Words by Jade Williams
The FDA has granted accelerated approval in the US for the first gene therapy delivered directly to the brain.
The drug is a treatment for a rare and fatal genetic disorder called aromatic l-amino acid decarboxylase (AADC) deficiency, which severely impairs dopamine synthesis in infancy and can lead to profound motor dysfunction.
The therapy, developed by PTC Therapeutics, is designed for patients across the severity of spectrum of AADC deficiency. It works by delivering a gene replacement directly to a patient’s putamen through a stereotactic neurosurgical procedure.
“PTC has once again pioneered a new approach to treating highly morbid neurologic diseases,” said Dr Matthew B. Klein, Chief Executive Officer, PTC Therapeutics. “We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency.”
The FDA approval was based on data from the company’s ongoing global clinical trial, which provided safety and efficacy results, with additional long-term data expected from follow-up of existing patients. The approval also granted PTC a Rare Disease Priority Review voucher, which the company intends to monetise.
PTC has completed preparations for the product’s launch in the US, including designating centres of excellence and training neurosurgeons to administer the therapy.
