Givosiran

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 `Mon Mar 31 23:12:00 GMT 2025` Edited by `admin` on `Mon Mar 31 23:12:00 GMT 2025`
Sequence Type	OLIGONUCLEOTIDE
Record UNII	ROV204583W
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

Givosiran

Official Name

English

givosiran [INN]

Preferred Name

English

GIVOSIRAN [USAN]

Common Name

English

ALN-AS1

Code

English

duplex of [(2S,4R)-1-{1-[(2-acetamido-2-deoxy-?-D-galactopyranosyl)oxy]-16,16-bis({3-[(3-{5-[(2-acetamido-2-deoxy-?-D-galactopyranosyl)oxy]pentanamido}propyl)amino]-3- oxopropoxy}methyl)-5,11,18-trioxo-14-oxa-6,10,17-triazanonacosan-29-oyl}-4-hydroxypyrr

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-?-D-galactopyranosyl]oxy]- 14,14-bis[

Systematic Name

English

Classification Tree Code System Code

NCI_THESAURUS

C307

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

FDA ORPHAN DRUG

535516

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

Code System Code Type Description

USAN

KL-44

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

NCI_THESAURUS

C146805

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

MERCK INDEX

m12182

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

CAS

1639325-43-1

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

INN

10280

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

SMS_ID

100000175520

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

RXCUI

2265712

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

WIKIPEDIA

Givosiran

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

DRUG CENTRAL

5354

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

EPA CompTox

DTXSID701027901

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

DAILYMED

ROV204583W

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

FDA UNII

ROV204583W

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

PRIMARY

DRUG BANK

DB15066

Created by admin on Mon Mar 31 23:12:00 GMT 2025 , Edited by admin on Mon Mar 31 23:12:00 GMT 2025

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


					T9O2IHM7K4

5-AMINOLEVULINATE SYNTHASE, NONSPECIFIC, MITOCHONDRIAL

TARGET->INHIBITOR OF EXPRESSION

Comments:

Results in downregulation of ALAS1 mRNA and prevents accumulation of neurotoxic ?-aminolevulinic acid and porphobilinogen levels that are associated with acute porphyria attacks.

Amount:


					5XE21E41RT

GIVOSIRAN SODIUM

SALT/SOLVATE -> PARENT

Amount:

Related Record Type Details


					ROV204583W

Givosiran

ACTIVE MOIETY

Amount:

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