Sefaxersen

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 `Tue Apr 01 22:56:22 GMT 2025` Edited by `admin` on `Tue Apr 01 22:56:22 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	T87K47QVF3
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

IONIS-FB-LRX FREE ACID

Preferred Name

English

Sefaxersen

Official Name

English

DNA,d(P-thio)([2?-O-(2-methoxyethyl)]rA-[2?-O-(2-methoxyethyl)]m5rU-[2?-O-( 2-methoxyethyl)]m5rC-[2?-O-(2-methoxyethyl)]m5rC-[2?-O-(2-methoxyethyl) ]m5rC-A-m5C-G-m5C-m5C-m5C-m5C-T-G-T-[2?-O-(2-methoxyethyl)]m5rC-[2?-O-( 2-methoxyethyl)]m5rC-[2?-O-(2-meth

Systematic Name

English

SEFAXERSEN [USAN]

Common Name

English

sefaxersen [INN]

Common Name

English

all-P-ambo-5?-O-{[(6-{5-[(tris{3-[6-(2-acetamido-2-deoxy-?-D-galactopyranosyloxy)hexylamino]-3-oxopropoxymethyl})methyl]amino-5-oxopentanamido}hexyl)]phospho}-2?-O-(2-methoxyethyl)-P-thioadenylyl-(3?-O?5?-O)-2?-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-

Systematic Name

English

Code System Code Type Description

CAS

2272975-74-1

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

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SMS_ID

300000041925

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

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USAN

KL-233

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

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NCI_THESAURUS

C203039

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

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FDA UNII

T87K47QVF3

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

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INN

12845

Created by admin on Tue Apr 01 22:56:22 GMT 2025 , Edited by admin on Tue Apr 01 22:56:22 GMT 2025

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Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_6-1_15	10 / 20
2'-O-Methoxyethyl ribose	1_1-1_5;1_16-1_20	10 / 20

Linkage	Site Range	Site Count
Phosporothioate	1_1-1_19	19 / 19

Related Record Type Details


					3635PCK4HN

COMPLEMENT FACTOR B

TARGET->INHIBITOR OF EXPRESSION

Amount:

Related Record Type Details


					T87K47QVF3

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_3] [1_4] [1_5] [1_7] [1_9] [1_10] [1_11] [1_12] [1_16] [1_17] [1_20]	SITE_SPECIFIC			5-METHYLCYTOSINE	6R795CQT4H
NUCLEOSIDE_SUBSTITUTION	[1_1]	5'-TERMINUS			UNSPECIFIED SUBSTANCE	6174f862 (not in database)
NUCLEOSIDE BASE SUBSTITUTION	[1_2]	SITE_SPECIFIC			THYMINE	QR26YLT7LT

Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
MOL_WEIGHT:NUMBER(CALCULATED)	CHEMICAL