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Review
. 2022 Sep;82(13):1427-1432.
doi: 10.1007/s40265-022-01775-3.

Eladocagene Exuparvovec: First Approval

Susan J Keam  1
Affiliations
Review

Eladocagene Exuparvovec: First Approval

Susan J Keam. Drugs. 2022 Sep.

Abstract

Eladocagene exuparvovec (Upstaza™) is a gene therapy developed by PTC Therapeutics for the treatment of human aromatic L-amino acid decarboxylase (AADC) deficiency. Eladocagene exuparvovec comprises an adeno-associated virus vector that delivers the dopa decarboxylase (DDC) gene, the gene for human AADC. Eladocagene exuparvovec was approved in July 2022 in the EU for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype (i.e. patients who cannot sit, stand or walk). This article summarizes the milestones in the development of eladocagene exuparvovec leading to this first approval for the treatment of patients aged 18 months and older with AADC deficiency.

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References

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    1. European Medicines Agency. Eladocagene exuparvovec: EU summary of product characteristics 2022. https://ec.europa.eu/health/documents/community-register/html/h1653.htm . Accessed 25 July 2022

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