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Drug ReportsAtidarsagene autotemcel
Atidarsagene autotemcel
Lenmeldy (atidarsagene autotemcel) is a gene pharmaceutical. Atidarsagene autotemcel was first approved as Lenmeldy on 2024-03-18. It is used to treat metachromatic leukodystrophy in the USA.
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Commercial
Therapeutic Areas
Therapeutic Area
MeSH
nervous system diseasesD009422
hereditary congenital and neonatal diseases and abnormalitiesD009358
nutritional and metabolic diseasesD009750
Trade Name
FDA
EMA
Lenmeldy
Drug Products
FDA
EMA
Reference product - 351(a)
Reference product - 351(a)
Interchangeable product - 351(k)
Interchangeable product - 351(k)
Biosimilar product - 351(k)
Biosimilar product - 351(k)
Atidarsagene autotemcel
Tradename
Proper name
Company
Number
Date
Products
Lenmeldyatidarsagene autotemcelOrchard TherapeuticsN-125758 RX2024-03-18
1 products
Labels
FDA
EMA
Brand Name
Status
Last Update
lenmeldyBiologic Licensing Application2025-08-12
Indications
FDA
EMA
Indication
Ontology
MeSH
ICD-10
metachromatic leukodystrophy—D007966E75.25
Agency Specific
FDA
EMA
Expiration
Code
atidarsagene autotemcel, Lenmeldy, Orchard Therapeutics (Europe) Ltd.
2036-03-18Reference product excl.
2031-03-18Orphan excl.
Patent Expiration
No data
ATC Codes
A: Alimentary tract and metabolism drugs
— A16: Other alimentary tract and metabolism products in atc
— A16A: Other alimentary tract and metabolism products in atc
— A16AB: Enzymes for alimentary tract and metabolism
— A16AB21: Atidarsagene autotemcel
HCPCS
No data
Clinical
Clinical Trials
3 clinical trials
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Indications Phases 4
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Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Metachromatic leukodystrophyD007966—E75.25121——3
Lysosomal storage diseasesD016464——121——3
Indications Phases 2
No data
Indications Phases 1
No data
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameAtidarsagene autotemcel
INNatidarsagene autotemcel
Description
Atidarsagene autotemcel, sold under the brand name Libmeldy among others, is a gene therapy treatment for metachromatic leukodystrophy developed by Orchard Therapeutics. It contains an autologous CD34⁺ cell enriched population that contains haematopoietic stem and progenitor cells transduced using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.
Classification
Gene
Drug class—
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Structure (InChI/SMILES or Protein Sequence)—
Identifiers
PDB—
CAS-ID—
RxCUI—
ChEMBL IDCHEMBL4802262
ChEBI ID—
PubChem CID—
DrugBank—
UNII IDEPP8G99QG4 (ChemIDplus, GSRS)
Target
No data
Variants
No data
Financial
No data
Trends
No data
Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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5 adverse events reported
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