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Drug updated on 4/16/2024

Dosage FormInjection (intrathecal; 100 mg/15 mL [6.7 mg/mL] solution in a single-dose vial)
Drug ClassAntisense oligonucleotides
Ongoing and
Completed Studies


  • Indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

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  • Tofersen (Qalsody) is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene. This medication demonstrated biochemical effectiveness by reducing concentrations of SOD1 and neurofilament light chains but did not show significant improvements in clinical endpoints within the trial period.
  • A randomized controlled study focused on comparing Qalsody's safety and efficacy with a placebo among ALS patients carrying an SOD1 gene mutation.
  • In terms of effectiveness, Qalsody did not demonstrate a significant advantage over placebo during a 28-week observation period based on changes in ALS Functional Rating Scale-Revised scores. However, extending observation to 52 weeks showed potential benefits of early-start Qalsody over delayed-start.
  • Safety concerns were noted as adverse events related to lumbar puncture and neurological issues occurred in 7% of Qalsody recipients. These findings underscore the necessity of careful monitoring due to the intrathecal administration of this medication.
  • Subgroup analyses indicate that Qalsody might be more effective or benefits might only become evident among specific subsets, such as those predicted with a rapidly progressing disease course; further studies are required to verify these observations.
  • Although direct comparisons with other treatments were not available from this single study review, the analysis implies that early initiation of therapy using Tofersen may offer distinct benefits in managing SOD1-related ALS cases, pending confirmation from longer follow-up studies.

Product Monograph / Prescribing Information

Document TitleYearSource
Qalsody (tofersen) Prescribing Information. 2023Biogen MA Inc., Cambridge, MA

Randomized Controlled Trials

Document TitleSex DistributionYearSource
Trial of antisense oligonucleotide tofersen for SOD1 ALS.
F: 43%
M: 57%
2022New England Journal of Medicine

Sex Distribution:




Source:New England Journal of Medicine

Clinical Practice Guidelines

Document TitleYearSource
Iranian clinical practice guideline for amyotrophic lateral sclerosis.2023Frontiers in Neurology