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

Stem definition	Drug id	CAS RN
antisense oligonucleotides	5357	1422959-91-8

Description:

Molecule	Description
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

Molecule

Description

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:

Date	Agency	Company	Orphan
Dec. 12, 2019	FDA	SAREPTA THERAPS 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	253.09	103.97	70	170	119641	34837050
Poor venous access	187.92	103.97	32	208	5151	34951540
Intentional dose omission	143.02	103.97	24	216	3476	34953215
No adverse event	108.39	103.97	26	214	22901	34933790

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	264.78	131.90	69	144	247468	79496707
Poor venous access	165.10	131.90	30	183	18119	79726056
Intentional dose omission	136.64	131.90	23	190	8794	79735381

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
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

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)	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 strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
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:

Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Mechanism action	Bioact source	MoA source
exon 53 of dystrophin pre-mRNA	RNA	P11532	DMD_HUMAN	ANTISENSE INHIBITOR				UNKNOWN	DRUG LABEL

External reference:

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

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
Vyondys 53	HUMAN PRESCRIPTION DRUG LABEL	1	60923-465	INJECTION	50 mg	INTRAVENOUS	NDA	26 sections
Vyondys 53	HUMAN PRESCRIPTION DRUG LABEL	1	60923-465	INJECTION	50 mg	INTRAVENOUS	NDA	26 sections