FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency

First FDA Approval for Direct-to-Brain Gene Therapy:
Kebilidi, developed by PTC Therapeutics, is the first gene therapy approved by the FDA that is administered directly into the brain1 3 5.

Treatment for AADC Deficiency:
Kebilidi is indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and life-shortening genetic disorder that affects both children and adults1 2 5.

Accelerated Approval:
The FDA granted accelerated approval based on the safety and clinical efficacy findings from the ongoing PTC-AADC-GT-002 global clinical trial. Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial2 4 5.

Clinical Trial Results:
The therapy demonstrated efficacy by achieving gross motor function improvement in 8 of the 12 treated patients measured at 48 weeks. Three patients achieved full head control, and two were able to sit with or without assistance1 3 5.

Priority Review Voucher:
PTC Therapeutics received a rare disease priority review voucher, which it intends to monetize2 4.

Administration:
Kebilidi is delivered through a precise neurosurgical procedure to the putamen of the brain, using the FDA-authorized SmartFlow Neuro Cannula5.

Market Impact:
The approval is seen as a positive for the broader gene therapy space, particularly for those pursuing similar routes of administration1 3.