EU/3/18/2037: Orphan designation for the treatment of Friedreich’s ataxia



On 27 June 2018, orphan designation (EU/3/18/2037) was granted by the European Commission to Dr Stefan Blesse, Germany, for omaveloxolone (also known as RTA 408) for the treatment of Friedreich's ataxia.

The sponsorship was transferred to Granzer Regulatory Consulting & Services, in November 2019.

The sponsorship was transferred to Reata Ireland Limited, Ireland, in August 2021.

Key facts

Active substance
Intended use
Treatment of Friedreich’s ataxia
Orphan designation status
EU designation number
Date of designation

Reata Ireland Limited

Block A George's Quay Plaza
George's Quay
Dublin 2
D02 E440
Co. Dublin


Review of designation

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

Update history

Date Update
January 2023

The sponsor's address was updated.

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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