eladocagene exuparvovec

Eladocagene exuparvovec is a recombinant adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder caused by mutations in the dopa decarboxylase (DDC) gene. This condition leads to severe disability due to impaired production of neurotransmitters such as dopamine and serotonin. The therapy works by delivering a functional copy of the human DDC gene directly into the putamen region of the brain via stereotactic neurosurgical infusion. This enables nerve cells to produce aromatic L-amino acid decarboxylase enzyme, restoring neurotransmitter synthesis and improving motor function in affected patients[1][2][5][7][8]. Developed by PTC Therapeutics, eladocagene exuparvovec is administered as a single-dose intraputaminal infusion during neurosurgery. It was approved under accelerated approval based on improvement in gross motor milestones at 48 weeks post-treatment[3][7]. The brand name "Kebilidi" is used in the US; "Upstaza" is used elsewhere[3][5]. It has orphan drug designation for AADC deficiency.

Brand names

KebilidiUpstaza

Other names

eladocagene exuparvovec-tneq