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Eladocagene exuparvovec
Upstaza (eladocagene exuparvovec) is a gene pharmaceutical. Eladocagene exuparvovec was first approved as Upstaza on 2022-07-18. It has been approved in Europe to treat inborn errors amino acid metabolism.
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Commercial
Therapeutic Areas
Therapeutic Area
MeSH
hereditary congenital and neonatal diseases and abnormalitiesD009358
nutritional and metabolic diseasesD009750
Trade Name
FDA
EMA
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Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
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Labels
FDA
EMA
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Indications
FDA
EMA
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Agency Specific
FDA
EMA
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Patent Expiration
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ATC Codes
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HCPCS
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Clinical
Clinical Trials
1 clinical trials
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Indications Phases 4
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Indications Phases 3
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Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Argininosuccinic aciduriaD056807—1———1
Indications Phases 1
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Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameELADOCAGENE EXUPARVOVEC
INNeladocagene exuparvovec
Description
Eladocagene exuparvovec, sold under the brand name Upstaza, is a gene therapy product for the treatment of aromatic L‑amino acid decarboxylase (AADC) deficiency. 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.
Classification
Gene
Drug class—
Image (chem structure or protein)
Structure (InChI/SMILES or Protein Sequence)—
Identifiers
PDB—
CAS-ID—
RxCUI—
ChEMBL IDCHEMBL4298189
ChEBI ID—
PubChem CID—
DrugBankDB16780
UNII IDS51J6N56M7 (ChemIDplus, GSRS)
Target
Agency Approved
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Alternate
No data
Variants
Clinical Variant
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Financial
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 27 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
0 adverse events reported
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