gene
Disease / condition
Treatment of choroideremia
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

Inserm-Transfert SA
7 rue Watt
75013 Paris
Tel: +33 1 55 03 01 69

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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