EU/3/17/1848

About

On 20 March 2017, orphan designation (EU/3/17/1848) was granted by the European Commission to IntraBio Ltd, United Kingdom, for acetylleucine for the treatment of Niemann-Pick disease.

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

Key facts

Active substance
Acetylleucine
Disease / condition
Treatment of Niemann-Pick disease
Date of first decision
20/03/2017
Outcome
Positive
EU designation number
EU/3/17/1848

Review of designation

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

Sponsor's contact details

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

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;

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