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

Acetylleucine

Overview

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
Acetylleucine
Intended use
Treatment of GM2 gangliosidosis
Orphan designation status
Positive
EU designation number
EU/3/17/1949
Date of designation
12/12/2017
Sponsor

IntraBio Ireland Ltd
10 Earlsfort Terrace
Dublin 2
Co. Dublin D02 T380
Ireland
Tel: +353 1 231 4600
E-mail: mm@intrabio.co.uk

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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