- First-ever FDA approval for gene therapy directly administered to the brain -

- Priority review voucher granted -

- Broad label including children and adults -

- PTC pioneers new approach for CNS drug delivery -

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain.

"PTC has once again pioneered a new approach to treating highly morbid neurologic diseases," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "I am proud of our team's unwavering commitment to achieve this important regulatory milestone. We look forward to bringing this transformational gene therapy to children and adults with AADC deficiency in the United States."

The gene therapy, which will be marketed in the United States with the brand name KEBILIDI™ (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity. Launch preparations are well underway, with centers of excellence already identified and surgeons trained in the procedure to deliver the gene therapy. 

AADC deficiency is a highly morbid and life-shortening rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function. KEBILIDI is a gene replacement therapy that is directly administered to the putamen of the brain through a stereotactic neurosurgical procedure. Clinical trial results demonstrate that following gene therapy, de novosynthesis of dopamine occurs, followed by the progressive acquisition of motor development milestones. 

KEBILIDI received accelerated approval based on the safety and clinical efficacy findings in the ongoing global clinical trial of the gene therapy (PTC-AADC-GT-002). Confirmatory evidence will be provided from the long-term follow up of patients already treated in the study.

Along with the Biologics License Application approval, a Rare Disease Priority Review Voucher was granted. The company plans to monetize the voucher.