patisiran 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |

Stem definitionDrug idCAS RN
small interfering RNA including siRNA, miRNA and piRNA 5313 1420706-45-1

Description:

MoleculeDescription

Synonyms:

  • patisiran
  • ALN-TTR02
  • ALN-18328
  • patisiran sodium
  • onpattro
Patisiran is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.
  • Molecular weight:
  • Formula:
  • CLOGP:
  • LIPINSKI: None
  • HAC: None
  • HDO: None
  • TPSA:
  • ALOGS:
  • ROTB: None

Drug dosage:

None

ADMET properties:

None

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

DateAgencyCompanyOrphan
June 18, 2019 PMDA ALNYLAM JAPAN K.K.
Oct. 8, 2018 FDA ALNYLAM PHARMS INC
Aug. 27, 2018 EMA ALNYLAM NETHERLANDS B.V.

FDA Adverse Event Reporting System (Female)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Infusion related reaction 55.21 38.42 26 247 416754 90211420
Vitamin A decreased 41.67 38.42 5 268 143 90628031

FDA Adverse Event Reporting System (Male)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Infusion related reaction 75.78 42.30 31 701 60775 42559828

FDA Adverse Event Reporting System (Geriatric)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Infusion related reaction 63.27 37.73 35 635 381127 110207502
Product dose omission issue 43.74 37.73 26 644 321756 110266873
Intentional dose omission 38.76 37.73 11 659 20069 110568560

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC N07XX12 NERVOUS SYSTEM
OTHER NERVOUS SYSTEM DRUGS
OTHER NERVOUS SYSTEM DRUGS
Other nervous system drugs
FDA CS M0406100 RNA, Small Interfering
FDA PE N0000008852 Decreased RNA Integrity
FDA PE N0000009531 Increased Protein Breakdown
FDA EPC N0000193848 Small Interfering RNA
FDA EPC N0000193849 Transthyretin-directed RNA Interaction

Related Drugs by ATC class:

N07XX Other nervous system drugs 23 drugs
amifampridinearimoclomoldeutetrabenazinedextromethorphanedaravoneeplontersenfampridineinotersen laquinimodlevacetylleucineomaveloxolonephenylbutanoic acid show all 23 →

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

DiseaseRelationSNOMED_IDDOID
Transthyretin familial amyloid polyneuropathy indication 42295001
Polyneuropathy in amyloidosis indication 193187004




🐶 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 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 8822668 April 15, 2029 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 9364435 April 15, 2029 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 10240152 Oct. 20, 2029 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 8741866 Oct. 20, 2029 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 9234196 Oct. 20, 2029 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 8802644 Oct. 21, 2030 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 8158601 Nov. 10, 2030 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 8168775 Aug. 10, 2032 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS 11079379 Aug. 27, 2035 TREATMENT OF POLYNEUROPATHY OF HEREDITARY TRANSTHYRETIN-MEDIATED AMYLOIDOSIS

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
EQ 10MG BASE/5ML (EQ 2MG BASE/ML) ONPATTRO ALNYLAM PHARMS INC N210922 Aug. 10, 2018 RX SOLUTION INTRAVENOUS Jan. 13, 2026 INFORMATION ADDED TO CLINICAL PHARMACOLOGY SECTION

Bioactivity Summary:

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

External reference:

IDSource
50FKX8CB2Y UNII
4037752 VANDF
C3896714 UMLSCUI
CHEBI:756470 CHEBI
CHEMBL3989987 ChEMBL_ID
DB14582 DRUGBANK_ID
D11116 KEGG_DRUG
9793 INN_ID
C000606954 MESH_SUPPLEMENTAL_RECORD_UI
13611 IUPHAR_LIGAND_ID
1217021003 SNOMEDCT_US
773605008 SNOMEDCT_US
773608005 SNOMEDCT_US
CHEMBL4297536 ChEMBL_ID
1386913-72-9 SECONDARY_CAS_RN
287397 MMSL
34742 MMSL
d08858 MMSL
017680 NDDF
2053490 RXNORM

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Onpattro HUMAN PRESCRIPTION DRUG LABEL 1 71336-1000 INJECTION, LIPID COMPLEX 2 mg INTRAVENOUS NDA 27 sections