lumasiran 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

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

Drug dosage:

None

ADMET properties:

None

Approvals:

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

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

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

Drug Use | Suggest Off label Use Form| |View source of the data|

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

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

None

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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	Oct. 6, 2025	EXPANDED INDICATION TO INCLUDE LOWERING OF PLASMA OXALATE LEVELS INADULT AND PEDIATRIC PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1 (PH1)
EQ 94.5MG/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	OXLUMO	ALNYLAM PHARMS INC	N214103	Nov. 23, 2020	RX	SOLUTION	SUBCUTANEOUS	Nov. 23, 2025	NEW CHEMICAL ENTITY
EQ 94.5MG/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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/0.5ML BASE (EQ 94.5MG/0.5ML BASE)	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

Bioactivity Summary:

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	DRUG LABEL

External reference:

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

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
OXLUMO	HUMAN PRESCRIPTION DRUG LABEL	1	71336-1002	INJECTION, SOLUTION	94.50 mg	SUBCUTANEOUS	NDA	27 sections
OXLUMO	HUMAN PRESCRIPTION DRUG LABEL	1	71336-1002	INJECTION, SOLUTION	94.50 mg	SUBCUTANEOUS	NDA	27 sections