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.
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
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Documents related to this orphan designation evaluation
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: