PTC wins gene therapy approval based on small, open-label trial

A drug approval this week illustrates the willingness of FDA’s Center for Biologics Evaluation and Research to lean into accelerated approvals of gene therapies for ultra-rare conditions, as the organization authorized use of a treatment from PTC based on data from a 13-patient study.

Late Wednesday, FDA granted accelerated approval to Kebilidi eladocagene exuparvovec to treat aromatic L-amino acid decarboxylase (AADC) deficiency, making it the first gene therapy approved by the agency for that disorder...