EU/3/19/2218

About

On 13 November 2019, orphan designation EU/3/19/2218 was granted by the European Commission to Idorsia Pharmaceuticals Deutschland GmbH, Germany, for (2S,3R,4R,5S)-2-(hydroxymethyl)-1-pentylpiperidine-3,4,5-triol (also known as ACT-519276) for the treatment of GM2 gangliosidosis.

Key facts

Active substance
(2S,3R,4R,5S)-2-(hydroxymethyl)-1-pentylpiperidine-3,4,5-triol
Disease / condition
Treatment of GM2 gangliosidosis
Date of first decision
13/11/2019
Outcome
Positive
EU designation number
EU/3/19/2218

Review of designation

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

Sponsor's contact details

Idorsia Pharmaceuticals Deutschland GmbH
Marie-Curie-Strasse 8
79539 Loerrach
Germany
Tel. +49 7621 5790384
E-mail: clinical-trials-disclosure@idorsia.com

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.

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