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](/en/medicines/human/orphan-designations/eu-3-16-1786). 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.
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\).
Therapeutic Area (MeSH)
ATC Code
A16AB26
ATC Item
N/A
Pharmacotherapeutic Group
N/A
Active Substance (Summary)
INN / Common Names
EMA Name
Upstaza
Medicine Name
Upstaza
Aliases
N/ANo risk management plan link.
