Qalsody (tofersen)
An approved treatment for Amyotrophic Lateral Sclerosis.
The same compound appears under different names depending on the context. Here is how to identify Tofersen wherever you encounter it, plus the key facts at a glance.
- Generic name
- Tofersen
- Brand name
- Qalsody
- Development code
- BIIB067
- Drug class
- Antisense oligonucleotide
- Manufacturer
- Biogen / Ionis Pharmaceuticals
- How it's taken
- Tofersen is given as an injection directly into the spinal fluid (intrathecal).
The first treatment specifically targeting the genetic cause of SOD1-ALS. Delivered by spinal injection, it reduces production of the misfolded SOD1 protein that damages motor neurons.
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Where Tofersen fits
The only disease-modifying therapy specifically targeting SOD1-ALS. Patients with SOD1 mutations are typically identified through genetic testing and may begin tofersen as soon as diagnosis is confirmed.
How Tofersen works
Tofersen is made of a short DNA-like molecule that targets and blocks the instructions for making a toxic SOD1 protein. In SOD1-ALS, a genetic mutation causes the body to make a dangerous form of the SOD1 protein that damages and kills nerve cells. This drug works by finding and destroying the instructions before the toxic protein is even made.
Mechanism: Antisense oligonucleotide targeting SOD1 mRNA
Side effects and safety
Inflammation of the spinal cord and nerve root irritation have been reported, including serious cases. The most common side effects in the main study were pain (including back pain and pain in the arms or legs), tiredness, joint pain, muscle pain, and a rise in white blood cells in the spinal fluid. The label also warns of swelling of the optic nerve (papilledema), increased pressure inside the skull, and inflammation of the lining of the brain (aseptic meningitis). Contact your healthcare provider right away if you have pins and needles, numbness, or weakness (possible signs of spinal cord or nerve root inflammation), blurred or double vision, or headache with fever, vomiting, or a stiff neck.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Tofersen
Tofersen is given as an injection directly into the spinal fluid (intrathecal). Patients receive 3 loading doses 2 weeks apart, then one dose every 4 weeks after that. It is given by a healthcare provider experienced in performing lumbar punctures, usually at an ALS treatment center.
Availability and cost
Only available as the brand-name product.
Antisense oligonucleotide requiring complex chemical synthesis and intrathecal delivery, targeting an extremely small patient population (only a small fraction of ALS cases carry the SOD1 mutation).
Help paying for Qalsody
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Biogen Copay Program for Qalsody: US residents with non-government insurance generally eligible regardless of income. Medicare, Medicaid, VA/DoD, TRICARE may not be eligible.
For: private insurance · source - Infusion cost help
Administration Copay Program covers only approved anesthesia, imaging and administration codes; not labs or facility costs. Not for MA, MN or RI residents.
For: private insurance · source - Insurance and case manager help
Biogen Support Coordinators help with insurance, financial assistance options and treatment education.
The official page does not say who qualifies. Ask the program. · source
Good to know: Drug and administration copay programs require separate enrollment.
- From a charity · HealthWell FoundationAmyotrophic Lateral Sclerosis fundOpen
Pays for: Copays, premiums or other treatment costs.
- From a charity · NORD RareCareALS Travel & Lodging Assistance fundOpen
Pays for: Travel and lodging for care.
The foundation says: “Accepting Applications” - From a charity · Muscular Dystrophy AssociationMDA Durable Medical Equipment (DME) Grant Program fundApply directly
Pays for: Medical equipment (wheelchairs, lifts, canes and other DME), up to $1,000 per year.
The foundation says: “Status not shown on page” - From a charity · The ALS AssociationHoffman ALS Financial Assistance Program fundApply directly
Pays for: Travel to ALS clinic (transportation, lodging, lost wages) and clinic copays/deductibles, up to $1,000 per year.
The foundation says: “Status not shown on page” - From a charity · The Assistance FundAmyotrophic Lateral Sclerosis (ALS) fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Access and eligibility
Qalsody is only for adults with ALS caused by a mutation in the SOD1 gene, which accounts for roughly 2% of all ALS cases. Genetic testing is required to confirm SOD1-ALS before starting treatment.
Source: Qalsody.com (Biogen)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
FDA granted accelerated approval to tofersen on April 25, 2023, based on a drop in a blood marker of nerve damage called neurofilament light chain (NfL). In the placebo-controlled VALOR study (Study 1 Part C), at week 28 tofersen lowered SOD1 protein in the spinal fluid by 35% and lowered plasma NfL by 55%, compared with a 12% rise on placebo. The main clinical measure, the ALSFRS-R function score, showed less decline on tofersen but the difference was not statistically significant. Continued approval depends on a confirmatory trial.
Development history
Tofersen was developed jointly by Ionis Pharmaceuticals and Biogen as the first approved treatment specifically targeting a genetic cause of ALS. It gained FDA accelerated approval in 2023, representing a major breakthrough for people with SOD1-ALS, which accounts for about 2% of all ALS cases.
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Common questions about Tofersen
▸What is Tofersen (Qalsody)?
The first treatment specifically targeting the genetic cause of SOD1-ALS. Delivered by spinal injection, it reduces production of the misfolded SOD1 protein that damages motor neurons.
▸How does Tofersen work?
Tofersen is made of a short DNA-like molecule that targets and blocks the instructions for making a toxic SOD1 protein. In SOD1-ALS, a genetic mutation causes the body to make a dangerous form of the SOD1 protein that damages and kills nerve cells. This drug works by finding and destroying the instructions before the toxic protein is even made.
▸What are the side effects of Tofersen?
Inflammation of the spinal cord and nerve root irritation have been reported, including serious cases. The most common side effects in the main study were pain (including back pain and pain in the arms or legs), tiredness, joint pain, muscle pain, and a rise in white blood cells in the spinal fluid. The label also warns of swelling of the optic nerve (papilledema), increased pressure inside the skull, and inflammation of the lining of the brain (aseptic meningitis). Contact your healthcare provider right away if you have pins and needles, numbness, or weakness (possible signs of spinal cord or nerve root inflammation), blurred or double vision, or headache with fever, vomiting, or a stiff neck.
▸How is Tofersen taken?
Tofersen is given as an injection directly into the spinal fluid (intrathecal). Patients receive 3 loading doses 2 weeks apart, then one dose every 4 weeks after that. It is given by a healthcare provider experienced in performing lumbar punctures, usually at an ALS treatment center.
▸Is Tofersen FDA approved?
Yes, Tofersen (Qalsody) is FDA approved (2023) for the treatment of Amyotrophic Lateral Sclerosis.
▸How is tofersen different from riluzole and edaravone?
Tofersen targets the genetic root cause of SOD1-ALS by blocking production of the toxic SOD1 protein, while riluzole and edaravone treat ALS symptoms more broadly. Riluzole reduces glutamate toxicity across all ALS types, and edaravone scavenges free radicals to slow oxidative damage. Tofersen is the only therapy that directly addresses a specific genetic mutation driving the disease, but it only works for the roughly 2% of ALS patients who carry a SOD1 mutation.
▸How do I know if I have SOD1-ALS?
SOD1-ALS is diagnosed through genetic testing, usually a blood test that sequences the SOD1 gene. Your neurologist can order this test. It is recommended for anyone with ALS and a family history of the disease, and increasingly for all ALS patients regardless of family history, since SOD1 mutations are also found in people with no known family history of ALS. Results typically take a few weeks. A confirmed SOD1 mutation is required before starting tofersen.
▸What does the intrathecal injection feel like?
Tofersen is delivered by lumbar puncture (spinal tap), where a needle is inserted into the lower back to inject the drug into the spinal fluid. Most patients report brief pressure or a pinch during insertion. The procedure takes about 15 to 30 minutes, and you may be asked to lie flat for a period afterward to reduce the chance of headache. Some patients experience post-procedure headaches, back pain, or soreness at the injection site that usually resolves within a day or two.
▸How quickly does tofersen start working?
In the main trial, plasma neurofilament light chain (a blood marker of nerve damage) fell until about day 113 (roughly 16 weeks) and then stayed down, and at week 28 SOD1 protein in the spinal fluid was 35% lower than at the start. Clinical stabilization may take longer to observe, and the degree of benefit can vary depending on how early in the disease course treatment begins.
▸Can tofersen be used alongside riluzole or edaravone?
Yes. Tofersen targets a different mechanism than riluzole or edaravone, and many patients in clinical trials were also taking riluzole. There are no known drug interactions that prevent combination use. Your neurologist will determine the best combination of therapies based on your specific situation.
▸What are the serious risks of tofersen treatment?
The most significant risks include myelitis (inflammation of the spinal cord) and radiculitis (nerve root irritation), which can cause pins and needles, numbness, or weakness. These events are uncommon but can be serious. Patients receiving tofersen should report any new or worsening neurological symptoms immediately. The label asks doctors to watch for these symptoms and to start a medical workup and treatment if they appear. It also lists swelling of the optic nerve (papilledema), increased pressure inside the skull, and aseptic meningitis as serious risks.
▸What happens if I stop taking tofersen?
Tofersen requires ongoing treatment to maintain its effect. If treatment is stopped, SOD1 protein levels are expected to rise again over time. The exact timeline of how quickly the disease may progress after stopping treatment is not fully established, but the drug does not permanently alter the SOD1 gene. Decisions about stopping or pausing treatment should be made with your neurologist.