On 2 April 2012, orphan designation (EU/3/12/980) was granted by the European Commission to Axcentua Pharmaceuticals AB, Sweden, for genistein sodium salt dihydrate for the treatment of mucopolysaccharidosis type III (Sanfilippo syndrome).

Key facts

Active substance
Genistein sodium salt dihydrate
Disease / condition
Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome)
Date of first decision
EU designation number

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

Axcentua Pharmaceuticals AB
Alfred Nobels Allé 10
SE-141 52 Huddinge
Tel. +46 8 524 88 620
Fax +46 8 524 86 234

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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