EU/3/10/822 : Orphan designation for the treatment of Fanconi anaemia type A

Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene

Overview

On 17 December 2010, orphan designation (EU/3/10/822) was granted by the European Commission to the Centre for Biomedical Network Research on Rare Diseases (CIBERER), Spain, for lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for the treatment of Fanconi anaemia type A.

Key facts

Active substance
Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene
Intended use
Treatment of Fanconi anaemia type A
Orphan designation status
Positive
EU designation number
EU/3/10/822
Date of designation
17/12/2010
Sponsor

Rocket Pharmaceuticals B.V.
Kingsfordweg 151
1043 GR Amsterdam
Noord-Holland
Netherlands
E-mail: regulatoryeurope@rocketpharma.com

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

DateUpdate
September 2023The sponsorship was transferred from Consorcio Centro De Investigacion Biomedica En Red, Spain to Rocket Pharmaceuticals B.V., Netherlands.
November 2022The sponsor's name was updated.
October 2017Centro de Investigación Biomédica en Red (CIBER) changed name to Consorcio Centro de Investigación Biomédica en Red, M.P.
July 2014Centre for Biomedical Network Research on Rare Diseases (CIBERER) changed name to Centro de Investigación Biomédica en Red (CIBER)

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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