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:
Treatment of galactosaemia
|Orphan designation status||
|EU designation number||
|Date of designation||
Advanz Pharma Limited
|May 2023||The sponsorship was transferred from Veristat Spain S.L., Spain, to Advanz Pharma Limited, Ireland.|
|March 2023||The sponsor’s name was changed from Drug Development and Regulation S.L. to Veristat Spain S.L. and the address was updated.|
Minutes of the COMP meeting on 10-12 May 2022 (PDF/517.18 KB)
First published: 21/07/2022
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: