Nusinersen

Antisense Oligonucleotide — MUSCULO-SKELETAL SYSTEM

Key facts

Nusinersen is a drug in the Antisense Oligonucleotide class catalogued in DrugCentral. It was first approved by the FDA in 2016. DrugCentral lists 1 molecular target, including SMN1; SMN2. Recorded indications include Spinal muscular atrophy.

• Drug class: Antisense Oligonucleotide; Survival Motor Neuron-2-directed RNA Interaction.
• Molecular targets: SMN1; SMN2 (1 listed in DrugCentral).
• Indications: Spinal muscular atrophy.
• ATC code: M09AX07 (Other drugs for disorders of the musculo-skeletal system).

Indications & contraindications

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

Indications (1)

• Spinal muscular atrophy

Mechanism & targets

• Established pharmacologic class: Antisense Oligonucleotide; Survival Motor Neuron-2-directed RNA Interaction

Molecular targets (1)

Target	Gene	Action	Activity
Survival motor neuron protein mRNA	SMN1; SMN2	other	—

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2017-07-03	PMDA	—	Biogen Japan Ltd	Yes
2017-05-30	EMA	—	Biogen Idec Ltd	Yes
2016-12-23	FDA	—	BIOGEN

ATC classification

ATC code	Therapeutic hierarchy
M09AX07	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: SMN2
• FDA label: SMN2
• HCSC label: SMN2

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
Post lumbar puncture syndrome	740.3
Scoliosis	468.0
Death	234.3
Protein urine present	185.5
Rhinovirus infection	175.1
Respiratory failure	158.5
Respiratory tract infection	153.2
Respiratory disorder	150.2
Cerebrospinal fluid leakage	131.9
Intracranial pressure increased	130.1
Respiratory syncytial virus infection	128.7
Pneumonia	108.1

Chemical structure

• Molecular formula: —
• Molecular weight: —
• CAS number: —
• InChIKey: —
• PubChem CID: 131801471

Frequently asked questions

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

What is Nusinersen used for?

DrugCentral lists the following indications for Nusinersen: Spinal muscular atrophy.

How does Nusinersen work?

Its molecular targets in DrugCentral include SMN1; SMN2.

When was Nusinersen first approved?

Nusinersen was first approved by the FDA in 2016.

What drug class does Nusinersen belong to?

Nusinersen is in the Antisense Oligonucleotide class.

Sources

• DrugCentral drug card 5207 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3301572 — compound record and bioactivity
• PubChem CID 131801471 — chemical identifiers
• DrugBank DB13161 — drug record
• FDA UNII 5Z9SP3X666 — substance identity
• DailyMed: FDA labels for Nusinersen — current prescribing information (label search)
• UniProt Q16637 (SMN1; SMN2) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.