PTC Therapeutics, Inc. (NASDAQ: PTCT) today 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.
"We are thrilled with the MHRA's rapid authorization of Upstaza," said CEO Stuart W. Peltz, "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.
Upstaza is a one-time gene replacement therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L–amino acid decarboxylase deficiency with a severe phenotype.
PTCT shares ditched a dime Thursday to $37.29.
Related Stories
