U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

2019
334
Substance Class Structurally Diverse
Created
by admin
on Wed Apr 02 01:43:26 GMT 2025
Edited
by admin
on Wed Apr 02 01:43:26 GMT 2025
Source Materials Class ORGANISM
Source Materials Type VIRUS
Source Materials State LIVE GENETICALLY MODIFIED NON-REPLICATING
Organism Family Parvoviridae
Organism Genus Dependoparvovirus
Organism Species adeno-associated dependoparvovirus a
Infraspecific Type RECOMBINANT VECTOR
Infraspecific Name AAV-9/SMN RECOMBINANT
Part whole
Record UNII
MLU3LU3EVV
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ONASEMNOGENE ABEPARVOVEC
INN   USAN  
Official Name English
ZOLGENSMA
Preferred Name English
DNA (SYNTHETIC ADENO-ASSOCIATED VIRUS 9 VECTOR SCAAV9.CB.HSMN HUMAN SURVIVOR MOTOR NEURON PROTEIN-SPECIFYING)
Systematic Name English
NON-REPLICATING RECOMBINANT SELF-COMPLEMENTARY (SC) ADENO-ASSOCIATED VIRUS SEROTYPE 9 (AAV9) VECTOR CONTAINING THE CDNA OF THE SURVIVAL OF MOTOR NEURON 2 (SMN2) GENE UNDER THE CONTROL OF THE HYBRID CYTOMEGALOVIRUS (CMV) ENHANCER/CHICKEN BETA-ACTIN PROMOT
Common Name English
ONASEMNOGENE ABEPARVOVEC [USAN]
Common Name English
AVXS-101
Code English
Onasemnogene abeparvovec [WHO-DD]
Common Name English
ONASEMNOGENE ABEPARVOVEC [MI]
Common Name English
onasemnogene abeparvovec [INN]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 444714
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
Code System Code Type Description
SMS_ID
100000177723
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
DRUG CENTRAL
5332
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
DRUG BANK
DB15528
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
WIKIPEDIA
Onasemnogene abeparvovec
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
INN
10619
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
CAS
1922968-73-7
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
USAN
EF-126
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
MERCK INDEX
m12206
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
FDA UNII
MLU3LU3EVV
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
NCI_THESAURUS
C174715
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
RXCUI
2170226
Created by admin on Wed Apr 02 01:43:26 GMT 2025 , Edited by admin on Wed Apr 02 01:43:26 GMT 2025
PRIMARY
Related Record Type Details
SURVIVAL MOTOR NEURON PROTEIN
EXPRESSED PROTEIN->VECTOR
Related Record Type Details
ONASEMNOGENE ABEPARVOVEC
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
INSERTED GENE EXPRESSION VECTOR UNSPECIFIED SUBSTANCE
75aa285d
(not in database)
VIRAL CAPSID ONASEMNOGENE ABEPARVOVEC MLU3LU3EVV
NIH
NCATS
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