On 20 March 2017, orphan designation (EU/3/17/1850) was granted by the European Commission to Dr Julien Baruteau, United Kingdom, for adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase for treatment of ornithine transcarbamylase deficiency.

The sponsor’s address was updated in December 2018.

Key facts

Active substance
Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase
Disease / condition
Treatment of ornithine transcarbamylase deficiency
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

Dr Julien Baruteau
4 ruelle Berthet
21200 Beaune
Tel. +33 3802 40926

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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