EU/3/13/1231: Orphan designation for the treatment of acute lymphoblastic leukaemia

(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1H-benzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl) amino)methyl)tetrahydrofuran-3,4-diol (pinometostat)

Overview

On 16 January 2014, orphan designation (EU/3/13/1231) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for (2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1H-benzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl) amino)methyl)tetrahydrofuran-3,4-diol (pinometostat) for the treatment of acute lymphoblastic leukaemia.

This medicine is now known as pinometostat.

In October 2021, Voisin Consulting S.A.R.L. changed name to Voisin Consulting Life Sciences.

Key facts

Active substance
(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1H-benzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl) amino)methyl)tetrahydrofuran-3,4-diol (pinometostat)
Intented use
Treatment of acute lymphoblastic leukaemia
Orphan designation status
Positive
EU designation number
EU/3/13/1231
Date of designation
16/01/2014
Sponsor

Voisin Consulting Life Sciences 
64 Avenue Pierre Grenier
92100 Boulogne-Billancourt
France
Tel. +33 1413 18300
E-mail: orphan@voisinconsulting.com

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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