Eladocagene Exuparvovec for the Treatment of Aromatic l-Amino Acid Decarboxylase Deficiency (AADCd): An Economic Evaluation from a US Perspective

Eladocagene exuparvovec is associated with considerable QALY gains compared with BSC. Within the context of other ultra-rare and/or one-time treatments, eladocagene exuparvovec provides substantial clinical improvements at lower cost than many other rare-disease treatments. Findings from this study highlight that eladocagene exuparvovec is an important treatment option for patients with AADCd.