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

Stem definition	Drug id	CAS RN
antisense oligonucleotides	5443	1422958-19-7

Description:

Molecule	Description
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

Molecule

Description

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

Approvals:

Date	Agency	Company	Orphan
Feb. 25, 2021	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	320.47	127.54	89	220	119622	34837000
No adverse event	241.28	127.54	52	257	22875	34933747
Poor venous access	192.71	127.54	34	275	5149	34951473
Intentional dose omission	136.68	127.54	24	285	3476	34953146

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	313.62	125.93	84	199	247453	79496652
No adverse event	263.98	125.93	52	231	37140	79706965
Poor venous access	174.95	125.93	33	250	18116	79725989
Intentional dose omission	136.50	125.93	24	259	8793	79735312

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
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
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)	AMONDYS 45	SAREPTA THERAPS INC	N213026	Feb. 25, 2021	RX	SOLUTION	INTRAVENOUS	RE48960	June 28, 2025	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	June 28, 2025	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 strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
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:

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
D11988	KEGG_DRUG
X8UHF7SX0R	UNII
C5139793	UMLSCUI
CHEMBL4297566	ChEMBL_ID
DB14984	DRUGBANK_ID
11444	IUPHAR_LIGAND_ID
018647	NDDF
1179224007	SNOMEDCT_US
1179225008	SNOMEDCT_US
2480096	RXNORM
343979	MMSL
39340	MMSL
d09718	MMSL
C000718147	MESH_SUPPLEMENTAL_RECORD_UI
10354	INN_ID

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
AMONDYS 45	HUMAN PRESCRIPTION DRUG LABEL	1	60923-227	INJECTION	50 mg	INTRAVENOUS	NDA	26 sections
AMONDYS 45	HUMAN PRESCRIPTION DRUG LABEL	1	60923-227	INJECTION	50 mg	INTRAVENOUS	NDA	26 sections