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

Approval Year

2023
446
Name Type Language
TOFERSEN
USAN   INN  
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
ACTIVE MOIETY
TOFERSEN
SALT/SOLVATE (PARENT)
TOFERSEN SODIUM
NIH
NCATS
PRIVACY NOTICE
ACCESSIBILITY
DISCLAIMER
HHS VULNERABILITY DISCLOSURE
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National Center for Advancing Translational Sciences (NCATS),
6701 Democracy Boulevard, Bethesda MD 20892-4874 • 301-594-8966