Tofersen

Pharmacology

• Tofersen is an antisense oligonucleotide (ASO)
• It is indicated for MND with SOD1 gene mutations
• It targets the SOD1 mRNA
• It may trigger an immune inflammatory response

Dosing

Adverse events

Outcomes

References

1. Miller T, Cudkowicz M, Shaw PJ, et al. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med 2020; 383:109-119.
2. Miller TM, Cudkowicz ME, Genge A, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med 2022; 387:1099-1110. 
3. Blair HA. Tofersen: first approval. Drugs 2023 (Online ahead of print).
4. Simonini C, Zucchi E, Martinelli I, et al. Neurodegenerative and neuroinflammatory changes in SOD1-ALS patients receiving tofersen. Sci Rep 2025; 15:11034. 
5. McGuigan A, Blair HA. Tofersen: a review in amyotrophic lateral sclerosis associated with SOD1 mutations. CNS Drugs 2025; 39:903-912. 
6. And 1 more. Subscribe to see the full list

Related checklists

• Riluzole
• Edaravone
• Motor neurone disease (MND): neurological symptomatic treatments
• Motor neurone disease (MND): systemic symptomatic treatments
• Motor neurone disease (MND): investigational drug treatments