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
Table of contents
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 |
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. |
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: