EU/3/11/917

About

On 27 October 2011, orphan designation (EU/3/11/917) was granted by the European Commission to Institut Pasteur, France, for adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase gene for the treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome).

Key facts

Active substance
Adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase gene
Disease / condition
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Date of first decision
27/10/2011
Outcome
Positive
EU designation number
EU/3/11/917

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Institut Pasteur
25-28 rue du Docteur Roux
75724 Paris Cedex 15
France
Tel. +33 1 44 38 91 01
Fax +33 1 40 61 39 77
E-mail: muriel.eliaszewicz@pasteur.fr

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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