Spinraza is a brand-name prescription drug. It’s FDA-approved to treat spinal muscular atrophy (SMA) in adults and children.

SMA is a rare genetic disorder* that affects the central nervous system, which includes your spinal cord and brain. Specifically, SMA is a type of motor neuron disease. This means it results from problems with the neurons (nerve cells) that control muscle movement. Over time, SMA leads to muscle weakness and trouble walking, speaking, breathing, and swallowing.

For more information about this condition, see the “Spinraza for SMA” section below.

* A genetic disorder is a condition that causes changes to the DNA in the body’s cells.

Drug details

Spinraza contains the active drug nusinersen. It belongs to a class of drugs* called antisense oligonucleotides. Spinraza works by targeting a certain protein that helps nerve cells function correctly. For more information, see the “How Spinraza works” section below.

Spinraza comes as a liquid solution inside of a vial. The drug is given as an intrathecal injection (an injection directly into the fluid in your spinal cord). Only a trained healthcare professional should give Spinraza injections. You won’t give yourself injections of the drug.

Spinraza comes in one strength: 12 milligrams (mg) per 5 milliliters (mL) of solution. To learn how often Spinraza is typically used, see the “Spinraza dosage” section below.

* A class of drugs is a group of medications that work in a similar way.

Orphan drug status

In 2016, the Food and Drug Administration (FDA) approved Spinraza to treat SMA, granting it orphan drug status. The FDA gives this status to medications that are prescribed for rare conditions, such as SMA. The purpose is to encourage more research into conditions that don’t have many treatment options.

A pharmaceutical company may request orphan drug status from the FDA for a drug it produces. But the drug’s safety and effectiveness still have to be shown in a clinical study.

Effectiveness

For information about the effectiveness of Spinraza, see the “Spinraza for SMA” section below.

Spinraza is available only as a brand-name medication. It’s not currently available in generic form.

A generic drug is an exact copy of the active drug in a brand-name medication. Generics usually cost less than brand-name drugs.

As with all medications, the cost of Spinraza can vary. To find current prices for Spinraza in your area, talk with your doctor or pharmacist. The actual price you’ll pay depends on your insurance plan and your location. It also depends on the cost of your visit to the treatment center where you receive your Spinraza dose.

Before approving coverage for Spinraza, your insurance company may require you to get prior authorization. This means that your doctor and insurance company will need to communicate about your prescription before the insurance company will cover the drug. The insurance company will review the prior authorization request and decide if the drug will be covered.

If you’re not sure if you’ll need to get prior authorization for Spinraza, contact your insurance company.

Financial and insurance assistance

If you need financial support to pay for Spinraza, or if you need help understanding your insurance coverage, help is available.

Biogen, the manufacturer of Spinraza, offers a program called SMA360°. For more information and to find out if you’re eligible for support, call 844-4SPINRAZA (844-477-4672) or visit the program website.

Generic version

Spinraza isn’t available in a generic form. A generic drug is an exact copy of the active drug in a brand-name medication. Generics tend to cost less than brand-name drugs.

Other drugs are available that can treat spinal muscular atrophy (SMA). Some may be a better fit for you than others. If you’re interested in finding an alternative to Spinraza, talk with your doctor. They can tell you about other medications that may work well for you.

Examples of other drugs that may be used to treat SMA include:

  • risdiplam (Evrysdi)
  • onasemnogene abeparvovec-xioi (Zolgensma)

The Food and Drug Administration (FDA) approves prescription drugs such as Spinraza to treat certain conditions. Spinraza may also be used off-label for other conditions. Off-label drug use means using a drug for a purpose other than what it’s been approved for by the FDA.

Spinraza is approved to treat spinal muscular atrophy (SMA) in adults and children.

About SMA

SMA is a rare genetic disorder* that affects the central nervous system, which includes your spinal cord and brain. Specifically, SMA is a type of motor neuron disease. This means it results from problems with the neurons (nerve cells) that control muscle movement.

The survival motor neuron (SMN) protein helps nerve cells work correctly. People with SMA don’t have enough of this protein. This happens because of a problem with a gene called SMN1, which makes the SMN protein.

Without enough SMN protein, nerve cells aren’t able to control muscle movements. Over time, SMA leads to muscle weakness and trouble walking, speaking, breathing, and swallowing.

There are several types of SMA:

  • SMA type 0. This is the most severe form of SMA. Symptoms usually begin before birth.
  • SMA type 1. This is the most common form of SMA. Symptoms usually begin within the first 6 months after birth.
  • SMA type 2. Symptoms for this type of SMA usually begin by age 18 months.
  • SMA type 3. Symptoms for this type of SMA usually begin after age 18 months but may not appear until early adulthood.
  • SMA type 4. This is the least severe form of SMA. Symptoms usually begin in adulthood, often after age 30 years.

* A genetic disorder is a condition that causes changes to the DNA in the body’s cells.

Symptoms of SMA

Symptoms of SMA vary depending on the age when they first appear. Below are common symptoms of SMA that may occur in different age groups:

  • In a fetus:
  • In infants and children:
    • breathing problems
    • inability to kick while lying on their back
    • lack of strong reflexes
    • inability to sit
    • problems eating that may lead to choking or trouble swallowing
  • In adolescents and adults:
    • inability to stand or walk without assistance
    • muscle weakness

Effectiveness for SMA

Spinraza has been found effective for treating SMA. It was the first drug to receive FDA approval for treating this condition.

More research has been done for Spinraza’s use in SMA types 1 and 2 than for other types of SMA. But because there are limited treatment options for this condition, the drug is approved to treat all SMA types.

For more information on how Spinraza performed in clinical studies, see the drug’s prescribing information.

Spinraza and children

Spinraza is FDA-approved to treat all types of SMA in children of any age. For more information, see the “Spinraza for SMA” section above.

To learn how well the drug worked for children in clinical studies, see Spinraza’s prescribing information.

Spinraza is approved by the Food and Drug Administration (FDA) to treat spinal muscular atrophy (SMA) in adults and children.

What happens with SMA

SMA is a rare genetic disorder* that affects the central nervous system, which includes your spinal cord and brain. Specifically, SMA is a type of motor neuron disease. This means it results from problems with the neurons (nerve cells) that control muscle movement.

The survival motor neuron (SMN) protein helps nerve cells work correctly. People with SMA don’t have enough of this protein.

Without enough SMN protein, nerve cells are unable to control muscle movements. Over time, SMA leads to muscle weakness and trouble walking, speaking, breathing, and swallowing.

* A genetic disorder is a condition that causes changes to the DNA in the body’s cells.

What Spinraza does

Spinraza contains the active drug nusinersen. It belongs to a class of drugs* called antisense oligonucleotides.

Spinraza works by increasing the amount of SMN protein that’s available in the body. In a person who has SMA, this helps nerve cells better control muscle movements. As a result, their symptoms of SMA decrease.

It’s important to note that Spinraza isn’t a gene therapy. With gene therapy for SMA, the SMN1 gene that isn’t working correctly is replaced with an SMN1 gene that is working correctly. Onasemnogene abeparvovec-xioi (Zolgensma) is currently the only gene therapy that is FDA-approved for use in treating SMA.

The way Spinraza works to treat SMA in the body is called its mechanism of action. For more information, visit the drug manufacturer’s website.

* A class of drugs is a group of medications that work in a similar way.

How long does it take to work?

Spinraza starts working to treat SMA as soon as it’s injected. In clinical studies, more than half of the people who were given Spinraza showed a decrease in their SMA symptoms within 15 months of their first dose.

Your doctor will typically start you on the dosage recommended by Spinraza’s manufacturer. Then they’ll monitor your condition over time to make sure the drug is working for you.

The following information describes dosages that are commonly used or recommended. But your doctor will determine the best dosage to fit your needs.

Drug form and strength

Spinraza comes as a liquid solution inside of a vial. A healthcare professional will give you the drug as an intrathecal injection. (This is an injection directly into the fluid in the spinal cord.) The injection takes about 1 to 3 minutes.

To inject the drug, the healthcare professional uses a procedure called a lumbar puncture. For this procedure, they insert a needle into the space between two of your vertebrae (the bones in your spine).

Spinraza comes in one strength: 12 milligrams (mg) per 5 milliliters (mL) of solution.

Dosage for SMA

When used to treat spinal muscular atrophy (SMA), the usual dose of Spinraza is 12 mg (5 mL) per injection.

Your dosing schedule for Spinraza will typically include loading dose injections and maintenance dose injections.

Your first four doses of Spinraza are called loading doses. These doses help quickly raise the level of the drug in your body. This way it starts working faster to treat your condition. For the first three doses of Spinraza, you’ll receive an injection every 14 days. Then you’ll receive the fourth dose 30 days after the third dose.

Your remaining doses of Spinraza are called maintenance doses. These doses help keep steady levels of the drug in your body over time. You’ll receive your first maintenance dose 4 months after the last loading dose (fourth injection). After that, you’ll receive a dose every 4 months.

Children’s dosage

Spinraza is approved to treat SMA in children of any age. The usual dosage of Spinraza for treating SMA in children is the same as the dosage used for adults. For dosage details, see the “Dosage for SMA” section just above.

What if I miss a dose?

What to do if you miss a dose of Spinraza depends on whether you’ve missed a loading dose or a maintenance dose.

If you miss an appointment to receive a loading dose (one of your first four doses), call your doctor to reschedule as soon as possible. After you’ve been given your missed dose, you’ll receive your next dose according to your normal dosing schedule:

  • If you missed one of your first two doses, you’ll receive your third dose 14 days later.
  • If you missed your third dose, you’ll receive your fourth dose 30 days later.
  • If you missed your fourth dose, you’ll receive your fifth dose 4 months later.

If you miss an appointment to receive a maintenance dose, call your doctor to reschedule as soon as possible. After you’ve been given your missed dose, you’ll receive your next dose of Spinraza at least 4 months later.

To help make sure that you don’t miss an appointment for your Spinraza dose, try using a medication reminder. This may include setting an alarm on your phone or downloading a reminder app.

Will I need to use this drug long term?

Spinraza is meant to be used as a long-term treatment. If you and your doctor determine that Spinraza is safe and effective for you, you’ll likely use it long term.

Here are answers to some frequently asked questions about Spinraza.

What’s the life expectancy for someone with SMA type 1 who’s using Spinraza?

The typical life expectancy for someone with spinal muscular atrophy (SMA) type 1 who’s using Spinraza isn’t known. (Life expectancy refers to the number of years a person is expected to live.)

SMA type 1 is the most common type of SMA. Symptoms typically begin within the first 6 months after birth. In general, the life expectancy for a person with SMA type 1 that hasn’t been treated is less than 2 years.

Spinraza is approved to treat SMA in adults and children. The drug received approval for this use from the Food and Drug Administration (FDA) in 2016. So there haven’t been enough long-term studies of Spinraza to show how it may affect life expectancy in people with SMA.

A study evaluating the long-term effects of Spinraza is planned but hasn’t started yet.

If you have questions about what to expect with SMA or Spinraza treatment, talk with your doctor.

Is Spinraza used for ALS?

No, Spinraza isn’t currently used to treat amyotrophic lateral sclerosis (ALS). Spinraza is only FDA-approved to treat SMA in adults and children.

Both SMA and ALS are motor neuron diseases. These diseases affect the central nervous system, which includes your spinal cord and brain. Both conditions lead to muscle weakness over time.

But SMA and ALS may have different causes. SMA is a genetic disorder* caused by a problem with a specific protein in the neurons (nerve cells) that control muscle movement.† The exact cause of ALS isn’t known but may include genetic or environmental factors.

Currently, edavarone (Radicava) and riluzola (Rilutek) are the only FDA-approved treatments for ALS.

If you have questions about treatment options for ALS, talk with your doctor.

* A genetic disorder is a condition that causes changes to the DNA in the body’s cells.
† For more information, see “About SMA” in the “Spinraza for SMA” section above.

Can muscular dystrophy be treated with Spinraza?

No, Spinraza isn’t currently used to treat muscular dystrophy. Spinraza is only FDA-approved to treat SMA in adults and children.

Both SMA and muscular dystrophy are genetic disorders* that lead to muscle weakness over time. Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy.

Although both SMA and DMD are genetic conditions, they have different causes. SMA is caused by a problem with a specific protein in the neurons (nerve cells) that control muscle movement. DMD is caused by a problem with a specific protein that’s found in muscle cells.

While Spinraza isn’t currently approved to treat DMD, other medications in the same drug class† are. Spinraza belongs to a class of drugs called antisense oligonucleotides (ASOs). Examples of ASOs approved to treat DMD are eteplirsen (Exondys 51) and viltolarsen (Viltepso).

If you have questions about treatment options for muscular dystrophy, talk with your doctor.

* A genetic disorder is a condition that causes changes to the DNA in the body’s cells.
† A class of drugs is a group of medications that work in a similar way.

Spinraza can cause mild or serious side effects. The following lists contain some of the key side effects that may occur while using Spinraza. These lists do not include all possible side effects.

For more information about the possible side effects of Spinraza, talk with your doctor or pharmacist. They can give you tips on how to deal with any side effects that may be concerning or bothersome.

Note: The Food and Drug Administration (FDA) tracks side effects of drugs it has approved. If you would like to notify the FDA about a side effect you’ve had with Spinraza, you can do so through MedWatch.

Mild side effects

Some of the possible mild side effects* of Spinraza are listed below.

In clinical studies,† certain side effects were more common in people whose spinal muscular atrophy (SMA) symptoms began before 6 months of age:

The following side effects were more common in people whose SMA symptoms began after 6 months of age:

Most of these side effects may go away within a few days or a couple of weeks. But if they become more severe or don’t go away, talk with your doctor or pharmacist.

* This is a partial list of mild side effects from Spinraza. To learn about other mild side effects, talk with your doctor or pharmacist, or view Spinraza’s prescribing information.
† Clinical trials for Spinraza only included children or adolescents. But it’s expected that adults taking the drug may experience similar side effects.

Serious side effects

Serious side effects from Spinraza aren’t common, but they can occur. Call your doctor right away if you have serious side effects. Call 911 or your local emergency number if your symptoms feel life threatening or if you think you’re having a medical emergency.

Serious side effects, which are explained in “Side effect details” below, include:

Side effects in children

Spinraza is approved to treat SMA in children of any age. Clinical trials for Spinraza only included children or adolescents. But it’s expected that side effects from Spinraza are generally the same in both children and adults. For more information, see “Mild side effects” and “Serious side effects” above.

Side effect details

Here’s some detail on certain side effects this drug may cause.

Hydrocephalus

In rare cases, Spinraza may cause hydrocephalus (a buildup of spinal fluid in the brain). This side effect wasn’t common in clinical studies of the drug. But it has been reported by people who’ve used the drug after it became available to the public.

Hydrocephalus is a serious condition that causes pressure on brain tissue. If it isn’t treated, the condition can be fatal.

Symptoms of hydrocephalus can vary depending on age. Below are common symptoms of hydrocephalus that may occur in different age groups:

  • In infants:
    • bulge on the fontanel (soft spot) of their head
    • eyes that look downward or aren’t able to turn outward
    • rapid increase in the size of their head
    • seizures
  • In children, adolescents, and adults:
    • headache
    • memory loss
    • problems with coordination or balance
    • slowed progress with talking or walking
  • In older adults:
    • inability to control their bladder
    • loss of balance or coordination
    • trouble thinking or remembering
    • trouble walking

Tell your doctor right away if you have any symptoms of hydrocephalus. They’ll likely perform tests to check the fluid pressure on your brain. Depending on the severity of your condition, you may need surgery to remove the fluid.

Bleeding disorders

Spinraza can cause bleeding disorders, such as a low level of platelets (which play a role in forming blood clots) and problems with coagulation (the process of blood clotting). Bleeding disorders were common in clinical studies of the drug.

These side effects can cause you to bruise or bleed more easily than usual. Tell your doctor if you notice any unusual bruising or bleeding after starting Spinraza.

Your doctor will give you blood tests to check your platelet level and blood clotting before each Spinraza dose. If your doctor finds any signs of bleeding disorders, they may recommend a different treatment for your condition.

Kidney problems

Spinraza can cause kidney problems such as glomerulonephritis (a type of inflammation in the kidney). Kidney problems were a very common side effect reported in clinical studies of the drug.

Symptoms of kidney problems can include:

Before each Spinraza dose, your doctor will check the level of protein in your urine. Protein in your urine can be a sign of serious kidney problems. If you have kidney problems, your doctor may recommend a treatment other than Spinraza for your condition.

Allergic reaction

As with most drugs, some people can have an allergic reaction after receiving Spinraza. Symptoms of a mild allergic reaction can include:

  • rash
  • itchiness
  • flushing (temporary warmth, redness, or deepening of skin color)

A more severe allergic reaction is rare but possible. Symptoms of a severe allergic reaction can include:

  • swelling under your skin, typically in your eyelids, lips, hands, or feet
  • swelling of your tongue, mouth, or throat
  • trouble breathing

Call your doctor right away if you have an allergic reaction to Spinraza, as the reaction could become severe. Call 911 or your local emergency number if your symptoms feel life threatening or if you think you’re having a medical emergency.

Spinraza is administered by a healthcare professional as an intrathecal injection. This means they inject the drug directly into the fluid in your spinal cord. You won’t give yourself doses of Spinraza.

For this injection, the healthcare professional uses a procedure called a lumbar puncture: They insert a needle into the space between two of your vertebrae (the bones in your spine). The injection takes about 1 to 3 minutes.

The way the drug enters your system is called its route of administration. For more information, visit the manufacturer’s site.

When it’s administered

Spinraza is given by a healthcare professional in a treatment center that’s approved to administer the drug. Your doctor will set up appointments for you to receive your Spinraza injections.

Your first four doses of Spinraza are called loading doses. These doses help quickly raise the level of the drug in your body. This way it starts working faster to treat your condition. For the first three doses of Spinraza, you’ll receive an injection every 14 days. Then you’ll receive the fourth dose 30 days after the third dose.

Your remaining doses of Spinraza are called maintenance doses. These doses help keep steady levels of the drug in your body over time. You’ll receive your first maintenance dose 4 months after the last loading dose (fourth injection). After that, you’ll receive a dose every 4 months.

To help make sure that you don’t miss an appointment for your Spinraza dose, try using a medication reminder. This may include setting an alarm on your phone or downloading a reminder app.

There isn’t a known interaction between Spinraza and alcohol. If you drink alcohol, talk with your doctor about the amount that’s safe for you to drink while using Spinraza.

Spinraza isn’t known to interact with any other medications, supplements, or foods. Spinraza’s manufacturer didn’t report any interactions from clinical studies of the drug. But this doesn’t mean that interactions can’t happen with Spinraza.

Before starting Spinraza, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you use. Sharing this information can help you avoid potential interactions.

If you have questions about drug interactions that may affect you, ask your doctor or pharmacist.

Spinraza hasn’t been studied in pregnancy. It isn’t known whether the drug is safe to use while pregnant.

Animal studies have shown harm to offspring born to females that were given the drug during pregnancy. But animal studies don’t always reflect what will happen in humans.

If you’re pregnant or planning a pregnancy, be sure to tell your doctor before starting Spinraza. They may recommend a different treatment for your condition.

It’s not known if Spinraza is safe to use during pregnancy. If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control needs while you’re using Spinraza.

For more information about receiving Spinraza during pregnancy, see the “Spinraza and pregnancy” section above.

The use of Spinraza during breastfeeding hasn’t been studied. It isn’t known if the drug is safe to use during breastfeeding.

Animal studies have found nusinersen (the active drug in Spinraza) in the breast milk of lactating mice that were given the drug. But animal studies don’t always indicate what will happen in humans.

If you’re breastfeeding or planning to breastfeed while receiving Spinraza, talk with your doctor. They can explain the risks and benefits of breastfeeding while using the drug.

Before starting Spinraza, talk with your doctor about your health history. Spinraza may not be right for you if you have certain medical conditions or other factors affecting your health. These include:

  • Allergic reaction. If you’ve had an allergic reaction to Spinraza or any of its ingredients, you shouldn’t receive Spinraza. Ask your doctor which other medications are better options for you.
  • Pregnancy. It’s not known whether Spinraza is safe to use during pregnancy. For more information, see the “Spinraza and pregnancy” section above.
  • Breastfeeding. It’s not known whether Spinraza can pass into breast milk during breastfeeding. For more information, see the “Spinraza and breastfeeding” section above.

Note: For more information about the potential negative effects of Spinraza, see the “Spinraza side effects” section above.

Disclaimer: Medical News Today has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or other healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.

Spinraza Images