Eladocagene exuparvovec explained

Gt Target Gene:	Aromatic L-amino acid decarboxylase
Atc Prefix:	A16
Atc Suffix:	AB26
Legal Eu:	Rx-only
Cas Number:	2098615-91-7
Drugbank:	DB16780
Unii:	S51J6N56M7
Kegg:	D12407
Synonyms:	PTC-AADC

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]} It infuses the gene encoding for the human AADC enzyme into the putamen region of the brain. The subsequent expression of AADC results in dopamine production and, as a result, development of motor function in patients with AADC deficiency.

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

Eladocagene exuparvovec was approved for medical use in the European Union in July 2022.

Medical uses

Eladocagene exuparvovec is indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.

Society and culture

Eladocagene exuparvovec is the international nonproprietary name (INN).^[3]

Legal status

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.^[4] As Upstaza is an advanced therapy medicinal product, the CHMP positive opinion is based on an assessment by the Committee for Advanced Therapies. 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.^{[6] [7]}

Further reading

• Tai CH, Lee NC, Chien YH, Byrne BJ, Muramatsu SI, Tseng SH, Hwu WL . 6 . Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency . Mol Ther . 30 . 2 . 509–518 . February 2022 . 34763085 . 10.1016/j.ymthe.2021.11.005 . free . doi . 8822132 .

External links

• Web site: Eladocagene exuparvovec . U.S. National Library of Medicine . Drug Information Portal .
• Web site: PTC Bio UPSTAZA and AADC manufacturer product page.

Notes and References

1. Web site: Eladocagene exuparvovec for aromatic l-amino acid decarboxylase deficiency . National Institute for Health and Care Research . 13 January 2022 . 22 May 2022.
2. PTC Therapeutics Announces Results from Long-Term AADC Deficiency Gene Therapy Treatment Demonstrating Sustained Improvements . . 24 October 2019 . 22 May 2022.
3. ((World Health Organization)) . 2019 . International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81 . WHO Drug Information . 33 . 1 . 10665/330896 . free . World Health Organization .
4. PTC Therapeutics Receives Positive CHMP Opinion for Upstaza for the Treatment of AADC Deficiency . . 20 May 2022 . 22 May 2022.
5. Web site: Upstaza: Pending EC decision . European Medicines Agency . 19 May 2022 . 22 May 2022 . 25 May 2022 . https://web.archive.org/web/20220525181301/https://www.ema.europa.eu/en/medicines/human/summaries-opinion/upstaza . dead . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
6. Web site: Upstaza EPAR . European Medicines Agency (EMA) . 13 April 2022 . 3 March 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
7. Web site: Upstaza Product information . Union Register of medicinal products . 3 March 2023.