On 21 May 2015, orphan designation (EU/3/15/1491) was granted by the European Commission to Cochamo Systems Ltd, United Kingdom, for adeno-associated viral vector serotype 9 containing the human HGSNAT gene for the treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome).

The sponsorship was transferred to Cochamo Pharma Limited, Ireland, in April 2019.

Key facts

Active substance
Adeno-associated viral vector serotype 9 containing the human HGSNAT gene
Disease / condition
Treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome)
Date of decision
Orphan decision 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

Unit 3d North Point House
North Point Business Park
New Mallow Road
T23 AT2P
Tel. +353 2 12066503

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;

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