This medicine is now known as pariglasgene brecaparvovec.
On 18 November 2016, orphan designation (EU/3/16/1771) was granted by the European Commission to Pharma Gateway AB, Sweden, for adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (also known as DTX401) for treatment of glycogen storage disease type Ia.
The sponsorship was transferred to Ultragenyx Germany GmbH, Germany, in September 2020.
adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (pariglasgene brecaparvovec)
|Disease / condition||
treatment of glycogen storage disease type Ia
|Date of first decision||
|EU designation number||
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.