This medicine is now known as avalotcagene ontaparvovec.
On 21 March 2016, orphan designation (EU/3/16/1623) was granted by the European Commission to Pharma Gateway AB, Sweden, for adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (also called DTX301) for the treatment of ornithine transcarbamylase deficiency.
The sponsorship was transferred to Ultragenyx Germany GmbH, Germany, in September 2020.
The sponsor’s address was updated in November 2020.
EU/3/16/1623: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency (PDF/123.42 KB)
First published: 04/05/2016
Last updated: 04/05/2016
Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec)
|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.