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

Stem definitionDrug idCAS RN
antisense oligonucleotides 5408 2055732-84-6

Description:

MoleculeDescription

Molfile

Synonyms:

  • viltolarsen
  • viltepso
  • NS-065/NCNP-01
  • NCNP-01
  • NS 065
Viltolarsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients. It is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
  • Molecular weight:
  • Formula:
  • CLOGP:
  • LIPINSKI: None
  • HAC: None
  • HDO: None
  • TPSA:
  • ALOGS:
  • ROTB: None

Drug dosage:

None

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
Aug. 12, 2020 FDA NIPPON SHINYAKU
March 25, 2020 PMDA Nippon Shinyaku

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

Pharmacologic Action:

SourceCodeDescription
ATC M09AX12 MUSCULO-SKELETAL SYSTEM
OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM
Other drugs for disorders of the musculo-skeletal system

Drug Use (View source of the data)

DiseaseRelationSNOMED_IDDOID
Duchenne muscular dystrophy indication 76670001 DOID:11723

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
250MG/5ML (50MG/ML) VILTEPSO NIPPON SHINYAKU N212154 Aug. 12, 2020 RX SOLUTION INTRAVENOUS 10870676 Aug. 31, 2031 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 53 SKIPPING

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
250MG/5ML (50MG/ML) VILTEPSO NIPPON SHINYAKU N212154 Aug. 12, 2020 RX SOLUTION INTRAVENOUS Aug. 12, 2025 NEW CHEMICAL ENTITY
250MG/5ML (50MG/ML) VILTEPSO NIPPON SHINYAKU N212154 Aug. 12, 2020 RX SOLUTION INTRAVENOUS Aug. 12, 2027 INDICATED FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 53 SKIPPING

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
exon 53 of dystrophin pre-mRNA RNA BINDING AGENT EC50 8.33 DRUG LABEL DRUG LABEL

External reference:

IDSource
D11528 KEGG_DRUG
SXA7YP6EKX UNII
4039646 VANDF
C5139804 UMLSCUI
CHEMBL4298062 ChEMBL_ID
154562176 PUBCHEM_CID
DB15005 DRUGBANK_ID
C000654848 MESH_SUPPLEMENTAL_RECORD_UI
11430 IUPHAR_LIGAND_ID
10771 INN_ID
2389840 RXNORM
897124003 SNOMEDCT_US
897127005 SNOMEDCT_US
337588 MMSL
38726 MMSL
d09616 MMSL
018472 NDDF

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Viltepso HUMAN PRESCRIPTION DRUG LABEL 1 73292-011 INJECTION, SOLUTION 250 mg INTRAVENOUS NDA 25 sections