EC approves PTC's gene therapy Upstaza for rare disorder

New Jersey, USA-based rare disease drug developer PTC Therapeutics (Nasdaq: PTCT) today announced 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.

The approval follows a positive recommendation from the European Medicines Agency’s advisory committee earlier this year.