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

Approval Year

Substance Class Nucleic Acid
Created
by admin
on Sat Dec 16 13:49:24 GMT 2023
Edited
by admin
on Sat Dec 16 13:49:24 GMT 2023
Nucleic Acid Type OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type COMPLETE
Record UNII
AIW6036FAS
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ETEPLIRSEN
INN   USAN   WHO-DD  
INN   USAN  
Official Name English
Eteplirsen [WHO-DD]
Common Name English
EXONDYS 51 COMPONENT ETEPLIRSEN
Brand Name English
ETEPLIRSEN [USAN]
Common Name English
ETEPLIRSEN [ORANGE BOOK]
Common Name English
AVI-4658
Code English
eteplirsen [INN]
Common Name English
ETEPLIRSEN COMPONENT OF EXONDYS 51
Brand Name English
ETEPLIRSEN [MI]
Common Name English
Classification Tree Code System Code
WHO-ATC M09AX06
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
NDF-RT N0000191626
Created by admin on Sat Dec 16 13:49:25 GMT 2023 , Edited by admin on Sat Dec 16 13:49:25 GMT 2023
FDA ORPHAN DRUG 248407
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
Code System Code Type Description
ChEMBL
CHEMBL2108278
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
EMA ASSESSMENT REPORTS
Exondys (Refused)
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
DRUG CENTRAL
5182
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
NCI_THESAURUS
C171739
Created by admin on Sat Dec 16 13:49:25 GMT 2023 , Edited by admin on Sat Dec 16 13:49:25 GMT 2023
PRIMARY
DAILYMED
AIW6036FAS
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
EVMPD
SUB129287
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
INN
9339
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
RXCUI
1810569
Created by admin on Sat Dec 16 13:49:25 GMT 2023 , Edited by admin on Sat Dec 16 13:49:25 GMT 2023
PRIMARY
NDF-RT
N0000011392
Created by admin on Sat Dec 16 13:49:25 GMT 2023 , Edited by admin on Sat Dec 16 13:49:25 GMT 2023
PRIMARY Oligonucleotides, Antisense [Chemical/Ingredient]
WIKIPEDIA
ETEPLIRSEN
Created by admin on Sat Dec 16 13:49:25 GMT 2023 , Edited by admin on Sat Dec 16 13:49:25 GMT 2023
PRIMARY
USAN
XX-107
Created by admin on Sat Dec 16 13:49:25 GMT 2023 , Edited by admin on Sat Dec 16 13:49:25 GMT 2023
PRIMARY
MERCK INDEX
m12193
Created by admin on Sat Dec 16 13:49:25 GMT 2023 , Edited by admin on Sat Dec 16 13:49:25 GMT 2023
PRIMARY
DRUG BANK
DB06014
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
FDA UNII
AIW6036FAS
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
EPA CompTox
DTXSID701027882
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
CAS
1173755-55-9
Created by admin on Sat Dec 16 13:49:24 GMT 2023 , Edited by admin on Sat Dec 16 13:49:24 GMT 2023
PRIMARY
Sugar Site Range Site Count
morpholino 1_1-1_30 30 / 30
Linkage Site Range Site Count
Phosphoryldimethylamine 1_2-1_30 29 / 29
Related Record Type Details
BINDER->LIGAND
BINDING
TARGET -> INDUCER
Exon 51 skipping. Eteplirsen is a morpholino antisense oligomer which triggers excision of exon 51 during pre-mRNA splicing of the dystrophin RNA transcript.
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
NUCLEOTIDE SUBSTITUTION [1_1] SITE_SPECIFIC CASIMERSEN 5'-CYTOSINE MODIFICATION 8CQO07490I