lumasiran

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Stem definition	Drug id	CAS RN
small interfering RNA including siRNA, miRNA and piRNA	5415	1834610-13-7

Stem definition

Drug id

CAS RN

small interfering RNA including siRNA, miRNA and piRNA

5415

1834610-13-7

Description:

Molecule	Description
Molfile Synonyms: lumasiran oxlumo ALN-G01 ALN-65585 AD-65585 lumasiran sodium	Lumasiran is a hydroxyacid oxidase 1 (HAO1)-directed double-stranded small interfering ribonucleic acid (siRNA), covalently linked to a ligand containing N-acetylgalactosamine (GalNAc). It reduces levels of glycolate oxidase (GO) enzyme by targeting the HAO1 gene messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. Decreased GO enzyme levels reduce the amount of available glyoxylate, a substrate for oxalate production. As the GO enzyme is upstream of the deficient alanine:glyoxylate aminotransferase (AGT) enzyme that causes PH1, the mechanism of action of lumasiran is independent of the underlying AGXT gene mutation. Molecular weight: Formula: CLOGP: LIPINSKI: None HAC: None HDO: None TPSA: ALOGS: ROTB: None

Molecule

Description

Molfile

Synonyms:

lumasiran
oxlumo
ALN-G01
ALN-65585
AD-65585
lumasiran sodium

Lumasiran is a hydroxyacid oxidase 1 (HAO1)-directed double-stranded small interfering ribonucleic acid (siRNA), covalently linked to a ligand containing N-acetylgalactosamine (GalNAc). It reduces levels of glycolate oxidase (GO) enzyme by targeting the HAO1 gene messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. Decreased GO enzyme levels reduce the amount of available glyoxylate, a substrate for oxalate production. As the GO enzyme is upstream of the deficient alanine:glyoxylate aminotransferase (AGT) enzyme that causes PH1, the mechanism of action of lumasiran is independent of the underlying AGXT gene mutation.

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

Date	Agency	Company	Orphan
Nov. 23, 2020	FDA	ALNYLAM PHARMS INC
Nov. 19, 2020	EMA	Alnylam Netherlands B.V.

Date

Agency

Company

Orphan

Nov. 23, 2020

FDA

ALNYLAM PHARMS INC

Nov. 19, 2020

EMA

Alnylam Netherlands B.V.

Source	Code	Description
ATC	A16AX18	ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Various alimentary tract and metabolism products
MeSH PA	D012076	Renal Agents

Source

Code

Description

ATC

A16AX18

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

MeSH PA

D012076

Renal Agents

Disease	Relation	SNOMED_ID	DOID
Primary hyperoxaluria, type I	indication	65520001	DOID:0111670

Disease

Relation

SNOMED_ID

DOID

Primary hyperoxaluria, type I

indication

65520001

DOID:0111670

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 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	10131907	Aug. 24, 2028	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	8828956	Dec. 4, 2028	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	8106022	Dec. 12, 2029	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	10435692	Dec. 26, 2034	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	10465195	Dec. 26, 2034	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	10487330	Dec. 26, 2034	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	11060093	Dec. 26, 2034	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	10612024	Aug. 14, 2035	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	10612027	Aug. 14, 2035	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	11401517	Aug. 14, 2035	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	10478500	Oct. 9, 2035	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	11261447	Nov. 20, 2038	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

Formulation strength

Trade name

Applicant

Application number

Approval date

Type

Dose form

Route

Patent number

Patent expiration date

Patent use

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

10131907

Aug. 24, 2028

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

8828956

Dec. 4, 2028

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

8106022

Dec. 12, 2029

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

10435692

Dec. 26, 2034

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

10465195

Dec. 26, 2034

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

10487330

Dec. 26, 2034

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

11060093

Dec. 26, 2034

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

10612024

Aug. 14, 2035

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

10612027

Aug. 14, 2035

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

11401517

Aug. 14, 2035

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

10478500

Oct. 9, 2035

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

11261447

Nov. 20, 2038

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1)

Orange Book exclusivity data (new drug applications)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	Nov. 23, 2027	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1) TO LOWER URINARY OXALATE LEVELS IN PEDIATRIC AND ADULT PATIENTS
EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	Oct. 6, 2029	TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1) TO LOWER PLASMA OXALATE LEVELS IN PEDIATRIC AND ADULT PATIENTS

Formulation strength

Trade name

Applicant

Application number

Approval date

Type

Dose form

Route

Exclusivity date

Description

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

Nov. 23, 2027

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1) TO LOWER URINARY OXALATE LEVELS IN PEDIATRIC AND ADULT PATIENTS

EQ 94.5MG BASE/0.5ML (EQ 94.5MG BASE/0.5ML)

OXLUMO

ALNYLAM PHARMS INC

N214103

Nov. 23, 2020

SOLUTION

SUBCUTANEOUS

Oct. 6, 2029

TREATMENT OF PRIMARY HYPEROXALURIA TYPE 1 (PH1) TO LOWER PLASMA OXALATE LEVELS IN PEDIATRIC AND ADULT PATIENTS

Bioactivity Summary:

Target	Class	Pharos	UniProt	Action	Bioact source	MoA source
Hydroxyacid oxidase 1 (HAO1) mRNA	RNA	Q9UJM8	HAOX1_HUMAN	ANTISENSE INHIBITOR	DRUG LABEL	DRUG LABEL
Asialoglycoprotein receptor 1	Membrane receptor	P07306	ASGR1_HUMAN	BINDING AGENT	DRUG LABEL	DRUG LABEL
Asialoglycoprotein receptor 2	Membrane receptor	P07307	ASGR2_HUMAN	BINDING AGENT	DRUG LABEL	DRUG LABEL

Target

Class

Pharos

UniProt

Action

Type

Activity value
(-log[M])

Mechanism
action

Bioact source

MoA source

Hydroxyacid oxidase 1 (HAO1) mRNA

RNA

Q9UJM8

HAOX1_HUMAN

ANTISENSE INHIBITOR

DRUG LABEL

Asialoglycoprotein receptor 1

Membrane receptor

P07306

ASGR1_HUMAN

BINDING AGENT

DRUG LABEL

Asialoglycoprotein receptor 2

Membrane receptor

P07307

ASGR2_HUMAN

BINDING AGENT

DRUG LABEL

External reference:

ID	Source
D11925	KEGG_DRUG
RZT8C352O1	UNII
1834612-06-4	SECONDARY_CAS_RN
C5418443	UMLSCUI
CHEBI:757227	CHEBI
CHEMBL4297779	ChEMBL_ID
DB15935	DRUGBANK_ID
CHEMBL4650275	ChEMBL_ID
10684	INN_ID
C000716350	MESH_SUPPLEMENTAL_RECORD_UI
13613	IUPHAR_LIGAND_ID
1137368005	SNOMEDCT_US
1137588005	SNOMEDCT_US
1217201009	SNOMEDCT_US
341103	MMSL
39040	MMSL
d09686	MMSL
018570	NDDF
018571	NDDF
2467140	RXNORM

Source

D11925

KEGG_DRUG

RZT8C352O1

UNII

1834612-06-4

SECONDARY_CAS_RN

C5418443

UMLSCUI

CHEBI:757227

CHEBI

CHEMBL4297779