On 18 November 2016, orphan designation (EU/3/16/1772) was granted by the European Commission to Audentes Therapeutics UK Limited, United Kingdom, for adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (also known as AT342) for the treatment of Crigler-Najjar syndrome.

The sponsorship was transferred to Audentes Therapeutics Netherlands B.V., Netherlands in May 2019.

Key facts

Active substance
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Disease / condition
Treatment of Crigler-Najjar syndrome
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

Audentes Therapeutics Netherlands B.V.
Schiphol Boulevard 359
Noord-Holland 1118 BJ
The Netherlands

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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