Biology:Eladocagene exuparvovec
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| Target gene | Aromatic L-amino acid decarboxylase |
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| Other names | PTC-AADC |
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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]
Medical uses
Eladocagene exuparvovec is indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.[1]
Society and culture
Eladocagene exuparvovec is the international nonproprietary name (INN).[4]
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.[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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References
- ↑ 1.0 1.1 1.2 1.3 1.4 1.5 1.6 1.7 "Upstaza EPAR". 13 April 2022. https://www.ema.europa.eu/en/medicines/human/EPAR/upstaza. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ 2.0 2.1 "Eladocagene exuparvovec for aromatic l-amino acid decarboxylase deficiency". 13 January 2022. https://www.io.nihr.ac.uk/techbriefings/eladocagene-exuparvovec-for-aromatic-l-amino-acid-decarboxylase-deficiency/.
- ↑ "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.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information 33 (1). 2019.
- ↑ 5.0 5.1 5.2 "Upstaza: Pending EC decision". 19 May 2022. https://www.ema.europa.eu/en/medicines/human/summaries-opinion/upstaza. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "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.
- ↑ "Upstaza Product information". https://ec.europa.eu/health/documents/community-register/html/h1653.htm.
- ↑ "Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency". Molecular Therapy 30 (2): 509–518. February 2022. doi:10.1016/j.ymthe.2021.11.005. PMID 34763085.
Further reading
- "Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency". Mol Ther 30 (2): 509–518. February 2022. doi:10.1016/j.ymthe.2021.11.005. PMID 34763085.
External links
- "Eladocagene exuparvovec". Drug Information Portal. U.S. National Library of Medicine. https://druginfo.nlm.nih.gov/drugportal/name/eladocagene%20exuparvovec.
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