TOFERSEN

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 `Wed Apr 02 08:41:09 GMT 2025` Edited by `admin` on `Wed Apr 02 08:41:09 GMT 2025`
Nucleic Acid Type	OLIGONUCLEOTIDE
Sequence Type	COMPLETE
Record UNII	2NU6F9601K
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

TOFERSEN

Official Name

English

BIIB067

Preferred Name

English

tofersen [INN]

Common Name

English

BIIB-067

Code

English

TOFERSEN [USAN]

Common Name

English

DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))R

Systematic Name

English

ALL-P-AMBO-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)ADENYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)-P-THIOGUANYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)GUANYLYL-(3'->5')-2'-O-(2-METHOXYETHYL)-P-THIOADENYLYL-(3'->5')-P-THIOTHYMIDYLYL-

Systematic Name

English

TOFERSEN [JAN]

Common Name

English

Tofersen [WHO-DD]

Common Name

English

ISIS-SOD1RX

Code

English

Classification Tree Code System Code

FDA ORPHAN DRUG

537216

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

Code System Code Type Description

USAN

EF-06

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

DRUG BANK

DB14782

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

CAS

2088232-70-4

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

DAILYMED

2NU6F9601K

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

SMS_ID

100000183529

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

FDA UNII

2NU6F9601K

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

NCI_THESAURUS

C166584

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

INN

10804

Created by admin on Wed Apr 02 08:41:09 GMT 2025 , Edited by admin on Wed Apr 02 08:41:09 GMT 2025

PRIMARY

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

Linkage	Site Range	Site Count
Phosphate	1_3;1_5;1_17;1_19	4 / 19
Phosporothioate	1_2;1_4;1_6-1_16;1_18;1_20	15 / 19

Related Record Type Details


					5YL205692C

TOFERSEN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					2NU6F9601K

TOFERSEN

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE_SUBSTITUTION	[1_20]				2'-O-(2-METHOXYETHYL)-5-METHYLURIDINE	9Q36I4D9VB
NUCLEOSIDE BASE SUBSTITUTION	[1_8] [1_13]				5-METHYLCYTOSINE	6R795CQT4H
NUCLEOSIDE_SUBSTITUTION	[1_1] [1_16] [1_19]		C		2'-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE	AOQ54IZZ6J