Upstaza
What Upstaza is used for
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).
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
- Approved under exceptional circumstances: full data cannot be collected, usually because the disease is very rare.
Key facts
- Brand
- Upstaza
- Active substance
- eladocagene exuparvovec
- Disease area
- Amino Acid Metabolism, Inborn Errors
- Medicine group
- Other alimentary tract and metabolism products;Enzymes
- ATC code
- A16AB26
- Marketing authorisation holder
- PTC Therapeutics International Limited
- Authorised in the EU since
- 2022
Official information
In the USA
Same active substance: Kebilidi
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Upstaza used for?
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).
What is the active substance in Upstaza?
Upstaza contains eladocagene exuparvovec.
Who makes Upstaza?
The EU marketing authorisation holder is PTC Therapeutics International Limited.
Is Upstaza an orphan medicine?
Yes. Upstaza is designated an orphan medicine in the EU, for a rare disease (Amino Acid Metabolism, Inborn Errors).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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