All About Spinraza

What is Spinraza?

If you have spinal muscular atrophy (SMA), your doctor may prescribe Spinraza. It’s a medication used to treat SMA in adults and children.

SMA is a group of rare genetic disorders. It involves the loss of motor neurons (nerve cells in the spinal cord that control muscle movement). This leads to muscle weakness and muscle atrophy (wasting away of muscle).

To learn more about Spinraza’s use, see “Is Spinraza used for SMA?” below.

Spinraza basics

The active ingredient in Spinraza is nusinersen. An active ingredient is what makes a drug work.

Spinraza comes as a vial of liquid solution. You’ll receive it as an intrathecal injection (an injection into the fluid surrounding the spinal cord). A specially trained healthcare professional will administer Spinraza to you at a designated treatment center.

Read on to learn about Spinraza’s cost, dosage, side effects, and more.

What does Spinraza cost?

Costs of prescription drugs can vary depending on many factors. These factors include what your insurance plan covers and which pharmacy you use. To find current prices for Spinraza injections in your area, talk with your doctor. Also, talk with them if you have questions about how to pay for your prescription.

You may also want to enroll in a support program for Spinraza called SMA360. This program can help you learn about the financial assistance options available for the drug. To enroll, you’ll need to have your doctor fill out a start form. For more information, see the program webpage or call 844-477-4672.

You can also check out this article to learn more about saving money on prescriptions.

What are some frequently asked questions about Spinraza?

Find answers to some commonly asked questions about Spinraza.

What is Spinraza’s mechanism of action?

To treat SMA, Spinraza’s mechanism of action (how it works) is to increase the levels of a certain protein in the body.

People with spinal muscular atrophy (SMA) have low levels of a protein called SMN. This protein is important for proper functioning of nerve cells that control muscle movement.

Genes called SMN1 and SMN2 usually provide instructions that tell cells how to make SMN. In people with SMA, these instructions are missing or incomplete. Without enough SMN, nerve cells become damaged and die. This causes symptoms such as muscle weakness, muscle atrophy (wasting away of muscle), and trouble walking or breathing.

Spinraza increases the levels of SMN in your body. It does this by providing a missing piece in the instructions that tell your cells how to make SMN. This leads to increased levels of SMN in your body, which may reduce the symptoms of SMA.

For more information about Spinraza’s mechanism of action, talk with your doctor.

How does Spinraza affect the life expectancy of someone with SMA type 1?

It’s not entirely clear. In studies, Spinraza treatment prolonged overall survival in children with SMA type 1. But the life expectancy of individuals receiving this treatment is unknown. (Life expectancy refers to how long someone is expected to live.)

SMA type 1 is the most common type of SMA. Symptoms usually start before the age of 6 months. Without treatment, most children with SMA type 1 live less than 2 years.

There are ongoing long-term studies to learn more about Spinraza and whether it might affect life expectancy. Your doctor can discuss with you what to expect with Spinraza treatment.

How does Spinraza compare with Zolgensma and Evrysdi?

Spinraza, Zolgensma, and Evrysdi are all used to treat SMA. Spinraza and Evrysdi are used in adults and children of any age, while Zolgensma is used only in children younger than 2 years old.

Below is a chart that shows some other ways these drugs differ:

* The reason “-xioi” appears at the end of this drug’s name is to show that the drug is distinct from similar medications that may be created in the future.

Talk with your doctor or pharmacist for more information about how these treatments compare.

Is Spinraza a kind of gene therapy?

No, Spinraza isn’t a gene therapy. Gene therapy involves replacing a gene that isn’t working with one that does.

Instead, Spinraza is a type of drug called an antisense oligonucleotide. It works by increasing the levels of a protein called SMN. This protein helps nerve cells that control muscle movement. People with SMA, the condition Spinraza treats, don’t have enough SMN.

Your doctor can tell you more about gene therapy options available for your condition.

Does Spinraza cause hydrocephalus?

Hydrocephalus wasn’t seen in Spinraza’s studies. Hydrocephalus occurs when fluid collects in the brain, causing the head to increase in size.

However, some people who’ve received Spinraza have reported this condition. These reports occurred after the drug became available for use. It is unknown how often it occurred or if factors other than taking Spinraza may have contributed.

For more information about the risk of hydrocephalus with Spinraza treatment, talk with your doctor.

How is Spinraza given?

Your doctor will explain how Spinraza will be given to you. They’ll also explain how much you’ll receive and how often.

Before starting Spinraza treatment, your doctor will have you get blood tests to check the health of your kidneys and the level of platelets (a type of blood cell). You’ll repeat these blood tests before each injection. The results of these tests will help your doctor determine if it’s safe for you to receive the drug.

A specially trained healthcare professional will administer Spinraza to you at a designated treatment center. You cannot give yourself this drug at home.

Spinraza comes as a vial of liquid solution. Its route of administration is intrathecal. This means a healthcare professional injects the drug into the fluid surrounding your spinal cord.

How a Spinraza injection is given

A healthcare professional will administer Spinraza as an intrathecal injection. This procedure is also called a lumbar puncture.

Here are the basic steps of Spinraza administration:

1. A healthcare professional will help you get into the proper position for the procedure. You may be seated or lying on your side or front.
2. They’ll choose an area with no signs of infection or inflammation and use a disinfecting solution to clean the skin on your lower back. They may also give you medications, such as sedatives and local anesthetics. (Sedatives make you feel calm and sleepy. Local anesthetics help to numb your skin at the injection site.)
3. The healthcare professional will insert a needle into your lower back. They may use ultrasound or other imaging techniques to help guide the needle into the space between two bones in your lower spine.
4. Once they place the needle, they’ll remove a small amount of cerebrospinal fluid (CSF). (CSF is the fluid that surrounds your spinal cord and brain.)
5. Then the healthcare professional will inject Spinraza for 1 to 3 minutes.

The manufacturer provides more details about this procedure. If you have questions about how Spinraza is given, talk with your doctor.

Questions for your doctor

You may have questions about Spinraza and your treatment plan. It’s important to discuss all your concerns with your doctor.

Here are a few tips that might help guide your discussion:

• Before your appointment, write down questions such as:
  • How will Spinraza affect my body, mood, or lifestyle?
• Bring someone with you to your appointment if doing so will help you feel more comfortable.
• If you don’t understand something related to your condition or treatment, ask your doctor to explain it to you.

Remember, your doctor and other healthcare professionals are available to help you. And they want you to get the best care possible. So don’t be afraid to ask questions or offer feedback on your treatment.

Is Spinraza used for SMA?

Spinraza is used to treat spinal muscular atrophy (SMA) in adults and children of any age.

About SMA and its types

SMA is a group of rare genetic disorders. It involves the loss of motor neurons (nerve cells in the spinal cord that control muscle movement).

People with SMA have low levels of a protein called SMN. This protein is important for motor neurons to work.

Genes called SMN1 and SMN2 usually provide instructions that tell your cells how to make SMN. In people with SMA, these instructions are missing or incomplete. Without enough SMN, motor neurons start to die. This causes symptoms such as muscle weakness, muscle atrophy (wasting away of muscle), and trouble walking or breathing. These symptoms usually worsen over time.

There are different types of SMA. Along with other differences, SMA types vary in their usual onset (when symptoms appear).

• SMA type 1: Symptoms start before the age of 6 months.
• SMA type 2: Symptoms appear between the ages of 6 to 18 months.
• SMA type 3: Symptoms start after the age of 18 months.
• SMA type 4: Symptoms can begin at age 18 years but usually appear after age 35 years.

Types 1 and 2 are sometimes called infant-onset SMA, while types 3 and 4 are referred to as later-onset SMA. Type 4 is also called adult SMA.

About Spinraza for SMA

Spinraza increases the levels of SMN in your body. It does this by providing a missing piece in the instructions that tell your cells how to make SMN. This leads to increased levels of SMN in your body, which may reduce the symptoms of SMA.

For information about how Spinraza works and its effectiveness for treating SMA, talk with your doctor.

What is Spinraza’s dosage?

Your doctor will recommend the dosage of Spinraza that’s right for you. Below are commonly used dosages, but your doctor will let you know your dosing schedule.

Before starting Spinraza treatment, your doctor will have you get blood tests to check the health of your kidneys and the level of platelets (a type of blood cell). You’ll repeat these blood tests before each injection. The results of these tests will help your doctor determine if it’s safe for you to receive the drug.

Form

Spinraza comes as a vial of liquid solution. You’ll receive it as an intrathecal injection (an injection into the fluid surrounding the spinal cord). A specially trained healthcare professional will administer Spinraza to you at a designated treatment center.

Recommended dosages

Spinraza treatment begins with a series of four loading doses. A loading dose is a higher dose of a medication given at the beginning of treatment. It helps the drug start working sooner than it would if you received a lower initial dose.

After the first loading dose of Spinraza, you’ll receive the second dose 14 days later. Then you’ll wait another 14 days for the third. The fourth loading dose is given 30 days after the third dose.

After the loading doses, you’ll likely receive the maintenance (ongoing) dosage of Spinraza. This is usually one dose every 4 months.

Questions about Spinraza’s dosage

Below are some common questions about Spinraza’s dosage.

• What if I miss a dose of Spinraza? If you miss an appointment to receive Spinraza, contact the treatment center right away. Someone there will help you reschedule your injection as soon as possible.
• Will I need to use Spinraza long term? If you and your doctor decide that Spinraza is working well for you, you’ll likely use it long term.
• How long does Spinraza take to work? Spinraza starts working as soon as you receive your first dose. But it may take a few months before you notice any improvement in your muscle movement.

If you have additional questions about Spinraza’s dosage, ask your doctor.

What are Spinraza’s side effects?

Like most drugs, Spinraza may cause mild or serious side effects. The lists below describe some of the more common side effects that Spinraza may cause. These lists don’t include all possible side effects.

Keep in mind that side effects of a drug can depend on:

• your age
• other health conditions you have
• other medications you take

Your doctor or pharmacist can tell you more about the potential side effects of Spinraza. They can also suggest ways to help reduce side effects.

Mild side effects

Here’s a short list of some of the mild side effects that Spinraza can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read Spinraza’s prescribing information.

These side effects may vary depending on when the spinal muscular atrophy (SMA) began. (SMA is the condition that Spinraza is used to treat.)

Mild side effects of Spinraza that have been reported include:

• lower respiratory infection, such as bronchitis
• constipation
• fever
• headache
• vomiting
• back pain
• rash
• mild allergic reaction*

* To learn more about this side effect, see the “Allergic reaction” section below.

Mild side effects of many drugs may go away within a few days to a couple of weeks. But if they become bothersome, talk with your doctor or pharmacist.

Serious side effects

It’s possible to have serious side effects from taking Spinraza. If you think you’ve developed serious side effects from Spinraza, call your doctor right away. But if you think you’re having a medical emergency, you should call 911 or your local emergency number.

Serious side effects of Spinraza that have been reported include:

• kidney problems
• low level of platelets (a type of blood cell) and abnormal blood test results, both of which may indicate an increased risk of bleeding
• severe allergic reaction†

* To learn more about this side effect, see the “Allergic reaction” section below.

Allergic reaction

Some people may have an allergic reaction to Spinraza. Allergic reaction wasn’t seen in Spinraza’s studies but has been reported since the drug became available for use.

Symptoms of a mild allergic reaction can include:

• skin 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, usually in your eyelids, lips, hands, or feet. They can also include swelling of your tongue, mouth, or throat, which can cause trouble breathing.

Call your doctor right away if you have an allergic reaction to Spinraza. But if you think you’re having a medical emergency, call 911 or your local emergency number.

What should be considered before taking Spinraza?

This section covers some important things to discuss with your doctor when you’re considering treatment with Spinraza.

Interactions

Taking a medication with certain vaccines, foods, and other things can affect how the medication works. These effects are called interactions.

Before taking Spinraza, be sure to tell your doctor about all medications you take, including prescription and over-the-counter types. Also, describe any vitamins, herbs, or supplements you use. Your doctor or pharmacist can tell you about any interactions these items may cause with Spinraza.

For information about drug-condition interactions, see the “Warnings” section below.

Interactions with drugs or supplements

There currently aren’t any medications or supplements known to interact with Spinraza. But this doesn’t mean drug interactions with Spinraza won’t be recognized in the future. For example, new medications may be approved that interact with Spinraza.

For this reason, you should still tell your doctor and pharmacist about any medications you take besides Spinraza. This way, they can check for any new interactions during your treatment.

Warnings

Spinraza may not be right for you if you have certain medical conditions. These are known as drug-condition interactions. Other factors may also affect whether Spinraza is a good treatment option for you.

Talk with your doctor about your health history before you take Spinraza. Factors to consider include those in the list below.

• Allergic reaction. If you’ve had an allergic reaction to Spinraza or any of its ingredients, your doctor will likely not prescribe Spinraza. Ask them what other medications are better options for you.

Spinraza and alcohol

There are no known interactions between Spinraza and alcohol. If you drink alcohol, talk with your doctor about how much, if any, is safe for you to consume during treatment.

Pregnancy and breastfeeding

It is unknown whether Spinraza is safe for use if you’re pregnant or breastfeeding.

If you’re pregnant or planning a pregnancy, talk with your doctor to learn more about the risks and benefits of receiving this medication while pregnant.

If you’re breastfeeding or planning to breastfeed, talk with your doctor. They can help you decide the best way to feed your child while receiving Spinraza.