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.
Authors
- Wuh‐Liang Hwu (ORCID: https://orcid.org/0000-0001-6690-4879)
- Berrin Monteleone (ORCID: https://orcid.org/0000-0001-6268-4995)
- Ioannis Tomazos (ORCID: https://orcid.org/0000-0003-0290-4583)
- Thomas O’Connell (ORCID: https://orcid.org/0000-0002-4634-8649)
- Rongrong Zhang (ORCID: https://orcid.org/0000-0002-1228-9345)
- Yan Teng (ORCID: https://orcid.org/0000-0002-8850-251X)
- Paul Castellano
Institutions
- Long Island University (US)
- National Taiwan University (TW)
- China Medical University (TW)
- Pfeiffer Medical Center (US)
- China Medical University Hospital (TW)
- National Taiwan University Hospital (TW)
- PTC Therapeutics (United States) (US)
Publication Details
- Journal
- PharmacoEconomics
- Published
- 2025-09-24
- DOI
- https://doi.org/10.1007/s40273-025-01542-8
- Primary Topic
- Metabolism and Genetic Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- PTC Therapeutics