FDA Approves AADC Deficiency Gene Therapy by PTC Therapeutics
PTC Therapeutics, Inc. has secured FDA accelerated approval for KEBILIDI (eladocagene exuparvovec-tneq), marking a historic milestone as the first-ever gene therapy directly administered to the brain in the United States. This innovative therapy addresses aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and life-threatening genetic disorder.
"PTC has once again pioneered a new approach to treating highly morbid neurologic diseases," said Dr. Matthew B. Klein, CEO of PTC Therapeutics. He emphasized the company’s commitment to delivering transformative treatments to patients with thi...