Pharma Focus Europe

Upstaza™ Gene Therapy Granted Marketing Authorization for AADC deficiency by UK's MHRA

Friday, November 18, 2022

PTC Therapeutics, Inc., announced that Upstaza™ was granted authorization by the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain. Upstaza is the first and only 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.

"We are thrilled with the MHRA's rapid authorization of Upstaza," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "Patients in the UK with AADC deficiency are one step closer to having access to a much-needed disease modifying therapy. This is another milestone towards our commitment to advance innovative treatments and improve outcomes for people living with rare diseases."

During Upstaza clinical studies, patients went from not achieving any developmental motor milestones to demonstrating a mastery of clinically meaningful motor skills, including the ability to ambulate independently. Milestone achievements including cognitive and language acquisition occurred from as early as three months following treatment, with clinically significant improvements shown to continue up to ten years after treatment. Upstaza also reduced symptoms that cause potentially life-threatening and morbid complications.

"The approval of an AADC deficiency gene therapy in the UK will provide the opportunity to transform the prognosis for those born and living with this disease, and we are hopeful for access in the coming months. Without treatment, most children born with AADC deficiency will have difficulty with their development and many of the symptoms can be distressing and life-threatening. The impact on those living with AADC and their communities is significant, with children facing frequent hospitalizations, emergency visits and requiring a multi-disciplinary team of highly trained specialists," stated Kirsty Hoyle, CEO, Metabolic Support UK.

Upstaza has marketing authorization in all 27 European Union member states, as well as Iceland, Norway, Liechtenstein, and Northern Ireland.

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