On 26 November 2010, orphan designation (EU/3/10/813) was granted by the European Commission to Shire Pharmaceuticals Ireland Limited, Ireland, for recombinant human arylsulfatase A for the treatment of metachromatic leukodystrophy.

Key facts

Active substance
Recombinant human arylsulfatase A
Disease / condition
treatment of metachromatic leukodystrophy
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Shire Pharmaceuticals Ireland Limited
Block 2 & 3 Miesian Plaza
50 - 58 Baggot Street Lower
Dublin 2
Tel. +353 1 6422161

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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