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 Sat Dec 16 10:21:31 GMT 2023
Edited
by admin
on Sat Dec 16 10:21:31 GMT 2023
Nucleic Acid Subtype
Sequence Type OLIGONUCLEOTIDE
Record UNII
ROV204583W
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
Givosiran
USAN   INN  
Official Name English
GIVOSIRAN [USAN]
Common Name English
ALN-AS1
Code English
duplex of [(2S,4R)-1-{1-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-16,16-bis({3-[(3-{5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanamido}propyl)amino]-3- oxopropoxy}methyl)-5,11,18-trioxo-14-oxa-6,10,17-triazanonacosan-29-oyl}-4-hydroxypy
Systematic Name English
GIVOSIRAN [MI]
Common Name English
Givosiran [WHO-DD]
Common Name English
RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)C-Am-Um-Cm-Um-Um-Am), 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]- 14,14-bis
Systematic Name English
givosiran [INN]
Common Name English
Classification Tree Code System Code
NCI_THESAURUS C307
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
FDA ORPHAN DRUG 535516
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
Code System Code Type Description
USAN
KL-44
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
NCI_THESAURUS
C146805
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
MERCK INDEX
m12182
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
CAS
1639325-43-1
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
INN
10280
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
RXCUI
2265712
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
WIKIPEDIA
Givosiran
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
DRUG CENTRAL
5354
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
EPA CompTox
DTXSID701027901
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
DAILYMED
ROV204583W
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
FDA UNII
ROV204583W
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
DRUG BANK
DB15066
Created by admin on Sat Dec 16 10:21:31 GMT 2023 , Edited by admin on Sat Dec 16 10:21:31 GMT 2023
PRIMARY
Sugar Site Range Site Count
2'-Fluoro-Ribose 1_2-1_4;1_6;1_8;1_10;1_12;1_14;1_16;1_18;1_20;2_7;2_9;2_11;2_13;2_15;2_22 17 / 45
2'-O-Methyl-Ribose 1_1;1_5;1_7;1_9;1_11;1_13;1_15;1_17;1_19;1_21-1_23;2_1-2_6;2_8;2_10;2_12;2_14;2_16-2_21 28 / 45
Linkage Site Range Site Count
Phosporothioate 1_2-1_3;1_22-1_23;2_4-2_5 6 / 43
Phosphate 1_4-1_21;2_4-2_22 37 / 43
Related Record Type Details
TARGET->INHIBITOR OF EXPRESSION
Results in downregulation of ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels that are associated with acute porphyria attacks.
SALT/SOLVATE -> PARENT
Related Record Type Details
ACTIVE MOIETY

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
NUCLEOTIDE SUBSTITUTION [2_22] X CEMDISIRAN, TERMINAL SUGAR MODIFICATION- CQQ59545BV
Name Property Type Amount Referenced Substance Defining Parameters References
MOL_WEIGHT:NUMBER(CALCULATED) CHEMICAL