Posted by Michael Wonder on 14 Nov 2024
PTC Therapeutics announces FDA approval of AADC deficiency gene therapy
13 November 2024 - PTC Therapeutics announced today the US FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first ever gene therapy approved in the US that is directly administered to the brain.
The gene therapy, which will be marketed in the US with the brand name Kebilidi (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity. Launch preparations are well underway, with centres of excellence already identified and surgeons trained in the procedure to deliver the gene therapy.
