On 7 November 2008, orphan designation (EU/3/08/582) was granted by the European Commission to Shire Pharmaceutical Development Limited, United Kingdom, for recombinant human heparan-N-sulfatase for the treatment of mucopolysaccharidosis III, type A (Sanfilippo A syndrome).
The sponsorship was transferred to Shire Pharmaceuticals Ireland Limited, Ireland, in September 2018.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2021 upon request of the Sponsor.
Recombinant human heparan-N-sulfatase
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A 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: