PTC Therapeutics’ Upstaza (eladocagene exuparvovec) has been 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.
“Today’s approval from the European Commission for Upstaza for the treatment of AADC deficiency is momentous for patients, for PTC, as well as for the larger gene therapy community,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “We are proud to bring this innovative therapy to the marketplace so that patients may benefit. Upstaza is the first and only approved disease-modifying treatment for patients living with AADC deficiency. We are ready to deliver this long-awaited treatment to patients as soon as possible.”
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.
“Before treatment, our daughter had not met any development milestones. She suffered from oculogyric crises that evolved into hours of pain, and we were told she would be bedridden for life,” said patient organization Teach RARE founder, Richard Poulin, whose daughter was treated as part of a clinical trial. “After receiving Upstaza, she is now speaking, walking, running, and even riding horses. We’re thrilled with the EMA approval and the hope that this milestone brings to other children and families impacted by AADC deficiency.”
The marketing authorization is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.
