EU/3/14/1330

About

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.

Key facts

Active substance
Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene
Disease / condition
Treatment of pyruvate kinase deficiency
Date of decision
22/08/2014
Outcome
Positive
Orphan decision number
EU/3/14/1330

Review of designation

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

Sponsor's contact details

Consorcio Centro de Investigación Biomédica en Red, M.P.
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

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.

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