EU/3/20/2305: Orphan designation for the treatment of myelodysplastic syndromes

Imetelstat sodium


On 27 July 2020, orphan designation EU/3/20/2305 was granted by the European Commission to Parexel International GmbH, Germany, for imetelstat sodium for the treatment of myelodysplastic syndromes.

Key facts

Active substance
Imetelstat sodium
Intended use
Treatment of myelodysplastic syndromes
Orphan designation status
EU designation number
Date of designation

Geron Netherlands B.V.
Naritaweg 165
1043 BW Amsterdam

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

June 2023The sponsorship was transferred to Geron Netherlands B.V., Netherlands in June 2023. 

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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