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.

Key facts

Active substance
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

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Ultragenyx Germany GmbH
Rahel-Hirsch-Strasse 10
10557 Berlin
Tel. +4930590083651

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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