All About Spinraza

Spinraza cost and savings

Costs of prescription drugs can vary depending on many factors, including where you get your injections and your insurance coverage if you have insurance. 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.

Frequently asked questions about Spinraza

Find answers to some commonly asked questions about Spinraza.

What is Spinraza’s mechanism of action?

Spinraza’s mechanism of action (how it works) for treating SMA is to increase the levels of a protein called SMN in the body. This protein helps nerve cells that control muscle movement. Spinraza increases SMN levels by providing a missing piece in the genetic instructions that tell your cells how to make SMN.

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:

Talk with your doctor 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.

Spinraza uses

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

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.

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.

How Spinraza is 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.

Spinraza injection procedure

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.

Spinraza dosage

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

Form and strength

Spinraza comes as a vial of liquid solution in a strength of 12 milligrams per 5 milliliters (also written as 2.4 mg/mL). 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. These loading doses are given more frequently at the beginning of treatment (every 14 days, instead of every 4 months). This 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 dose. 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

Here 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 receive Spinraza long term? If you and your doctor decide that Spinraza is working well for you, you’ll likely take 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.

Spinraza side effects

Like most drugs, Spinraza may cause mild or serious side effects. The following lists 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 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 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 include:

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

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.

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 can 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
• hydrocephalus
• serious infections such as meningitis
• severe allergic reaction

Allergic reaction

For some people, Spinraza can cause an allergic reaction. In general, symptoms of an allergic reaction can be mild or serious.

What might help

If you have mild symptoms of an allergic reaction, such as a mild rash, call your doctor right away. They may suggest a treatment to manage your symptoms and can determine whether you should keep taking Spinraza.

If you have symptoms of a severe allergic reaction, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms could be life threatening and require immediate medical care.

If your doctor confirms you’ve had a serious allergic reaction to Spinraza, they may have you switch to a different treatment.

Spinraza interactions

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.

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

If you have questions about interactions that may affect you, talk with your doctor.