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

Stem definitionDrug idCAS RN
antisense oligonucleotides 5357 1422959-91-8

Description:

MoleculeDescription

Synonyms:

  • golodirsen
  • vyondys 53
  • SRP-4053
Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. 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: 13503.45
  • Formula: C454H755N138O238P50
  • CLOGP:
  • LIPINSKI: None
  • HAC: None
  • HDO: None
  • TPSA: 4404.96
  • ALOGS:
  • ROTB: None

Drug dosage:

None

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
Dec. 12, 2019 FDA SAREPTA THERAPS INC

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

Pharmacologic Action:

SourceCodeDescription
ATC M09AX08 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
MeSH PA D015335 Molecular Probes
MeSH PA D016376 Oligonucleotides, Antisense

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
100MG/2ML (50MG/ML) VYONDYS 53 SAREPTA THERAPS INC N211970 Dec. 12, 2019 RX SOLUTION INTRAVENOUS 10266827 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 53 SKIPPING
100MG/2ML (50MG/ML) VYONDYS 53 SAREPTA THERAPS INC N211970 Dec. 12, 2019 RX SOLUTION INTRAVENOUS 10995337 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING 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
100MG/2ML (50MG/ML) VYONDYS 53 SAREPTA THERAPS INC N211970 Dec. 12, 2019 RX SOLUTION INTRAVENOUS Dec. 12, 2024 NEW CHEMICAL ENTITY
100MG/2ML (50MG/ML) VYONDYS 53 SAREPTA THERAPS INC N211970 Dec. 12, 2019 RX SOLUTION INTRAVENOUS Dec. 12, 2026 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 ANTISENSE INHIBITOR UNKNOWN DRUG LABEL

External reference:

IDSource
4039025 VANDF
CHEMBL4297762 ChEMBL_ID
D11707 KEGG_DRUG
C000710673 MESH_SUPPLEMENTAL_RECORD_UI
10355 INN_ID
DB15593 DRUGBANK_ID
033072U4MZ UNII
2267207 RXNORM
838470000 SNOMEDCT_US
838482009 SNOMEDCT_US
328504 MMSL
37857 MMSL
d09468 MMSL
018234 NDDF
C5222004 UMLSCUI

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Vyondys 53 HUMAN PRESCRIPTION DRUG LABEL 1 60923-465 INJECTION 50 mg INTRAVENOUS NDA 26 sections