EMA — authorised 18 July 2022
- Application: EMEA/H/C/005352
- Marketing authorisation holder: PTC Therapeutics International Limited
- Local brand name: Upstaza
- 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).
- Pathway: exceptional circumstances, orphan, ATMP
- Status: approved