Treatment

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FDA APPROVED — GENE-SPECIFIC · Qalsody

FDA APPROVED — GENE-SPECIFIC

Key facts

Generic name
tofersen
Brand name(s)
Qalsody
Gene requirement
SOD1
Genetic confirmation
Required / verify with the care team and official indication
ALS population
Adults with ALS associated with a mutation in the SOD1 gene
Category
ALS disease-targeting treatment
Regulatory status
FDA APPROVED — GENE-SPECIFIC
FDA approval date
04-25-2023
Approval pathway
Accelerated approval
Country / availability
United States
Route(s)
Intrathecal spinal injection / lumbar puncture
Administration
Requires administration or supervision by an experienced healthcare professional.

What is it?

A gene-specific antisense treatment for adults with SOD1-associated ALS. It is not approved as a general ALS treatment or for other ALS genes.

Indication: FDA approved Qalsody for patients with ALS associated with a mutation in the SOD1 gene.

Who is it for?

Adults with ALS associated with a mutation in the SOD1 gene

How does it work?

Mechanism: Antisense oligonucleotide that targets SOD1 mRNA

Target: SOD1 mRNA / SOD1 protein production

How is it administered?

Intrathecal spinal injection / lumbar puncture

The official record describes administration by or with experienced healthcare professionals.

Available formulations

What evidence supported approval?

The FDA Drug Trials Snapshot says the findings are reasonably likely to predict clinical benefit and that continued approval may depend on confirmatory verification of clinical benefit.

What is known about benefit?

The FDA approval was based on a reduction in plasma neurofilament light, a biomarker of nerve injury and neurodegeneration. Biomarker evidence is not the same as confirmed clinical benefit.

Important safety information

Qalsody is administered intrathecally by experienced healthcare professionals and has important neurologic and procedure-related safety information. Review the official prescribing information and discuss risks with the care team.

This page does not provide dosing, prescribing, or individualized medical advice.

Gene connection

Related research

Research opportunities are separate from treatment availability. Eligibility, recruitment, and study status must be confirmed in the Research record.

Official Sources

U.S. Food and Drug Administration

FDA — Qalsody Drug Trials Snapshot

A direct source for reviewing the medical, genetic, research, or policy information discussed on this page.

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Last verified: 09-30-2026

Treatment information is provided for education and navigation and does not replace medical advice. Whether a treatment is appropriate depends on the individual’s diagnosis, genetic status, symptoms, health status, and other clinical factors. Treatment decisions should be made with the person’s ALS care team.

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