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

Stem definitionDrug idCAS RN
small interfering RNA 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:

None

Approvals:

DateAgencyCompanyOrphan
March 2, 2020 EMA Alnylam Netherlands B.V.
Nov. 20, 2019 FDA ALNYLAM PHARMS INC

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

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

Drug Use (View source of the data)

DiseaseRelationSNOMED_IDDOID
Hepatic porphyria indication 55056006 DOID:3133

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

None

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
4038984 VANDF
C4722734 UMLSCUI
CHEMBL4297760 ChEMBL_ID
CHEMBL4594265 ChEMBL_ID
DB15066 DRUGBANK_ID
D11702 KEGG_DRUG
119058042 PUBCHEM_CID
C000630124 MESH_SUPPLEMENTAL_RECORD_UI
10280 INN_ID
ROV204583W UNII
2265712 RXNORM
327500 MMSL
37780 MMSL
d09456 MMSL
018210 NDDF
018211 NDDF
830100005 SNOMEDCT_US
830239005 SNOMEDCT_US

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
GIVLAARI HUMAN PRESCRIPTION DRUG LABEL 1 71336-1001 INJECTION, SOLUTION 189 mg SUBCUTANEOUS NDA 27 sections