Casimersen

Antisense Oligonucleotide — MUSCULO-SKELETAL SYSTEM

Key facts

Casimersen is a drug in the Antisense Oligonucleotide class catalogued in DrugCentral. It was first approved by the FDA in 2021. DrugCentral lists 1 molecular target, including DMD. Recorded indications include Duchenne muscular dystrophy.

• Drug class: Antisense Oligonucleotide.
• Molecular targets: DMD (1 listed in DrugCentral).
• Indications: Duchenne muscular dystrophy.
• ATC code: M09AX13 (Other drugs for disorders of the musculo-skeletal system).

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

• Duchenne muscular dystrophy

Mechanism & targets

• Established pharmacologic class: Antisense Oligonucleotide

Molecular targets (1)

Target	Gene	Action	Activity
exon 53 of dystrophin pre-mRNA	DMD	other	—

Regulatory approvals (1)

Date	Agency	Marketed as	Applicant	Orphan
2021-02-25	FDA	—	SAREPTA THERAPS INC	Yes

ATC classification

ATC code	Therapeutic hierarchy
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

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label
• FDA label: DMD · Testing Required
• HCSC label

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Product dose omission issue	712.3
No adverse event	650.1
Poor venous access	466.8
Intentional dose omission	332.8
Device issue	292.9

Chemical structure

• Molecular formula: —
• Molecular weight: —
• CAS number: —
• InChIKey: —
• PubChem CID: —

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Casimersen used for?

DrugCentral lists the following indications for Casimersen: Duchenne muscular dystrophy.

How does Casimersen work?

Its molecular targets in DrugCentral include DMD.

When was Casimersen first approved?

Casimersen was first approved by the FDA in 2021.

What drug class does Casimersen belong to?

Casimersen is in the Antisense Oligonucleotide class.

Sources

• DrugCentral drug card 5443 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL4297566 — compound record and bioactivity
• DrugBank DB14984 — drug record
• FDA UNII X8UHF7SX0R — substance identity
• DailyMed: FDA labels for Casimersen — current prescribing information (label search)
• UniProt P11532 (DMD) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

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