eladocagene exuparvovec

This medicine is authorised for use in the European Union.


Upstaza is a gene therapy medicine that is used in adults and children aged 18 months and older with severe aromatic L-amino acid decarboxylase (AADC) deficiency with a genetically confirmed diagnosis.

AADC deficiency is an inherited disease that affects the nervous system leading to symptoms such as developmental delays, weak muscle tone and inability to control the movement of the limbs.

AADC deficiency is rare, and Upstaza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 18 November 2016. Further information on the orphan designation can be found here:

Upstaza is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

Upstaza contains eladocagene exuparvovec, a functional version of the AADC gene within a modified virus (adeno-associated viral vector). The virus used in this medicine is not known to cause a disease in humans.

This EPAR was last updated on 09/06/2023

Authorisation details

Product details
Agency product number
Active substance
Eladocagene exuparvovec
International non-proprietary name (INN) or common name
eladocagene exuparvovec
Therapeutic area (MeSH)
Amino Acid Metabolism, Inborn Errors
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Exceptional circumstancesExceptional circumstances

This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
PTC Therapeutics International Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address

70 Sir John Rogerson's Quay
Dublin 2
D02 R296

Product information

08/06/2023 Upstaza - EMEA/H/C/005352 - IB/0012

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Product information documents contain:

You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.

Pharmacotherapeutic group

  • Other alimentary tract and metabolism products

  • Enzymes

Therapeutic indication

Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L amino acid decarboxylase (AADC) deficiency with a severe phenotype (see section 5.1).

Assessment history

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