FDA Approves Kebilidi Gene Therapy for the Treatment of AADC Deficiency
PTC Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain.
- The gene therapy, which will be marketed in the United States 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.
- AADC deficiency is a highly morbid and life-shortening rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function. Kebilidi is a gene replacement therapy that is directly administered to the putamen of the brain through a stereotactic neurosurgical procedure.
