PTC Therapeutics Gains FDA Approval for Gene Therapy Targeting AADC Deficiency
Friday, November 15, 2024
PTC Therapeutics has received accelerated approval from the U.S. Food and Drug Administration (FDA) for its gene therapy, KEBILIDI™ (eladocagene exuparvovec-tneq), marking the first gene therapy in the United States administered directly into the brain.
AADC deficiency significantly affects quality of life from infancy, often causing severe disability and complications that require extensive medical support, including surgery and therapies to manage symptoms like dystonia and severe feeding and respiratory issues. By targeting the genetic cause of AADC deficiency, KEBILIDI provides a pioneering therapeutic option for patients in the United States.
This treatment is intended for both children and adults affected by aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and severe genetic disorder.
AADC deficiency prevents patients from producing dopamine, a critical neurotransmitter for motor function, leading to significant disability and shortened life expectancy.
The gene therapy is designed to deliver a working DDC gene into the brain’s putamen, restoring the production of dopamine and supporting the gradual development of motor skills.
Administered through a stereotactic neurosurgical procedure, KEBILIDI is currently being implemented by trained neurosurgeons in specialised centres.
Approval of KEBILIDI is based on clinical evidence demonstrating both safety and effectiveness in ongoing global trials (PTC-AADC-GT-002), with additional long-term data to be collected from current participants.
Alongside the approval, PTC Therapeutics has been granted a Rare Disease Priority Review Voucher, which it plans to monetise.
Source: ptcbio.com
