Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in June 2023 on request of the Sponsor.
On 17 January 2018, orphan designation (EU/3/17/1956) 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 iduronate 2-sulfatase gene (also called SB-913) for the treatment of mucopolysaccharidosis type II (Hunter's syndrome).
Key facts
Active substance |
Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene
|
Intended use |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
|
Orphan designation status |
Withdrawn
|
EU designation number |
EU/3/17/1956
|
Date of designation |
17/01/2018
|
Sponsor |
Sangamo Therapeutics France S.A.S. |
Update history
Date | Update |
---|---|
September 2023 | Product withdrawn from the Union Register of orphan medicinal products on request of the Sponsor. |
July 2019 | TxCell S.A.changed name to Sangamo Therapeutics France S.A.S. |
February 2019 | The sponsorship was transferred to TxCell S.A., France |
June 2018 | The sponsorship was transferred to Sangamo Therapeutics UK LTD, United Kingdom and the address updated. |
May 2018 | The Sponsor, Quintiles Ireland Limited, changed name to IQVIA RDS Ireland Limited. |
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: