Eladocagene exuparvovec

Eladocagene exuparvovec is an experimental 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]

Eladocagene exuparvovec
Gene therapy
Target geneAromatic L-amino acid decarboxylase
Clinical data
Other namesPTC-AADC
ATC code
  • None
Identifiers
CAS Number
DrugBank
UNII
KEGG

The most common side effects include initial insomnia, irritability and dyskinesia.[1]

As of May 2022, eladocagene exuparvovec is recommended for approval by the European Commission.[1][4]

Society and culture

Eladocagene exuparvovec is the international nonproprietary name (INN).[5]

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.[1][4] As Upstaza is an advanced therapy medicinal product, the CHMP positive opinion is based on an assessment by the Committee for Advanced Therapies.[1] The applicant for this medicinal product is PTC Therapeutics International Limited.[1]

Figures
The principle of action of eladocagene exuparvovec, from Tai et al., 2022.[6]

References
  1. "Upstaza: Pending EC decision". European Medicines Agency. 19 May 2022. Retrieved 22 May 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  2. "Eladocagene exuparvovec for aromatic l-amino acid decarboxylase deficiency". National Institute for Health and Care Research. 13 January 2022. Retrieved 22 May 2022.
  3. "PTC Therapeutics Announces Results from Long-Term AADC Deficiency Gene Therapy Treatment Demonstrating Sustained Improvements". PTC Therapeutics (Press release). 24 October 2019. Retrieved 22 May 2022.
  4. "PTC Therapeutics Receives Positive CHMP Opinion for Upstaza for the Treatment of AADC Deficiency". PTC Therapeutics (Press release). 20 May 2022. Retrieved 22 May 2022.
  5. World Health Organization (2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information. 33 (1). hdl:10665/330896.
  6. Tai CH, Lee NC, Chien YH, Byrne BJ, Muramatsu SI, Tseng SH, Hwu WL (February 2022). "Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency". Molecular Therapy. 30 (2): 509–518. doi:10.1016/j.ymthe.2021.11.005. PMC 8822132. PMID 34763085.

Further reading


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