EU/3/10/813: Orphan designation for the treatment of metachromatic leukodystrophy

Recombinant human arylsulfatase A


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.

The sponsorship was transferred to Takeda Pharmaceuticals International AG Ireland Branch, Ireland, in October 2021.

Key facts

Active substance
Recombinant human arylsulfatase A
Intended use
treatment of metachromatic leukodystrophy
Orphan designation status
EU designation number
Date of designation

Takeda Pharmaceuticals International AG Ireland Branch
Block 2
Miesian Plaza
50-58 Baggot Street Lower
Dublin 2
D02 HW68
Tel: +1 800937970

Review of designation

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

Update history

Date Update
October 2022 The sponsor's address was updated in October 2022.

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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