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.
EU/3/17/1906: Public summary of opinion on orphan designation: Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein for the treatment of Friedreich’s ataxia (PDF/126.47 KB)
First published: 17/10/2017
Last updated: 17/10/2017
Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein
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Review of designation
Sponsor's contact details
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.