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

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

DateAgencyCompanyOrphan
Dec. 12, 2019 FDA SAREPTA THERAPS INC

FDA Adverse Event Reporting System (Female)

None

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
Product dose omission issue 355.64 75.44 112 427 154253 42466543
Poor venous access 319.40 75.44 58 481 7194 42613602
No adverse event 271.87 75.44 68 471 38953 42581843
Intentional dose omission 149.57 75.44 32 507 8954 42611842
Device issue 105.45 75.44 26 513 13483 42607313

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
Product dose omission issue 396.47 95.52 110 328 321672 110267189
No adverse event 291.58 95.52 65 373 73774 110515087
Poor venous access 271.62 95.52 51 387 24471 110564390
Intentional dose omission 155.13 95.52 31 407 20049 110568812
Device issue 100.82 95.52 24 414 34511 110554350

FDA Adverse Event Reporting System (Pediatric)

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
FDA CS M0025055 Oligonucleotides, Antisense
FDA PE N0000009533 Increased Protein Synthesis
FDA EPC N0000191626 Antisense Oligonucleotide

Related Drugs by ATC class:

M09AX Other drugs for disorders of the musculo-skeletal system 11 drugs
aceneuramic acidatalurencasimerseneteplirsengivinostathyaluronic acidnusinersenonasemnogene abeparvovecpalovarotenerisdiplamviltolarsen

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

DiseaseRelationSNOMED_IDDOID
Duchenne muscular dystrophy indication 76670001 DOID:11723




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

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
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
CHEBI:757211 CHEBI
CHEMBL4297762 ChEMBL_ID
D11707 KEGG_DRUG
C000710673 MESH_SUPPLEMENTAL_RECORD_UI
13605 IUPHAR_LIGAND_ID
DB15593 DRUGBANK_ID
838470000 SNOMEDCT_US
838482009 SNOMEDCT_US
C5222004 UMLSCUI
10355 INN_ID
328504 MMSL
37857 MMSL
d09468 MMSL
018234 NDDF
033072U4MZ UNII
2267207 RXNORM

Pharmaceutical products:

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