- The European Commission (EC) approved PTC Therapeutics' ( NASDAQ: PTCT ) one-time gene replacement therapy Upstaza to treat aromatic L-amino acid decarboxylase (AADC) deficiency in children and adults.
- The company said Upstaza (eladocagene exuparvovec) is the first approved disease-modifying treatment for AADC deficiency and the first marketed gene therapy directly infused into the brain. It was approved for patients 18 months and older.
- The EC approval follows a positive opinion by a panel of the European Medicines Agency (EMA) in May for the therapy.
- The use of ClearPoint Neuro's ( CLPT ) CE Marked SmartFlow Neuro Ventricular Cannula for minimally invasive infusion of the gene therapy was included in the submission for administration of Upstaza.
- AADC deficiency is an ultra-rare, inherited genetic disease which usually shows up within the first year of birth. Patients with AADC experience developmental delays, weak muscle tone and inability to control the movement of the limbs. As per estimates, the condition affects 1 in 118K people in the EU, according to the EMA.
For further details see:
PTC Therapeutics gene therapy Upstaza wins approval in EU for ultra-rare neuromuscular disorder