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ETEPLIRSEN
AIW6036FAS
US Approved Rx (2016)
First approved in 2016
General
Record Details
Names
9
Classification
3
Identifiers
16
Subunits
1
Sugars
1
Linkages
1
Relationships
2
Active Moiety
1
Modifications
1
ETEPLIRSEN
AIW6036FAS
US Approved Rx (2016)
First approved in 2016
General
Approval Year
2016
495
Record Details
Substance Class
Nucleic Acid
Nucleic Acid Type
OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type
COMPLETE
Record UNII
AIW6036FAS
Record Status
Validated (UNII)
Record Version
27
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Names
9
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entries
Name
Type
Language
Name
Type
Language
ETEPLIRSEN
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
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Classification
3
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Classification Tree
Code System
Code
Classification Tree
Code System
Code
Other drugs for disorders of the musculo-skeletal system
ATC
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
eteplirsen
WHO-ATC
M09AX06
Established Pharmacologic Class
Established Pharmacologic Class
[EPC]
Antisense Oligonucleotide
[EPC]
NDF-RT
N0000191626
Designated/Approved
ORPHAN DRUG
Designated/Approved
Treatment of Duchenne Muscular Dystrophy.
FDA ORPHAN DRUG
248407
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Identifiers
16
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Code System
Code
Type
Description
Code System
Code
Type
Description
ChEMBL
CHEMBL2108278
PRIMARY
EMA ASSESSMENT REPORTS
Exondys (Refused)
PRIMARY
DRUG CENTRAL
5182
PRIMARY
NCI_THESAURUS
C171739
PRIMARY
DAILYMED
AIW6036FAS
PRIMARY
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Subunits
1
Sugars
1
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Sugar
Site Range
Site Count
Sugar
Site Range
Site Count
morpholino
1_1-1_30
30 / 30
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Linkages
1
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Linkage
Site Range
Site Count
Linkage
Site Range
Site Count
Phosphoryldimethylamine
1_2-1_30
29 / 29
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2
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Related Record
Type
Details
Related Record
Type
Details
6D53G0FD0Z
PLASMA PROTEIN FRACTION (HUMAN)
BINDER->LIGAND
Interaction Type:
BINDING
Amount:
[6.1 to 16.5] % (average)
60RX8439K2
DYSTROPHIN
TARGET -> INDUCER
Comments:
Exon 51 skipping. Eteplirsen is a morpholino antisense oligomer which triggers excision of exon 51 during pre-mRNA splicing of the dystrophin RNA transcript.
Amount:
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Active Moiety
1
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Related Record
Type
Details
Related Record
Type
Details
AIW6036FAS
ETEPLIRSEN
ACTIVE MOIETY
Amount:
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Modifications
1
Structural Modifications
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Modification Type
Location Site
Location Type
Residue Modified
Extent
Fragment Name
Fragment Approval
Modification Type
Location Site
Location Type
Residue Modified
Extent
Fragment Name
Fragment Approval
NUCLEOTIDE SUBSTITUTION
[1_1]
SITE_SPECIFIC
CASIMERSEN 5'-CYTOSINE MODIFICATION
8CQO07490I
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