Eladocagene exuparvovec, sold under the brand name Upstaza, is a gene therapy product for the treatment of aromatic L‑amino acid decarboxylase (AADC) deficiency.[1][2][3] It infuses the gene encoding for the human AADC enzyme into the putamen region of the brain.[1][2] The subsequent expression of AADC results in dopamine production and, as a result, development of motor function in patients with AADC deficiency.[1]
The most common side effects include initial insomnia, irritability and dyskinesia.[1]
Eladocagene exuparvovec was approved for medical use in the European Union in July 2022.[1]
Eladocagene exuparvovec is indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.[1]
Eladocagene exuparvovec is the international nonproprietary name (INN).[4]
On 19 May 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization under exceptional circumstances for the medicinal product Upstaza, intended for the treatment of aromatic L‑amino acid decarboxylase (AADC) deficiency.[5][6] As Upstaza is an advanced therapy medicinal product, the CHMP positive opinion is based on an assessment by the Committee for Advanced Therapies.[5] The applicant for this medicinal product is PTC Therapeutics International Limited.[5] Eladocagene exuparvovec was approved for medical use in the European Union in July 2022.[1][7]
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