EU/3/14/1330: Orphan designation for the treatment of pyruvate kinase deficiency

Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene

Overview

On 22 August 2014, orphan designation (EU/3/14/1330) was granted by the European Commission to Centro de Investigación Biomédica en Red (CIBER), Spain, for lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene for the treatment of pyruvate kinase deficiency.

In October 2017 Centro de Investigación Biomédica en Red (CIBER) changed name to Consorcio Centro de Investigación Biomédica en Red, M.P.

The sponsor's name was updated in November 2022.

Key facts

Active substance
Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene
Intended use
Treatment of pyruvate kinase deficiency
Orphan designation status
Positive
EU designation number
EU/3/14/1330
Date of designation
22/08/2014
Sponsor

Consorcio Centro de Investigación Biomédica en Red
Instituto de Salud Carlos III
Pabellón 11, Planta 0
C/ Monforte de Lemos 3-5
28029 Madrid
Spain               
Tel. +34 91 171 8114
E-mail: gestores@ciber.es

Review of designation

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

Update history

Date Update
July 2014 Centre for Biomedical Network Research on Rare Diseases (CIBERER) changed name to Centro de Investigación Biomédica en Red (CIBER)
October 2017 Centro de Investigación Biomédica en Red (CIBER) changed name to Consorcio Centro de Investigación Biomédica en Red, M.P.
November 2022 The sponsor's name was updated.

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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