onasemnogene abeparvovec Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definitionDrug idCAS RN
5332 None

Description:

MoleculeDescription

Synonyms:

  • onasemnogene abeparvovec
  • onasemnogene abeparvovec-xioi
  • zolgensma
  • AVXS-101
ZOLGENSMA is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Intravenous administration of ZOLGENSMA that results in cell transduction and expression of the SMN protein has been observed in two human case studies
  • Molecular weight:
  • Formula:
  • CLOGP:
  • LIPINSKI: None
  • HAC: None
  • HDO: None
  • TPSA:
  • ALOGS:
  • ROTB: None

Drug dosage:

None

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
May 24, 2019 FDA NOVARTIS PHARMS CORP

FDA Adverse Event Reporting System (Female)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Aspartate aminotransferase increased 169.16 64.49 59 718 78641 46606644
Alanine aminotransferase increased 143.82 64.49 54 723 88397 46596888
Troponin I increased 109.28 64.49 20 757 1880 46683405
Pyrexia 85.49 64.49 59 718 348743 46336542
Thrombocytopenia 74.87 64.49 38 739 126543 46558742
Hepatic enzyme increased 64.68 64.49 30 747 81757 46603528

FDA Adverse Event Reporting System (Male)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Troponin I increased 111.35 82.92 20 450 1813 29950195
Aspartate aminotransferase increased 88.25 82.92 34 436 63388 29888620

Pharmacologic Action:

SourceCodeDescription
ATC M09AX09 MUSCULO-SKELETAL SYSTEM
OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
Other drugs for disorders of the musculo-skeletal system

Drug Use (View source of the data)

DiseaseRelationSNOMED_IDDOID
Spinal muscular atrophy indication 5262007 DOID:12377

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

None

External reference:

IDSource
1922968-73-7 SECONDARY_CAS_RN
C5140028 UMLSCUI
CHEMBL4297240 ChEMBL_ID
D11559 KEGG_DRUG
DB15528 DRUGBANK_ID
C000710948 MESH_SUPPLEMENTAL_RECORD_UI
10619 INN_ID
MLU3LU3EVV UNII
2170225 RXNORM
36975 MMSL
36979 MMSL
d09287 MMSL
018004 NDDF
788110002 SNOMEDCT_US
789426004 SNOMEDCT_US

Pharmaceutical products:

None