Source: European Medicines Agency (EU) Revision Year: 2023 Publisher: PTC Therapeutics International Limited, 70 Sir John Rogersons Quay, Dublin 2, Ireland
Upstaza 2.8 × 1011 vector genomes (vg)/0.5 mL solution for infusion.
| Pharmaceutical Form |
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Solution for infusion. Following thaw from frozen, the solution for infusion is a clear to slightly opaque, colourless to faint-white liquid. |
Eladocagene exuparvovec is a gene therapy medicinal product that expresses the human aromatic L-amino acid decarboxylase enzyme (hAADC). It is a non-replicating recombinant adeno-associated virus serotype 2 (AAV2) based vector containing the cDNA of the human dopa decarboxylase (DDC) gene under the control of the cytomegalovirus immediate-early promoter.
Eladocagene exuparvovec is produced in human embryonic kidney cells by recombinant DNA technology.
Each single-dose vial contains 2.8 × 1011 vg of eladocagene exuparvovec in 0.5 extractable mL of solution. Each mL of solution contains 5.6 × 1011 vg of eladocagene exuparvovec.
For the full list of excipients, see section 6.1.
| Active Ingredient | Description | |
|---|---|---|
| Eladocagene exuparvovec |
Eladocagene exuparvovec is a gene therapy based on recombinant AAV2 vector containing the human cDNA for the DDC gene. After infusion into the putamen, the product results in the expression of the AADC enzyme and subsequent production of dopamine, and consequently, development of motor function in treated AADC-deficient patients. |
| List of Excipients |
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Potassium chloride |
Type I borosilicate glass vial, with a siliconised chlorobutyl stopper with coating sealed with an aluminium/plastic cap.
Pack size of one vial.
PTC Therapeutics International Limited, 70 Sir John Rogerson’s Quay, Dublin 2, Ireland
EU/1/22/1653/001
Date of first authorisation: 18 July 2022
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