On 14 February 2001, orphan designation (EU/3/01/025) was granted by the European Commission to ClinTrials Research Limited, United Kingdom, for N-acetylgalactosamine-4-sulfatase for the treatment of mucopolysaccharidosis, type VI (Maroteaux-Lamy syndrome).
The sponsorship was transferred to Dr. Gertrud Thormann, France in December 2002 and subsequently to BioMarin Europe Ltd, Ireland, in January 2005.
N-acetylgalactosamine-4-sulfatase has been authorised in the EU as Naglazyme since 24 January 2006.
|Disease / condition||
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
|Date of first decision||
|EU designation number||
Sponsor's contact details
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;