EU/3/17/1906

About

On 23 August 2017, orphan designation (EU/3/17/1906) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein (also known as AGIL-FA) for the treatment of Friedreich's ataxia.

The sponsorship was transferred to PTC Therapeutics International Limited, Ireland, in December 2018.

Key facts

Active substance
Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein
Medicine name
-
Disease / condition
Treatment of Friedreich's ataxia
Date of first decision
23/08/2017
Outcome
Positive
EU designation number
EU/3/17/1906

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

PTC Therapeutics International Limited
5th floor, 3 Grand Canal Plaza
Grand Canal Street Upper
Dublin 4, D04 EE70
Ireland
Tel: +353 01 906 8737
E-mail: medinfo@ptcbio.com

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;

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