EU/3/20/2297: Orphan designation for the treatment of metachromatic leukodystrophy

Adeno-associated virus serotype HSC15 expressing human arylsulfatase A gene


On 26 June 2020, orphan designation EU/3/20/2297 was granted by the European Commission to YES Pharmaceutical Development Services GmbH, Germany, for adeno-associated virus serotype HSC15 expressing human arylsulfatase A gene (also known as HMI-202) for the treatment of metachromatic leukodystrophy.

Key facts

Active substance
Adeno-associated virus serotype HSC15 expressing human arylsulfatase A gene
Intended use
treatment of metachromatic leukodystrophy
Orphan designation status
EU designation number
Date of designation

Propharma Group The Netherlands B.V.
Schipholweg 73
2316 ZL Leiden

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

July 2023Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2023 on request of the Sponsor.
October 2022The sponsorship was transferred to Propharma Group The Netherlands B.V., Netherlands.
June 2021The sponsorship was transferred to Diamond Pharma Services Ireland Limited, Ireland.

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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