PTC Therapeutics has secured FDA accelerated approval for Kebilidi (eladocagene exuparvovec-tneq), a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, marking the first U.S. approval for a gene therapy directly administered to the brain.
The therapy, authorized for both children and adults, addresses a severe genetic disorder that impairs dopamine production, essential for motor function.
Kebilidi’s approval is based on safety and efficacy findings from ongoing clinical trials. Patients treated with the therapy demonstrated dopamine synthesis and improvements in motor development milestones. The therapy is administered through a minimally invasive neurosurgical procedure targeting the brain's putamen.
AADC deficiency is a rare, debilitating condition that significantly impacts quality of life and often requires extensive supportive care.
Symptoms include severe motor impairments and life-threatening complications, such as infections and respiratory issues. Kebilidi aims to address these challenges by directly targeting the disorder’s underlying genetic cause.
With the FDA approval, PTC also received a rare disease priority review voucher, which the company intends to monetize.