FDA Approves PTC Therapeutics' Brain-Delivered Gene Therapy Kebilidi for AADC Deficiency

Nov 14, 2024, 01:23 PM

The U.S. Food and Drug Administration (FDA) has approved PTC Therapeutics' gene therapy for Aromatic L-amino acid decarboxylase (AADC) deficiency, a potentially fatal enzyme deficiency disorder. This therapy, marketed under the name Kebilidi, is the first brain-delivered gene therapy approved in the United States. The approval was granted following an open-label study involving 13 patients and the use of historical control data.