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.
EU/3/17/1850: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase for treatment of ornithine transcarbamylase deficiency (PDF/126.05 KB)
First published: 05/05/2017
Last updated: 05/05/2017
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
Sponsor's contact details
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;