EU/3/16/1761: Orphan designation for the treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)

Self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene

Table of contents

Overview

On 14 October 2016, orphan designation (EU/3/16/1761) was granted by the European Commission to Ser-mes Planificación SL, Spain, for self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene for the treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome).

Key facts

Active substance
Self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene
Intended use
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)
Orphan designation status
Positive
EU designation number
EU/3/16/1761
Date of designation
20/09/2016
Sponsor

Ultragenyx Germany GmbH
Rahel-Hirsch-Strasse 10
Moabit
10557 Berlin
Germany
Tel. +4930590083651
E-mail: medinfo@ultragenyx.com

Update history

DateUpdate
September 2022The sponsorship was transferred to Ultragenyx Germany GmbH, Germany.

Documents related to this orphan designation evaluation

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.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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