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

Stem definition	Drug id	CAS RN
antisense oligonucleotides	5182	1173755-55-9

Description:

Molecule	Description
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

Molecule

Description

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:

Date	Agency	Company	Orphan
Sept. 19, 2016	FDA	SAREPTA THERAPEUTICS INC

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Product dose omission issue	997.08	67.05	298	995	119413	34836225
Intentional dose omission	495.70	67.05	89	1204	3411	34952227
No adverse event	450.67	67.05	114	1179	22813	34932825
Poor venous access	333.25	67.05	68	1225	5115	34950523
Device issue	227.96	67.05	56	1237	9721	34945917
Exposure to SARS-CoV-2	204.91	67.05	32	1261	502	34955136

FDA Adverse Event Reporting System (Geriatric)

MedDRA adverse event term	Likelihood ratio	Likelihood ratio threshold	Patients taking drug having adverse event	Patients taking drug not having adverse event	Patients not taking drug having adverse event	Patients not taking drug not having adverse event
Product dose omission issue	998.24	61.57	285	888	247252	79495963
No adverse event	500.91	61.57	114	1059	37078	79706137
Intentional dose omission	489.26	61.57	88	1085	8729	79734486
Poor venous access	283.19	61.57	63	1110	18086	79725129
Device issue	202.83	61.57	52	1121	27056	79716159
Exposure to SARS-CoV-2	170.67	61.57	29	1144	2015	79741200

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
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|

Disease	Relation	SNOMED_ID	DOID
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 strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent 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 strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
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:

Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Mechanism action	Bioact source	MoA source
exon 51 of dystrophin pre-mRNA	RNA	P11532	DMD_HUMAN	BINDING AGENT				UNKNOWN	DRUG LABEL

External reference:

ID	Source
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
016988	NDDF
724033001	SNOMEDCT_US
763585001	SNOMEDCT_US
C000611335	MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
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