Posted by Michael Wonder on 22 Jul 2022
Upstaza granted marketing authorisation by European Commission as first disease-modifying treatment for AADC deficiency
20 July 2022 - PTC Therapeutics to launch first gene therapy directly administered into the brain.
PTC Therapeutics today announced that Upstaza (eladocagene exuparvovec) was granted marketing authorisation by the European Commission.
Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older.
