EU/3/01/025: Orphan designation for the treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

N-acetylgalactosamine 4-sulfatase

Table of contents


Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2016 at the end of the period of market exclusivity.

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.

Key facts

Active substance
N-acetylgalactosamine 4-sulfatase
Medicine name
Intended use
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Orphan designation status
EU designation number
Date of designation
BioMarin Europe Ltd.
10 Bloomsbury Way
London WC1A 2SL
United Kingdom
Tel. + 44 (0)20 7420 0800
Fax + 44 (0)20 7420 0829

Documents related to this orphan designation evaluation

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.

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:

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