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

Stem definitionDrug idCAS RN
small interfering RNA including siRNA, miRNA and piRNA 5354 1639325-43-1

Description:

MoleculeDescription

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

Drug dosage:

None

ADMET properties:

PropertyValueReference
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

Approvals:

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

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:

SourceCodeDescription
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

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

DiseaseRelationSNOMED_IDDOID
Hepatic porphyria indication 55056006 DOID:3133




🐶 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 strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent use
EQ 189MG BASE/ML (EQ 189MG BASE/ML) GIVLAARI ALNYLAM PHARMS INC N212194 Nov. 20, 2019 RX SOLUTION SUBCUTANEOUS 9708610 Jan. 1, 2024 TREATMENT OF ACUTE HEPATIC PORPHYRIA
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

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
EQ 189MG BASE/ML (EQ 189MG BASE/ML) GIVLAARI ALNYLAM PHARMS INC N212194 Nov. 20, 2019 RX SOLUTION SUBCUTANEOUS Nov. 20, 2024 NEW CHEMICAL ENTITY
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)

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
aminolevulinate synthase1 (ALAS1) mRNA RNA ANTISENSE INHIBITOR UNKNOWN DRUG LABEL

External reference:

IDSource
ROV204583W UNII
C4688584 UMLSCUI
CHEMBL4297760 ChEMBL_ID
156323004 PUBCHEM_CID
DB15066 DRUGBANK_ID
CHEMBL4594265 ChEMBL_ID
D11702 KEGG_DRUG
10280 INN_ID
018210 NDDF
018211 NDDF
1217169003 SNOMEDCT_US
830100005 SNOMEDCT_US
830239005 SNOMEDCT_US
4038984 VANDF
2265712 RXNORM
327500 MMSL
37780 MMSL
d09456 MMSL
C000630124 MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
GIVLAARI HUMAN PRESCRIPTION DRUG LABEL 1 71336-1001 INJECTION, SOLUTION 189 mg SUBCUTANEOUS NDA 28 sections
GIVLAARI HUMAN PRESCRIPTION DRUG LABEL 1 71336-1001 INJECTION, SOLUTION 189 mg SUBCUTANEOUS NDA 28 sections
GIVLAARI HUMAN PRESCRIPTION DRUG LABEL 1 71336-1001 INJECTION, SOLUTION 189 mg SUBCUTANEOUS NDA 28 sections