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

Stem definitionDrug idCAS RN
antisense oligonucleotides 5443 1422958-19-7

Description:

MoleculeDescription

Molfile

Synonyms:

  • casimersen
  • amondys 45
  • SRP-4045
Casimersen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping.
  • Molecular weight: 5106.90
  • Formula: C187H396N46O71P22
  • CLOGP:
  • LIPINSKI: None
  • HAC: None
  • HDO: None
  • TPSA: 1004.48
  • ALOGS:
  • ROTB: None

Drug dosage:

None

ADMET properties:

None

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

DateAgencyCompanyOrphan
Feb. 25, 2021 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 683.94 92.31 218 868 154147 42466102
No adverse event 650.52 92.31 157 929 38864 42581385
Poor venous access 496.16 92.31 94 992 7158 42613091
Intentional dose omission 333.11 92.31 70 1016 8916 42611333
Device issue 293.95 92.31 68 1018 13441 42606808

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 738.84 91.78 211 709 321571 110266808
No adverse event 693.84 91.78 151 769 73688 110514691
Poor venous access 444.27 91.78 87 833 24435 110563944
Intentional dose omission 343.34 91.78 68 852 20012 110568367
Device issue 279.07 91.78 63 857 34472 110553907

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC M09AX13 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 acidatalureneteplirsengivinostatgolodirsenhyaluronic 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)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent use
100MG/2ML (50MG/ML) AMONDYS 45 SAREPTA THERAPS INC N213026 Feb. 25, 2021 RX SOLUTION INTRAVENOUS RE48960 Feb. 16, 2029 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 45 SKIPPING
100MG/2ML (50MG/ML) AMONDYS 45 SAREPTA THERAPS INC N213026 Feb. 25, 2021 RX SOLUTION INTRAVENOUS RE48960 Feb. 16, 2029 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 45 SKIPPING BY INDUCING EXON-SKIPPING OF EXON 45
100MG/2ML (50MG/ML) AMONDYS 45 SAREPTA THERAPS INC N213026 Feb. 25, 2021 RX SOLUTION INTRAVENOUS 10781450 Nov. 12, 2030 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 45 SKIPPING BY RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION
100MG/2ML (50MG/ML) AMONDYS 45 SAREPTA THERAPS INC N213026 Feb. 25, 2021 RX SOLUTION INTRAVENOUS 9758783 Nov. 12, 2030 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 45 SKIPPING
100MG/2ML (50MG/ML) AMONDYS 45 SAREPTA THERAPS INC N213026 Feb. 25, 2021 RX SOLUTION INTRAVENOUS 9758783 Nov. 12, 2030 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 45 SKIPPING BY RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
100MG/2ML (50MG/ML) AMONDYS 45 SAREPTA THERAPS INC N213026 Feb. 25, 2021 RX SOLUTION INTRAVENOUS Feb. 25, 2026 NEW CHEMICAL ENTITY
100MG/2ML (50MG/ML) AMONDYS 45 SAREPTA THERAPS INC N213026 Feb. 25, 2021 RX SOLUTION INTRAVENOUS Feb. 25, 2028 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 45 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
D11988 KEGG_DRUG
X8UHF7SX0R UNII
C5139793 UMLSCUI
CHEBI:757041 CHEBI
CHEMBL4297566 ChEMBL_ID
DB14984 DRUGBANK_ID
10354 INN_ID
C000718147 MESH_SUPPLEMENTAL_RECORD_UI
11444 IUPHAR_LIGAND_ID
1179224007 SNOMEDCT_US
1179225008 SNOMEDCT_US
343979 MMSL
39340 MMSL
d09718 MMSL
018647 NDDF
2480096 RXNORM

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
AMONDYS 45 HUMAN PRESCRIPTION DRUG LABEL 1 60923-227 INJECTION 50 mg INTRAVENOUS NDA 25 sections