EU/3/17/1927: Orphan designation for the treatment of acute myeloid leukaemia



On 16 October 2017, orphan designation (EU/3/17/1927) was granted by the European Commission to Helsinn Birex Pharmaceuticals Ltd, Ireland, for pracinostat for the treatment of acute myeloid leukaemia.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in February 2022 on request of the Sponsor.

Key facts

Active substance
Intended use
Treatment of acute myeloid leukaemia
Orphan designation status
EU designation number
Date of designation
Helsinn Birex Pharmaceuticals Ltd
Dublin 15
Tel. +353 1 808 96 46 / +41 91 985 2121

Review of designation

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

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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