MHRA (UK) approves Upstaza to treat aromatic L-amino acid decarboxylase (AADC) deficiency.- PTC Therapeutics
PTC Therapeutics, Inc. announced that Upstaza (eladocagene exuparvovec) 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.
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.
Related news and insights
The European Medicines Agency (EMA) has validated two marketing authorisation applications (MAAs) for AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan (Dato-DXd) in two types of cancer
Bayer has acquired the exclusive marketing rights for acoramidis in Europe from Eidos Therapeutics Inc., BridgeBio International GmbH and BridgeBio Europe B.V.
Novartis presented new data that continue to support the clinical benefits of Zolgensma (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA)