ETEPLIRSEN

Approval Year

Substance Class	Nucleic Acid
Nucleic Acid Type	OLIGONUCLEOTIDE
Nucleic Acid Subtype
Sequence Type	COMPLETE
Record UNII	AIW6036FAS
Record Status	`Validated (UNII)`
Record Version

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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 Tree

Code System

Code

Other drugs for disorders of the musculo-skeletal system

WHO-ATC

M09AX06

Established Pharmacologic Class

NDF-RT

N0000191626

Designated/Approved

FDA ORPHAN DRUG

248407

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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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Subunit 0

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Sugar	Site Range	Site Count
morpholino	1_1-1_30	30 / 30

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Linkage	Site Range	Site Count
Phosphoryldimethylamine	1_2-1_30	29 / 29

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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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Related Record

Type

Details