FDA Grants Historic Approval for PTC's Brain-Delivered Gene Therapy Kebilidi Targeting AADC Deficiency

The FDA approval of PTC Therapeutics' Kebilidi marks a historic achievement as the first gene therapy administered directly into the brain for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and fatal genetic disorder[1][2]. This accelerated approval follows the successful demonstration of therapy improvements in motor development and dopamine production by introducing a functioning DDC gene to the brain’s putamen[1]. The decision by the FDA was informed by a single-arm study where 67% of the patients reached new motor milestones at 48 weeks[2]. This approval is pivotal for PTC as the company has faced prior challenges, and it now offers a potential market advantage, boosted by a rare pediatric disease priority review voucher[3].