EU/3/16/1786: Orphan designation for the treatment of aromatic L-amino acid decarboxylase deficiency
recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein / Eladocagene exuparvovec
On 18 November 2016, orphan designation (EU/3/16/1786) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein (also known as AGIL-AADC) for the treatment of aromatic L-amino acid decarboxylase deficiency.
The medicinal product has been authorised in the EU as Upstaza since 18 July 2022.
Treatment of aromatic L-amino acid decarboxylase deficiency
|Orphan designation status||
|EU designation number||
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Review of designation
More information is available in the orphan medicine assessment report.
|December 2018||The sponsorship was transferred to PTC Therapeutics International Limited, Ireland, in December 2018.|
|September 2018||The sponsorship was transferred to Voisin Consulting S.A.R.L, France, in September 2018.|
|October 2017||The sponsorship was transferred to Diamond BioPharm Limited, United Kingdom, in October 2017.|
Documents related to this orphan designation evaluation
EU/3/16/1786: Public summary of positive opinion for orphan designation of recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein for the treatmen... (PDF/131.38 KB)
First published: 14/12/2016
Last updated: 14/12/2016
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: