EU approves Upstaza for aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC Therapeutics 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.
During Upstaza clinical studies, patients went from not achieving any developmental motor milestones to demonstrating clinically meaningful motor skills from as early as three months following treatment, with transformational improvements shown to continue up to ten years after treatment. In addition, cognitive skills improved in all treated patients. Upstaza also reduced symptoms that cause potentially life-threatening and morbid complications.
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