PTC Therapeutics’ AADC deficiency gene therapy granted FDA approval

PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the U.S. that is directly administered to the brain. The gene therapy, which will be marketed in the U.S. with the brand name Kebilidi, is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity. Launch preparations are 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. Clinical trial results demonstrate that following gene therapy, de novo synthesis of dopamine occurs, followed by the progressive acquisition of motor development milestones. Along with the Biologics License Application approval, a Rare Disease Priority Review Voucher was granted. The company plans to monetize the voucher.