EU/3/17/1956: Orphan designation for the treatment of mucopolysaccharidosis type II (Hunter's syndrome)

Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene

Table of contents

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Please note that this product was withdrawn from the Union Register of orphan medicinal products in June 2023 on request of the Sponsor.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Withdrawn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.
Les Cardoulines Ht1
Allee De La Nertiere
06560 Valbonne
France
Tel. +33 4972 18301
E-mail: communications@sangamo.com

Update history

DateUpdate
September 2023Product withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.
July 2019TxCell S.A.changed name to Sangamo Therapeutics France S.A.S.
February 2019The sponsorship was transferred to TxCell S.A., France
June 2018The sponsorship was transferred to Sangamo Therapeutics UK LTD, United Kingdom and the address updated.
May 2018The Sponsor, Quintiles Ireland Limited, changed name to IQVIA RDS Ireland Limited.

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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