EU/3/17/1949: Orphan designation for the treatment of GM2 gangliosidosis



On 12 December 2017, orphan designation (EU/3/17/1949) was granted by the European Commission to IntraBio Ltd, United Kingdom, for acetylleucine (also known as IBI1000) for the treatment of GM2 gangliosidosis.

The sponsorship was transferred to IntraBio Ireland Ltd, Ireland, in April 2019.

Key facts

Active substance
Intended use
Treatment of GM2 gangliosidosis
Orphan designation status
EU designation number
Date of designation

IntraBio Ireland Ltd
10 Earlsfort Terrace
Dublin 2
Co. Dublin D02 T380
Tel: +353 1 231 4600

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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