November 19, 2024

The U.S. Food and Drug Administration (FDA) has granted fast-track approval to Kebilidi (Eladocagene exuparvovec), the first gene therapy in the United States administered via direct intracerebral injection. This approval represents a significant advance in the treatment landscape for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and debilitating neurogenetic disorder.