FDA nod for AADC deficiency gene therapy from PTC

The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ (Nasdaq: PTCT) gene therapy for the treatment of AADC (aromatic L-amino acid decarboxylase) deficiency, making it the first-ever gene therapy approved in the USA that is directly administered to the brain.

The news sent PTC’s shares up 10.8% to $48.80 as an early reaction, but the stock fell back before formal trading commenced.

"PTC has once again pioneered a new approach to treating highly morbid neurologic diseases," said Dr Matthew Klein, chief executive of PTC Therapeutics. "I am proud of our team's unwavering commitment to achieve this important regulatory milestone. We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency in the United States," he added.