EU/3/16/1771

Table of contents

About

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.

Key facts

Active substance
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
18/11/2016
Outcome
Positive
EU designation number
EU/3/16/1771

Sponsor's contact details

Ultragenyx Germany GmbH
Friedrichstrasse 191
10117 Berlin
Germany
Tel. +49 30 2065 9217
E-mail: inquiry@ultragenyx.com

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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