eteplirsen 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definitionDrug idCAS RN
antisense oligonucleotides 5182 1173755-55-9

Description:

MoleculeDescription

Synonyms:

  • eteplirsen
  • Exondys 51
  • AVI-4658
  • AVI 4658
Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.
  • Molecular weight: 10305.89
  • Formula: C364H569N177O122P30
  • CLOGP:
  • LIPINSKI: None
  • HAC: None
  • HDO: None
  • TPSA: 3305.26
  • ALOGS:
  • ROTB: None

Drug dosage:

None

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
Sept. 19, 2016 FDA SAREPTA THERAPEUTICS 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 879.99 73.18 248 692 96135 29477452
Intentional dose omission 337.34 73.18 58 882 2008 29571579
Poor venous access 332.05 73.18 61 879 3100 29570487
No adverse event 209.25 73.18 57 883 17421 29556166
Device issue 190.51 73.18 45 895 7735 29565852
Exposure to SARS-CoV-2 173.30 73.18 23 917 121 29573466

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 859.82 80.40 233 601 194514 64303384
Intentional dose omission 324.64 80.40 57 777 5595 64492303
Poor venous access 273.92 80.40 56 778 12361 64485537
No adverse event 222.39 80.40 55 779 28506 64469392
Device issue 167.92 80.40 42 792 22606 64475292
Exposure to SARS-CoV-2 138.87 80.40 20 814 536 64497362

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC M09AX06 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
FDA CS M0025055 Oligonucleotides, Antisense
FDA EPC N0000191626 Antisense Oligonucleotide

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) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING BY CORRECTING A DEFECTIVE GENE FOR DYSTROPHIN
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING BY INDUCING SKIPPING OF EXON 51
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING BY RESTORING OR INCREASING FUNCTIONAL DYSTROPHIN PROTEIN PRODUCTION
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING BY CORRECTING A DEFECTIVE GENE FOR DYSTROPHIN
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING BY INDUCING SKIPPING OF EXON 51
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING BY RESTORING OR INCREASING FUNCTIONAL DYSTROPHIN PROTEIN PRODUCTION
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE47751 June 28, 2025 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9243245 Oct. 27, 2028 INCREASING PRODUCTION OF FUNCTIONAL DYSTROPHIN PROTEIN IN DMD PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9243245 Oct. 27, 2028 TREATMENT OF DMD IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE48468 Oct. 27, 2028 TREATMENT OF DMD IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9243245 Oct. 27, 2028 INCREASING PRODUCTION OF FUNCTIONAL DYSTROPHIN PROTEIN IN DMD PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9243245 Oct. 27, 2028 TREATMENT OF DMD IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS RE48468 Oct. 27, 2028 TREATMENT OF DMD IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 10337003 March 14, 2034 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 10364431 March 14, 2034 RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 10364431 March 14, 2034 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9506058 March 14, 2034 RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9506058 March 14, 2034 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 10337003 March 14, 2034 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 10364431 March 14, 2034 RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 10364431 March 14, 2034 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9506058 March 14, 2034 RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS 9506058 March 14, 2034 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
100MG/2ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS Sept. 19, 2023 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING
500MG/10ML (50MG/ML) EXONDYS 51 SAREPTA THERAPS INC N206488 Sept. 19, 2016 RX SOLUTION INTRAVENOUS Sept. 19, 2023 TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) IN PATIENTS WHO HAVE A CONFIRMED MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
exon 51 of dystrophin pre-mRNA RNA BINDING AGENT UNKNOWN DRUG LABEL

External reference:

IDSource
AIW6036FAS UNII
4036083 VANDF
C4283710 UMLSCUI
CHEMBL2108278 ChEMBL_ID
D09900 KEGG_DRUG
DB06014 DRUGBANK_ID
9339 INN_ID
7534 IUPHAR_LIGAND_ID
1810569 RXNORM
244179 MMSL
31970 MMSL
d08476 MMSL
724033001 SNOMEDCT_US
763585001 SNOMEDCT_US
016988 NDDF
C000611335 MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Exondys 51 HUMAN PRESCRIPTION DRUG LABEL 1 60923-284 INJECTION 50 mg INTRAVENOUS NDA 27 sections
Exondys 51 HUMAN PRESCRIPTION DRUG LABEL 1 60923-284 INJECTION 50 mg INTRAVENOUS NDA 27 sections
Exondys 51 HUMAN PRESCRIPTION DRUG LABEL 1 60923-363 INJECTION 50 mg INTRAVENOUS NDA 27 sections
Exondys 51 HUMAN PRESCRIPTION DRUG LABEL 1 60923-363 INJECTION 50 mg INTRAVENOUS NDA 27 sections