On 21 May 2015, orphan designation (EU/3/15/1491) was granted by the European Commission to Cochamo Systems Ltd, United Kingdom, for adeno-associated viral vector serotype 9 containing the human HGSNAT gene for the treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome).
The sponsorship was transferred to Cochamo Pharma Limited, Ireland, in April 2019.
EU/3/15/1491: Public summary of opinion on orphan designation: Triamcinolone acetonide for the treatment of non-infectious Adeno-associated viral vector serotype 9 containing the human HGSNAT gene for the treatment of mu... (PDF/124.53 KB)
First published: 30/06/2015
Last updated: 30/06/2015
Adeno-associated viral vector serotype 9 containing the human HGSNAT gene
|Disease / condition||
Treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome)
|Date of decision||
|Orphan decision number||
Review of designation
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;