onasemnogene abeparvovec 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | 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

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

DateAgencyCompanyOrphan
May 24, 2019 FDA NOVARTIS PHARMS CORP
May 18, 2020 EMA Novartis Europharm Limited

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 353.26 45.47 129 2703 102808 90522807
Alanine aminotransferase increased 282.72 45.47 114 2718 118224 90507391
Troponin I increased 209.03 45.47 40 2792 2515 90623100
Pyrexia 196.04 45.47 162 2670 660323 89965292
Thrombocytopenia 175.57 45.47 98 2734 208627 90416988
Vomiting 124.99 45.47 137 2695 788156 89837459
Transaminases increased 117.38 45.47 44 2788 37148 90588467
Thrombotic microangiopathy 106.47 45.47 33 2799 15710 90609905
Platelet count decreased 97.97 45.47 58 2774 136911 90488704
Blood lactate dehydrogenase increased 94.05 45.47 34 2798 25898 90599717
Troponin T increased 87.80 45.47 18 2814 1605 90624010
Scoliosis 83.35 45.47 24 2808 8883 90616732
Troponin increased 77.98 45.47 25 2807 13256 90612359
Liver function test increased 63.38 45.47 37 2795 84818 90540797
Proteinuria 50.41 45.47 21 2811 23270 90602345
Hypertransaminasaemia 47.25 45.47 18 2814 15807 90609808

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
Aspartate aminotransferase increased 305.87 38.35 129 2470 77034 42541702
Alanine aminotransferase increased 250.89 38.35 117 2482 88289 42530447
Troponin I increased 245.67 38.35 53 2546 3093 42615643
Pyrexia 165.21 38.35 157 2442 393885 42224851
Hepatic enzyme increased 127.49 38.35 60 2539 45724 42573012
Vomiting 95.89 38.35 102 2497 289621 42329115
Transaminases increased 91.14 38.35 43 2556 32842 42585894
Blood lactate dehydrogenase increased 81.27 38.35 37 2562 26044 42592692
Liver function test increased 80.58 38.35 34 2565 19964 42598772
Rhinovirus infection 74.81 38.35 24 2575 6524 42612212
Thrombocytopenia 69.90 38.35 71 2528 190208 42428528
Platelet count decreased 53.15 38.35 53 2546 138528 42480208
Increased bronchial secretion 50.35 38.35 14 2585 2332 42616404
Respiratory syncytial virus infection 42.12 38.35 16 2583 7136 42611600
Troponin increased 41.01 38.35 21 2578 19041 42599695
Bronchiolitis 39.27 38.35 13 2586 3893 42614843

FDA Adverse Event Reporting System (Geriatric)

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 534.56 43.67 208 4062 159728 110425301
Alanine aminotransferase increased 432.43 43.67 186 4084 183587 110401442
Troponin I increased 235.02 43.67 52 4218 5400 110579629
Pyrexia 230.92 43.67 223 4047 898042 109686987
Thrombocytopenia 198.40 43.67 138 4132 348747 110236282
Transaminases increased 167.72 43.67 70 4200 63320 110521709
Blood lactate dehydrogenase increased 148.41 43.67 58 4212 44276 110540753
Vomiting 138.45 43.67 172 4098 910503 109674526
Platelet count decreased 137.36 43.67 94 4176 230349 110354680
Hepatic enzyme increased 95.61 43.67 87 4183 320805 110264224
Thrombotic microangiopathy 93.51 43.67 37 4233 29112 110555917
Rhinovirus infection 87.88 43.67 28 4242 11761 110573268
Liver function test increased 82.46 43.67 48 4222 88500 110496529
Troponin T increased 71.24 43.67 19 4251 4287 110580742
Hypertransaminasaemia 54.85 43.67 24 4246 24231 110560798
Atelectasis 54.06 43.67 27 4243 36685 110548344
Scoliosis 53.97 43.67 18 4252 8715 110576314
Troponin increased 53.21 43.67 25 4245 29741 110555288
Respiratory syncytial virus infection 52.14 43.67 20 4250 14464 110570565
Increased bronchial secretion 49.46 43.67 14 4256 3934 110581095
Bronchiolitis 44.89 43.67 14 4256 5479 110579550

FDA Adverse Event Reporting System (Pediatric)

None

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

Related Drugs by ATC class:

M09AX Other drugs for disorders of the musculo-skeletal system 11 drugs
aceneuramic acidatalurencasimerseneteplirsengivinostatgolodirsenhyaluronic acidnusinersenpalovarotenerisdiplamviltolarsen

Drug Use | Suggest Off label Use Form| |View source of the data|

DiseaseRelationSNOMED_IDDOID
Spinal muscular atrophy indication 5262007 DOID:12377




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

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
MLU3LU3EVV UNII
C5140028 UMLSCUI
CHEBI:756746 CHEBI
CHEMBL4297240 ChEMBL_ID
DB15528 DRUGBANK_ID
D11559 KEGG_DRUG
10619 INN_ID
C000710948 MESH_SUPPLEMENTAL_RECORD_UI
788110002 SNOMEDCT_US
789426004 SNOMEDCT_US
36975 MMSL
36979 MMSL
d09287 MMSL
018004 NDDF
1922968-73-7 SECONDARY_CAS_RN
2170225 RXNORM

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
ITVISMA HUMAN PRESCRIPTION DRUG LABEL 1 71894-200 INJECTION, SUSPENSION 40000000000000 [arbU] INTRATHECAL BLA 30 sections