Tofersen
Qalsody (tofersen) is an antisense oligonucleotide therapy designed for adults with amyotrophic lateral sclerosis (ALS) who have a mutation in the superoxide dismutase 1 (SOD1) gene. It is the first FDA-approved treatment targeting a genetic cause of ALS, working to reduce the production of toxic SOD1 protein that contributes to disease progression. This personalized medicine represents a significant advancement in treating this specific subtype of ALS.
Qalsody is an antisense oligonucleotide therapy for ALS patients with SOD1 gene mutations, designed to reduce production of a toxic protein driving their disease.
Key facts at a glance
- RxNorm
- 2635001
- Generic
- Qalsody
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Qalsody is indicated for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene. This represents a specific genetic subtype of ALS affecting a subset of patients. Genetic testing is required to confirm the SOD1 mutation before treatment initiation.
Qalsody is an antisense oligonucleotide (ASO) that binds to SOD1 messenger RNA (mRNA), which is the genetic template for producing the SOD1 protein. By binding to this mRNA, the drug promotes its degradation, thereby reducing the production of the mutant SOD1 protein believed to be toxic and contributing to motor neuron damage in affected patients.
Qalsody is administered via intrathecal injection (into the spinal canal). Common side effects may include pain, fatigue, and CSF-related reactions. Close monitoring of clinical response and potential side effects is recommended. Treatment should be initiated and supervised by neurologists experienced in managing ALS. Efficacy is specific to patients with confirmed SOD1 mutations.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy