Qalsody
Qalsody (tofersen) is a medication used in adults with amyotrophic lateral sclerosis (ALS) who have the SOD1 mutation (also known as "SOD1-ALS"). It's given as an injection into the spine by a healthcare provider once per month. A common side effect of Qalsody (tofersen) is pain throughout your body and in your muscles, joints, and nerves.
What is Qalsody (tofersen)?
What is Qalsody (tofersen) used for?
- Amyotrophic lateral sclerosis (ALS) in adults with the SOD1 mutation (also known as "SOD1-ALS")
How Qalsody (tofersen) works
Qalsody (tofersen) is a type of molecule called an antisense oligonucleotide. It works by stopping a protein called SOD1 from being made. One of the causes of amyotrophic lateral sclerosis (ALS) is a buildup of toxic SOD1 in the brain and spinal cord due to a mutation in the protein. At this time, it's unclear whether stopping mutated SOD1 from being made is helpful for treating SOD1-ALS.
Drug facts
Side effects of Qalsody (tofersen)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Pain (42%)
- Fatigue (17%)
- Joint pain (14%)
- Muscle pain (14%)
Less Common Side Effects
- Muscle stiffness
- Nerve pain
Qalsody (tofersen) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- High blood pressure in the brain: changes in vision, headache, nausea, vomiting
- Brain inflammation: severe headache, neck stiffness, sensitivity to light, painful eye movements
The following Qalsody (tofersen) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Fever, neck pain or stiffness, sensitivity to light, headache, nausea, vomiting, confusion, which may be signs of meningitis
- Increased pressure around the brain—severe headache, blurry vision, change in vision, nausea, vomiting
- Pinched nerve—pain, tingling, or numbness located along the pathway of the nerve
- Severe back pain, numbness or weakness of the hands, arms, legs, or feet, loss of coordination, loss of bowel or bladder control
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Fatigue
- Joint pain
- Muscle pain
Pros and cons of Qalsody (tofersen)
Pros
- Given once per month (after the first 3 loading doses)
- Quick injection (takes 1 to 3 minutes)
- First and only treatment that targets a genetic cause of ALS
Cons
- Needs to be given as an injection into the spinal fluid by a healthcare provider
- Unclear what the benefits of the medication are at this time
- Can cause different types of pain as a side effect
Pharmacist tips for Qalsody (tofersen)
- Make sure to mark your appointments on your calendar for when you need to see your provider and get your Qalsody (tofersen) injections. Going to your appointments on time will help your provider see how well the medication is working and check for side effects.
- If you miss an injection of Qalsody (tofersen), contact your provider to get it as soon as possible. Your provider will let you know when you can get your next injection.
- Qalsody (tofersen) can cause different types of pain. This includes muscle pain, joint pain, nerve pain, and pain in your back, arms, or legs. If you experience pain that's too much to handle after you get your Qalsody (tofersen) injection, ask your provider if a pain medication is right for you.
Common questions about Qalsody (tofersen)
No. Qalsody (tofersen) is specifically approved for people with ALS who have the SOD1 mutation (also known as "SOD1-ALS"). This medication works by preventing mutated SOD1 from being made in your body. To know if you have SOD1-ALS, your provider will discuss with you about genetic testing. Once your results come back, they'll go over what your results mean and what treatment options there are for you.
Qalsody (tofersen) is given by a healthcare provider as an intrathecal injection. The injection will take about 1 to 3 minutes. The medication is injected into your lower back directly into your spinal fluid. Intrathecal injections help Qalsody (tofersen) get directly to your brain because the fluid from your spine flows to your brain. This allows the medication to work directly in your nervous system to prevent the buildup of mutated SOD1, which has been linked to ALS.
Studying a new medication usually takes many years. Sometimes, a medication can be approved in a shorter amount of time because it fills a medical need that isn’t currently met by other medications already available in the market. When the FDA approves a medication under the Accelerated Approval Program, the decision is based on a “marker” that suggests a clinical benefit (e.g., a medical condition improving). But for the medication to receive full approval, the manufacturer must prove the medication’s clinical benefit through additional studies at a later time. Qalsody (tofersen) got an accelerated approval after early studies showed that it lowered blood levels of proteins called neurofilament light (NfL). The level of these neurofilament proteins are typically high when there's nerve damage. So, a lower NfL level could mean Qalsody (tofersen) helps to prevent nerve damage. But additional studies are needed to confirm whether having lower NfL levels actually slows the worsening of ALS.
In clinical studies, people who took Qalsody (tofersen) for 6.5 months had less physical symptoms of ALS, but so did the placebo group. It's important to note that the Qalsody (tofersen) group had much lower blood levels of neurofilament light (NfL), which are proteins that typically indicate nerve damage. A lower level of NfLs might mean that Qalsody (tofersen) can help prevent nerve damage and slow progression of ALS, but more studies are needed to confirm this.
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Risks and warnings for Qalsody (tofersen)
Qalsody (tofersen) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Spinal cord inflammation and pinched nerve
Since Qalsody (tofersen) is injected through your back and into your spine, it's possible that the injection can pinch a nerve or cause inflammation and pain in your spinal cord. Let your provider know if you have symptoms such as weakness in the arms or legs; sharp lower back pain; burning, tingling, or numbness in the legs; or frequent urge to use the bathroom.
High blood pressure in the brain
Some people taking Qalsody (tofersen) had high pressure in the brain. This can lead to symptoms like changes in your vision, headache, nausea, and vomiting. Your provider will check your eyes for swelling, since this can also be a sign of high pressure in the brain. Let your provider know right away if you have any signs or symptoms of high pressure in the brain, such as frequent headaches, blurry vision, and other changes in vision.
Brain inflammation
Although rare, some people who took Qalsody (tofersen) developed brain inflammation (called aseptic meningitis). Contact your provider if you experience symptoms of meningitis, including severe headache, neck stiffness, sleepiness, eye sensitivity to light, painful eye movements, nausea, or vomiting.
Qalsody (tofersen) dosage
Typical dosage for Qalsody (tofersen)
The typical dose is 100 mg injected into the fluid of your spine (called an intrathecal injection). Qalsody (tofersen) is injected by a healthcare provider every 2 weeks for 3 doses, then once every 4 weeks.
Qalsody (tofersen) images
