On 17 January 2018, orphan designation (EU/3/17/1955) was granted by the European Commission to Quintiles Ireland Limited, Ireland, for adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene (also known as SB-318) for the treatment of mucopolysaccharidosis type I.
In May 2018 the sponsor, Quintiles Ireland Limited changed name to IQVIA RDS Ireland Limited.
The sponsor's address was updated in June 2018.
The sponsorship was transferred to TxCell S.A., France, in February 2019.
EU/3/17/1955: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene for the treatment of mucopolysaccharidosis t... (PDF/129.84 KB)
First published: 12/03/2018
Last updated: 12/03/2018
Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene
|Disease / condition||
Treatment of mucopolysaccharidosis type I
|Date of decision||
|Orphan decision 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;