PTC Therapeutics has received accelerated approval for its gene therapy, Kebilidi, which can be used for the treatment of AADC deficiency.
The treatment is the first gene therapy to be approved by the FDA for direct administration into the brain.
Kebilidi, otherwise known as eladocagene exuparvovec-tneq is suitable for both children and adults with AADC deficiency, and surgeons across the country are already being trained to deliver it.
AADC deficiency is a rare genetic disorder that shortens an individual's life significantly, as it results in the inability to synthesise dopamine — an essential neurotransmitter involved in motor function.
By acting as a gene replacement therapy, Kebilidi can initiate the de novo synthesis of dopamine, allowing patients to undergo motor development milestones.
Kebilidi received accelerated approval from the FDA based on the safety and efficacy profiles demonstrated in clinical trials thus far.
As well as receieving approval for its Biologics License Application, PTC Therapeutics has also received Rare Disease Priority Review status from the regulator.
CEO of PTC Therapeutics, Matthew Klein, commented: "PTC has once again pioneered a new approach to treating highly morbid neurologic diseases,"
"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."