Table of contents
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: ema.europa.eu/medicines/human/orphan-designations/eu3161786.
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.
Upstaza : EPAR - Medicine overview (PDF/102.94 KB)
First published: 09/08/2022
Upstaza : EPAR - Risk management plan summary (PDF/172.29 KB)
First published: 09/08/2022
|Agency product number||
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Amino Acid Metabolism, Inborn Errors
|Anatomical therapeutic chemical (ATC) code||
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.
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.
PTC Therapeutics International Limited
|Date of issue of marketing authorisation valid throughout the European Union||
70 Sir John Rogerson's Quay
08/06/2023 Upstaza - EMEA/H/C/005352 - IB/0012
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
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.
Other alimentary tract and metabolism products
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).