PTC Therapeutics Granted Marketing Authorization By EU For Upstaza To Treat AADC Deficiency
(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced Wednesday that Upstaza (eladocagene exuparvovec) was granted marketing authorization by the European Commission. Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older.
Upstaza is a one-time gene replacement therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype.
The marketing authorization is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.
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