R R

What Is Qalsody?

Qalsody is a prescription medication containing tofersen, an antisense oligonucleotide used to treat amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene. SOD1-related ALS is a genetic form of a progressive neurological disease that damages motor neurons responsible for voluntary muscle movement. Qalsody is administered into the fluid surrounding the spinal cord through an intrathecal injection. It received FDA accelerated approval based on a reduction in plasma neurofilament light chain, a biomarker associated with nerve injury. Its clinical benefit remains subject to confirmatory evaluation.

Link to This Resource Page

Provide a valuable resource to your clients or customers by linking to this resource page. Just place the following link on your website.

To display this...

What Is Qalsody?

Qalsody is a prescription medication containing tofersen, an antisense oligonucleotide used to treat amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene. SOD1-related ALS is a genetic form of a progressive neurological disease that damages motor neurons responsible for voluntary muscle movement. Qalsody is administered into the fluid surrounding the spinal cord through an intrathecal injection. It received FDA accelerated approval based on a reduction in plasma neurofilament light chain, a biomarker associated with nerve injury. Its clinical benefit remains subject to confirmatory evaluation.

read more about qalsody ...

Copy this HTML:

Copy HTML Copied!

How Does Qalsody Work?

Qalsody contains tofersen, which binds to messenger RNA produced from the SOD1 gene. This promotes degradation of the targeted RNA and reduces production of SOD1 protein. Certain SOD1 mutations are associated with toxic protein activity that contributes to motor neuron damage in affected patients. Reducing SOD1 production is intended to address this disease-related pathway. Qalsody does not reverse motor neuron loss that has already occurred. The FDA's accelerated approval was based on reduced plasma neurofilament light chain concentrations rather than confirmed improvement in long-term clinical outcomes.

Who Can Receive Qalsody Treatment?

Qalsody is approved for adults with ALS who have a confirmed mutation in the SOD1 gene. Genetic testing is necessary to identify patients with this form of ALS because the medication targets SOD1 production. It is not approved for ALS patients without an SOD1 mutation or for other neurological disorders. A neurologist considers the genetic diagnosis, disease progression, functional status, and risks associated with repeated lumbar punctures before recommending treatment. Patients require monitoring for neurological complications, including inflammation affecting the spinal cord or nerve roots.

How Is Qalsody Administered?

Qalsody is administered by intrathecal injection through a lumbar puncture performed by or under the direction of a healthcare professional experienced with the procedure. The recommended dose is 100 mg in 15 mL per administration. Treatment begins with three loading doses given at 14-day intervals, followed by a maintenance dose every 28 days. Before injection, approximately 10 mL of cerebrospinal fluid is removed according to the prescribing instructions. Qalsody is then administered as an intrathecal bolus over one to three minutes. Patients should follow the scheduled appointments and receive monitoring for procedure-related and neurological symptoms.

What Are the Side Effects and Warnings of Qalsody?

Common adverse effects include pain, fatigue, joint pain, muscle pain, and increased white blood cells in the cerebrospinal fluid. Serious neurological complications include myelitis, radiculitis, papilledema with elevated intracranial pressure, and aseptic meningitis. These conditions can cause headache, visual changes, nerve pain, weakness, or other neurological symptoms. Lumbar punctures can also cause procedure-related headache and discomfort. Patients should promptly report new or worsening weakness, persistent severe headache, eye symptoms, fever, neck stiffness, or unusual sensory changes. Treatment complications require medical assessment and appropriate management.

Common Questions About Qalsody

Is Qalsody the same as tofersen?

Yes. Qalsody is the brand name for tofersen, an antisense oligonucleotide administered intrathecally for SOD1-related ALS.

Can Qalsody treat every type of ALS?

No. Qalsody is approved only for adults with ALS associated with an SOD1 gene mutation. Genetic testing is required to establish eligibility.

How often is Qalsody administered?

Qalsody begins with three intrathecal loading doses given 14 days apart, followed by a maintenance dose every 28 days.

Can Qalsody cause vision problems?

Yes. Papilledema and elevated intracranial pressure have been reported. New blurred vision, significant headaches, or other visual changes require prompt medical evaluation.

References

DailyMed - QALSODY (tofersen) injection. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=81356b45-1cb7-4eef-88ea-e44cc18b47c5. Date Accessed November 2, 2026.

Drug Trials Snapshots: QALSODY. U.S. Food and Drug Administration. https://www.fda.gov/drugs/drug-trials-snapshots/drug-trials-snapshots-qalsody. Date Accessed November 2, 2026.

FDA Approves Treatment of Amyotrophic Lateral Sclerosis Associated With a Mutation in the SOD1 Gene. U.S. Food and Drug Administration. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene. Date Accessed November 2, 2026.

Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. The New England Journal of Medicine. https://pubmed.ncbi.nlm.nih.gov/32640130/. Date Accessed November 2, 2026.

Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. The New England Journal of Medicine. https://www.nejm.org/doi/full/10.1056/NEJMoa2204705. Date Accessed November 2, 2026.