Tofersen

Antisense Oligonucleotide — NERVOUS SYSTEM

Key facts

Tofersen is a drug in the Antisense Oligonucleotide class catalogued in DrugCentral. It was first approved by the FDA in 2023. DrugCentral lists 1 molecular target, including SOD1. Recorded indications include Amyotrophic lateral sclerosis.

• Drug class: Antisense Oligonucleotide.
• Molecular targets: SOD1 (1 listed in DrugCentral).
• Indications: Amyotrophic lateral sclerosis.
• ATC code: N07XX22 (Other nervous system drugs).
• Molecular formula: C230H317N72O123P19S15; molecular weight 7127.96 g/mol.

Indications & contraindications

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

Indications (1)

• Amyotrophic lateral sclerosis

Mechanism & targets

• Established pharmacologic class: Antisense Oligonucleotide

Molecular targets (1)

Target	Gene	Action	Activity
Superoxide dismutase 1 (SOD1) mRNA	SOD1	other	—

Regulatory approvals (1)

Date	Agency	Marketed as	Applicant	Orphan
2023-04-25	FDA	—	BIOGEN MA	Yes

ATC classification

ATC code	Therapeutic hierarchy
N07XX22	NERVOUS SYSTEM › OTHER NERVOUS SYSTEM DRUGS › OTHER NERVOUS SYSTEM DRUGS › Other nervous system drugs

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• FDA label: SOD1

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	118.1
CSF protein increased	113.3

Chemical structure

• Molecular formula: C₂₃₀H₃₁₇N₇₂O₁₂₃P₁₉S₁₅
• Molecular weight: 7127.96 g/mol
• CAS number: —
• InChIKey: AZBXTZOFHIRMCD-UHFFFAOYSA-N
• PubChem CID: —

COCCO[C@@H]1[C@H](O)[C@@H](COP(S)(=O)O[C@@H]2[C@@H](COP(O)(=O)O[C@@H]3[C@@H](COP(S)(=O)O[C@@H]4[C@@H](COP(O)(=O)O[C@@H]5[C@@H](COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@H]6C[C@@H](O[C@@H]6COP(S)(=O)O[C@@H]6[C@@H](COP(O)(=O)O[C@@H]7[C@@H](COP(S)(=O)O[C@@H]8[C@@H](COP(O)(=O)O[C@@H]9[C@@H](COP(S)(=O)O[C@@H]%10[C@@H](CO)O[C@H]([C@@H]%10OCCOC)N%10C=C(C)C(N)=NC%10=O)O[C@H]([C@@H]9OCCOC)N9C=NC%10=C9N=CN=C%10N)O[C@H]([C@@H]8OCCOC)N8C=NC9=C8N=C(N)NC9=O)O[C@H]([C@@H]7OCCOC)N7C=NC8=C7N=C(N)NC8=O)O[C@H]([C@@H]6OCCOC)N6C=NC7=C6N=CN=C7N)N6C=C(C)C(=O)NC6=O)N6C=NC7=C6N=CN=C7N)N6C=C(C)C(N)=NC6=O)N6C=NC7=C6N=CN=C7N)N6C=C(C)C(=O)NC6=O)N6C=C(C)C(=O)NC6=O)N6C=C(C)C(=O)NC6=O)N6C=C(C)C(N)=NC6=O)N6C=C(C)C(=O)NC6=O)N6C=NC7=C6N=CN=C7N)O[C@H]([C@@H]5OCCOC)N5C=C(C)C(N)=NC5=O)O[C@H]([C@@H]4OCCOC)N4C=NC5=C4N=CN=C5N)O[C@H]([C@@H]3OCCOC)N3C=NC4=C3N=C(N)NC4=O)O[C@H]([C@@H]2OCCOC)N2C=C(C)C(N)=NC2=O)O[C@H]1N1C=C(C)C(=O)NC1=O

Frequently asked questions

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

What is Tofersen used for?

DrugCentral lists the following indications for Tofersen: Amyotrophic lateral sclerosis.

How does Tofersen work?

Its molecular targets in DrugCentral include SOD1.

When was Tofersen first approved?

Tofersen was first approved by the FDA in 2023.

What drug class does Tofersen belong to?

Tofersen is in the Antisense Oligonucleotide class.

What is the chemical formula of Tofersen?

Tofersen has the molecular formula C230H317N72O123P19S15 and a molecular weight of 7127.96 g/mol.

Sources

• DrugCentral drug card 5733 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3833346 — compound record and bioactivity
• DrugBank DB14782 — drug record
• FDA UNII 2NU6F9601K — substance identity
• DailyMed: FDA labels for Tofersen — current prescribing information (label search)
• UniProt P00441 (SOD1) — 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.