PTC Therapeutics' Kebilidi Gene Therapy for AADC Deficiency Gains FDA Approval, Stock Up 3.3%

Nov 14, 2024, 01:23 PM

PTC Therapeutics has received FDA approval for its gene therapy targeting aromatic L-amino acid decarboxylase (AADC) deficiency, an ultra-rare and fatal enzyme disorder. The therapy, named Kebilidi, is the first of its kind approved for treating both adult and pediatric patients with this condition. Following the announcement, PTC Therapeutics' stock price rose by 3.3% in pre-market trading.