givosiran

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Stem definition	Drug id	CAS RN
small interfering RNA including siRNA, miRNA and piRNA	5354	1639325-43-1

Stem definition

Drug id

CAS RN

small interfering RNA including siRNA, miRNA and piRNA

5354

1639325-43-1

Description:

Molecule	Description
Synonyms: givosiran givosiran sodium ALN-AS1 givlaari	Givosiran is a double-stranded small interfering RNA that causes degradation of aminolevulinate synthase1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of AHP. Molecular weight: Formula: CLOGP: LIPINSKI: None HAC: None HDO: None TPSA: ALOGS: ROTB: None

Molecule

Description

Synonyms:

givosiran
givosiran sodium
ALN-AS1
givlaari

Givosiran is a double-stranded small interfering RNA that causes degradation of aminolevulinate synthase1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of AHP.

Molecular weight:
Formula:
CLOGP:
LIPINSKI: None
HAC: None
HDO: None
TPSA:
ALOGS:
ROTB: None

Property	Value	Reference
S (Water solubility)	357 mg/mL	Bocci G, Oprea TI, Benet LZ
BDDCS (Biopharmaceutical Drug Disposition Classification System)	1	Bocci G, Oprea TI, Benet LZ

Property

Value

Reference

S (Water solubility)

357 mg/mL

Bocci G, Oprea TI, Benet LZ

BDDCS (Biopharmaceutical Drug Disposition Classification System)

Bocci G, Oprea TI, Benet LZ

Date	Agency	Company
June 23, 2021	PMDA	ALNYLAM JAPAN K.K
March 2, 2020	EMA	Alnylam Netherlands B.V.
Nov. 20, 2019	FDA	ALNYLAM PHARMS INC

Date

Agency

Company

Orphan

June 23, 2021

PMDA

ALNYLAM JAPAN K.K

March 2, 2020

EMA

Alnylam Netherlands B.V.

Nov. 20, 2019

FDA

ALNYLAM PHARMS INC

Pharmacologic Action:

Source	Code	Description
ATC	A16AX16	ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Various alimentary tract and metabolism products
FDA CS	M0406100	RNA, Small Interfering
FDA PE	N0000008852	Decreased RNA Integrity
FDA EPC	N0000193848	Small Interfering RNA
FDA EPC	N0000194024	Aminolevulinate Synthase 1-directed RNA Interaction

Source

Code

Description

ATC

A16AX16

ALIMENTARY TRACT AND METABOLISM
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Various alimentary tract and metabolism products

FDA CS

M0406100

RNA, Small Interfering

FDA PE

N0000008852

Decreased RNA Integrity

FDA EPC

N0000193848

Small Interfering RNA

FDA EPC

N0000194024

Aminolevulinate Synthase 1-directed RNA Interaction

Disease	Relation	SNOMED_ID	DOID
Hepatic porphyria	indication	55056006	DOID:3133

Disease

Relation

SNOMED_ID

DOID

Hepatic porphyria

indication

55056006

DOID:3133

Orange Book patent data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent use
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	10131907	Aug. 24, 2028	TREATMENT OF ACUTE HEPATIC PORPHYRIA
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	8828956	Dec. 4, 2028	TREATMENT OF ACUTE HEPATIC PORPHYRIA
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	8106022	Dec. 12, 2029	TREATMENT OF ACUTE HEPATIC PORPHYRIA
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	10125364	March 15, 2033	TREATMENT OF ACUTE HEPATIC PORPHYRIA
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	9631193	March 15, 2033	TREATMENT OF ACUTE HEPATIC PORPHYRIA
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	9133461	Nov. 30, 2033	TREATMENT OF ACUTE HEPATIC PORPHYRIA
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	10119143	Oct. 3, 2034	TREATMENT OF ACUTE HEPATIC PORPHYRIA
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	11028392	Oct. 3, 2034	TREATMENT OF ACUTE HEPATIC PORPHYRIA

Formulation strength

Trade name

Applicant

Application number

Approval date

Type

Dose form

Route

Patent number

Patent expiration date

Patent use

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

10131907

Aug. 24, 2028

TREATMENT OF ACUTE HEPATIC PORPHYRIA

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

8828956

Dec. 4, 2028

TREATMENT OF ACUTE HEPATIC PORPHYRIA

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

8106022

Dec. 12, 2029

TREATMENT OF ACUTE HEPATIC PORPHYRIA

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

10125364

March 15, 2033

TREATMENT OF ACUTE HEPATIC PORPHYRIA

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

9631193

March 15, 2033

TREATMENT OF ACUTE HEPATIC PORPHYRIA

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

9133461

Nov. 30, 2033

TREATMENT OF ACUTE HEPATIC PORPHYRIA

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

10119143

Oct. 3, 2034

TREATMENT OF ACUTE HEPATIC PORPHYRIA

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

11028392

Oct. 3, 2034

TREATMENT OF ACUTE HEPATIC PORPHYRIA

Orange Book exclusivity data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
EQ 189MG BASE/ML (EQ 189MG BASE/ML)	GIVLAARI	ALNYLAM PHARMS INC	N212194	Nov. 20, 2019	RX	SOLUTION	SUBCUTANEOUS	Nov. 20, 2026	INDICATED FOR THE TREATMENT OF ADULTS WITH ACUTE HEPATIC PORPHYRIA (AHP)

Formulation strength

Trade name

Applicant

Application number

Approval date

Type

Dose form

Route

Exclusivity date

Description

EQ 189MG BASE/ML (EQ 189MG BASE/ML)

GIVLAARI

ALNYLAM PHARMS INC

N212194

Nov. 20, 2019

SOLUTION

SUBCUTANEOUS

Nov. 20, 2026

INDICATED FOR THE TREATMENT OF ADULTS WITH ACUTE HEPATIC PORPHYRIA (AHP)

Bioactivity Summary:

Target	Class	Pharos	UniProt	Action	Bioact source	MoA source
aminolevulinate synthase1 (ALAS1) mRNA	RNA	P13196	HEM1_HUMAN	ANTISENSE INHIBITOR	UNKNOWN	DRUG LABEL
Asialoglycoprotein receptor 1	Membrane receptor	P07306	ASGR1_HUMAN	BINDING AGENT	UNKNOWN	DRUG LABEL
Asialoglycoprotein receptor 2	Membrane receptor	P07307	ASGR2_HUMAN	BINDING AGENT	UNKNOWN	DRUG LABEL

Target

Class

Pharos

UniProt

Action

Type

Activity value
(-log[M])

Mechanism
action

Bioact source

MoA source

aminolevulinate synthase1 (ALAS1) mRNA

RNA

P13196

HEM1_HUMAN

ANTISENSE INHIBITOR

UNKNOWN

DRUG LABEL

Asialoglycoprotein receptor 1

Membrane receptor

P07306

ASGR1_HUMAN

BINDING AGENT

UNKNOWN

DRUG LABEL

Asialoglycoprotein receptor 2

Membrane receptor

P07307

ASGR2_HUMAN

BINDING AGENT

UNKNOWN

DRUG LABEL

External reference:

ID	Source
ROV204583W	UNII
4038984	VANDF
C4688584	UMLSCUI
CHEBI:757209	CHEBI
CHEMBL4297760	ChEMBL_ID
DB15066	DRUGBANK_ID
CHEMBL4594265	ChEMBL_ID
D11702	KEGG_DRUG
10280	INN_ID
C000630124	MESH_SUPPLEMENTAL_RECORD_UI
13614	IUPHAR_LIGAND_ID
1217169003	SNOMEDCT_US
830100005	SNOMEDCT_US
830239005	SNOMEDCT_US
327500	MMSL
37780	MMSL
d09456	MMSL
018210	NDDF
018211	NDDF
2265712	RXNORM

Source

ROV204583W

UNII

4038984

VANDF

C4688584

UMLSCUI

CHEBI:757209

CHEBI

CHEMBL4297760

ChEMBL_ID

DB15066