EU/3/20/2346: Orphan designation for the treatment of GM1 gangliosidosis

Adeno-associated virus serotype hu68 containing the human GLB1 gene


On 19 October 2020, orphan designation EU/3/20/2346 was granted by the European Commission to Pharma Gateway AB, Sweden, for adeno-associated virus serotype hu68 containing the human GLB1 gene (also known as PBGM01) for the treatment of GM1 gangliosidosis.

Key facts

Active substance
Adeno-associated virus serotype hu68 containing the human GLB1 gene
Intended use
Treatment of GM1 gangliosidosis
Orphan designation status
EU designation number
Date of designation

Pharma Gateway AB
Johanneslundsvägen 2
194 61 Upplands Väsby
Stockholms Lan
Tel. +46 8 590 778 00

Review of designation

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

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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