PTC's Kebilidi Gains FDA Approval as First Brain-Delivered Gene Therapy for AADC Deficiency

Kebilidi represents a groundbreaking advancement in the field of gene therapy as it becomes the first U.S.-approved treatment that can be administered directly to the brain to address aromatic L-amino acid decarboxylase (AADC) deficiency. This rare and devastating disorder manifests in severe physical, mental, and behavioral disabilities from infancy, with only around 350 documented cases. By introducing a functional human DDC gene into the brain, Kebilidi aims to enhance enzyme activity and restore dopamine production, thus significantly improving motor development in affected individuals[1][2][3]. The therapy is administered through neurosurgical infusion directly into the brain, providing an innovative approach to correcting the underlying genetic defect. This approval highlights a broader acceptance and expansion of gene therapy applications, providing PTC Therapeutics with a crucial market advantage despite previous setbacks in its other drug development endeavors[1][3].