EU/3/08/582: Orphan designation for the treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)

Recombinant human heparan-N-sulfatase

Table of contents


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Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2021 upon request of the Sponsor.

Key facts

Active substance
Recombinant human heparan-N-sulfatase
Intended use
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)
Orphan designation status
EU designation number
Date of designation

Shire Pharmaceuticals Ireland Limited
Block 2 & 3 Miesian Plaza
50-58 Baggot Street Lower
Dublin 2, D02 Y754
Tel. + 353 1 429 7700

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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