Gene therapy | |
---|---|
Target gene | Aromatic L-amino acid decarboxylase |
Clinical data | |
Other names | PTC-AADC |
ATC code | |
Legal status | |
Legal status |
|
Identifiers | |
CAS Number | |
DrugBank | |
UNII | |
KEGG |
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]
Gene therapy | |
---|---|
Target gene | Aromatic L-amino acid decarboxylase |
Clinical data | |
Other names | PTC-AADC |
ATC code | |
Legal status | |
Legal status |
|
Identifiers | |
CAS Number | |
DrugBank | |
UNII | |
KEGG |
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]