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).
Self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)
|Orphan designation status||
|EU designation number||
|Date of designation||
|September 2022||The sponsorship was transferred to Ultragenyx Germany GmbH, Germany.|
Documents related to this orphan designation evaluation
EU/3/16/1761: Public summary of positive opinion for orphan designation of self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene for the treatment of mucopolysaccharidosis type IIIA (Sanfil... (PDF/127.66 KB)
First published: 15/11/2016
Last updated: 15/11/2016
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: