Overview
Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2016 at the end of the period of market exclusivity.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 |
Naglazyme
|
Intended use |
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
|
Orphan designation status |
Expired
|
EU designation number |
EU/3/01/025
|
Date of designation |
15/02/2001
|
Sponsor |
BioMarin Europe Ltd.
10 Bloomsbury Way London WC1A 2SL United Kingdom Tel. + 44 (0)20 7420 0800 Fax + 44 (0)20 7420 0829 E-mail: biomarin-europe@bmrn.com |
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: