EU/3/22/2642: Orphan designation for the treatment of galactosaemia


Table of contents


This medicine was designated as an orphan medicine for the treatment of galactosaemia in the European Union on 21 June 2022.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

Key facts

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

Advanz Pharma Limited
Unit 17
Northwood House
Northwood Crescent
Dublin 9
D09 V504
Co. Dublin

Update history

May 2023The sponsorship was transferred from Veristat Spain S.L., Spain, to Advanz Pharma Limited, Ireland.  
March 2023The sponsor’s name was changed from Drug Development and Regulation S.L. to Veristat Spain S.L. and the address was updated.  
  • List item

    Minutes of the COMP meeting on 10-12 May 2022 (PDF/517.18 KB)

    First published: 21/07/2022

  • 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:

    How useful was this page?

    Add your rating