EU/3/20/2367: Orphan designation for the treatment of Prader-Willi syndrome

(R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate


On 9 December 2020, orphan designation EU/3/20/2367 was granted by the European Commission to Helsinn Birex Pharmaceuticals Limited, Ireland, for (R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate (also known as HM04 Fumarate or 12-HM04) for the treatment of Prader-Willi syndrome.

Key facts

Active substance
(R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate
Intended use
Treatment of Prader-Willi syndrome
Orphan designation status
EU designation number
Date of designation

Helsinn Birex Pharmaceuticals Limited
Dublin 15
Tel. +353 1 8225404

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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