EU/3/15/1462: Orphan designation for the treatment of retinitis pigmentosa

Sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one

Overview

On 19 March 2015, orphan designation (EU/3/15/1462) was granted by the European Commission to Universitätsklinikum Tübingen, Germany, for sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one for the treatment of retinitis pigmentosa.

Key facts

Active substance
Sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one
Intented use
Treatment of retinitis pigmentosa
Orphan designation status
Positive
EU designation number
EU/3/15/1462
Date of designation
19/03/2015
Sponsor
Universitätsklinikum Tübingen (UKT)
Geissweg 3
72076 Tübingen
Germany
Tel. +49 7071 29 87 430
Fax +49 7071 29 57 77
E-mail: francois.paquet-durand@klinikum.uni-tuebingen.de

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:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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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