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

Overview

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.

Key facts

Active substance
  • recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein
  • Eladocagene exuparvovec
Intended use
Treatment of aromatic L-amino acid decarboxylase deficiency
Orphan designation status
Positive
EU designation number
EU/3/16/1786
Date of designation
18/11/2016
Sponsor

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

Review of designation

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

The Committee for Orphan Medicinal Products reviewed the orphan designation of Upstaza at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the orphan medicine assessment report.

Update history

DateUpdate
December 2018The sponsorship was transferred to PTC Therapeutics International Limited, Ireland, in December 2018.
September 2018The sponsorship was transferred to Voisin Consulting S.A.R.L, France, in September 2018.
October 2017The sponsorship was transferred to Diamond BioPharm Limited, United Kingdom, in October 2017.

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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