Starting ZOLGENSMA

Ask your doctor if ZOLGENSMA® (onasemnogene abeparvovec-xioi) is right for your child.

To learn if ZOLGENSMA is right for your child, visit your child’s doctor to discuss the benefits and risks of treatment and if your child qualifies for ZOLGENSMA. Your doctor can address any questions or concerns you may have. If you and your doctor agree to pursue treatment with ZOLGENSMA, take a look at the steps to starting below.

Take your knowledge further with A Caregiver’s Guide to ZOLGENSMA.

Download A Caregiver's Guide to ZOLGENSMA

Starting ZOLGENSMA

Ask your doctor if ZOLGENSMA is right for your child

To learn if ZOLGENSMA is right for your child, visit your child’s doctor to discuss the benefits and risks of treatment and if your child qualifies for ZOLGENSMA. Your doctor can address any questions or concerns you may have. If you and your doctor agree to pursue treatment with ZOLGENSMA, take a look at the steps to starting below.

Take your knowledge further with A Caregiver’s Guide to ZOLGENSMA.

Download A Caregiver's Guide to ZOLGENSMA
Scroll Down Icon

Starting ZOLGENSMA

Ask your doctor if ZOLGENSMA® (onasemnogene abeparvovec-xioi) is right for your child.

To learn if ZOLGENSMA is right for your child, visit your child’s doctor to discuss the benefits and risks of treatment and if your child qualifies for ZOLGENSMA. Your doctor can address any questions or concerns you may have. If you and your doctor agree to pursue treatment with ZOLGENSMA, take a look at the steps to starting below.

Take your knowledge further with A Caregiver’s Guide to ZOLGENSMA.

Download A Caregiver's Guide to ZOLGENSMA

Starting ZOLGENSMA

Ask your doctor if ZOLGENSMA is right for your child

To learn if ZOLGENSMA is right for your child, visit your child’s doctor to discuss the benefits and risks of treatment and if your child qualifies for ZOLGENSMA. Your doctor can address any questions or concerns you may have. If you and your doctor agree to pursue treatment with ZOLGENSMA, take a look at the steps to starting below.

Take your knowledge further with A Caregiver’s Guide to ZOLGENSMA.

Download A Caregiver's Guide to ZOLGENSMA

Find a treatment center

If you and your doctor decide that ZOLGENSMA is right for your child, there are a few places you can contact to find a treatment center. The organizations below have a list of treatment centers that are unique to each of them. For a full list of treatment centers, you may want to contact both organizations to find the one closest or most convenient for your family.

See the steps to starting ZOLGENSMA

Read the transcript

NARRATOR: Welcome to the steps to treatment for ZOLGENSMA® (onasemnogene abeparvovec-xioi).

Once you and your child’s doctor have chosen treatment with ZOLGENSMA, there are a few steps that need to be taken.

Before ZOLGENSMA can be given to your child, a diagnosis of spinal muscular atrophy, or SMA, has to be confirmed by a genetic test. Your doctor will order a genetic test for your child to confirm the SMN1 gene is missing or nonworking. In addition, your doctor may also test your child’s SMN2 gene copy number. Your doctor will also need to perform additional lab tests—an AAV9 antibody test and baseline medical tests. These tests include liver function, creatinine, complete blood count, including hemoglobin and platelet count, and troponin-I.

While waiting for the genetic confirmation and AAV9 antibody tests, your doctor can submit 2 forms: a ZOLGENSMA Prescription Form and a Patient Consent Form. By signing the consent form, you will be connected with the OneGene Program. This is a dedicated resource provided by Novartis Gene Therapies that supports your family and helps you to navigate the insurance process, including prior authorizations and benefits.

You will also be assigned a Patient Resource Manager, who can share educational resources and guidance about preparing for treatment.

Your doctor’s office will follow up on the status of your insurance coverage for ZOLGENSMA. If approved, and if not previously completed, your doctor will perform baseline medical tests before treatment with ZOLGENSMA.

Your doctor will also prescribe an oral corticosteroid, a medicine that must be given to your child the day before infusion, on infusion day, and continued after infusion as directed by your doctor. This will help manage potential reactions to ZOLGENSMA by your child’s immune system.

On treatment day your child will be infused with ZOLGENSMA. The actual ZOLGENSMA infusion takes about 60 minutes. Your child will also receive a second dose of the oral corticosteroid. After infusion, your doctor and care team will talk to you about post-treatment follow-up and additional medical monitoring.

You will continue to give your child the oral corticosteroid as it is prescribed by your doctor, which is recommended for at least 2 months after infusion. In addition to liver function, your doctor will monitor other markers through blood tests, which will require weekly appointments for the first month and less frequently in the second and third months, or longer, as determined by your doctor.

Contact your doctor immediately if your child misses a dose of the corticosteroid or vomits it up, or if their skin and/or whites of the eyes appear yellowish. You should also talk to your doctor about what to watch for after infusion, including the signs and symptoms of a viral respiratory infection, adjustments to your child’s vaccination schedule, and how to handle bodily waste in the first month.

Thrombotic microangiopathy (TMA) has been reported to occur approximately one week following infusion. Seek immediate medical attention if your child experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

It’s important to understand that your child still has SMA after treatment with ZOLGENSMA. So work with your doctor and care team to understand any additional therapies and support that can help manage your child’s SMA. This could include physical and occupational therapy, along with nutritional and breathing support or cough assist, or both.

For more helpful information, read the complete steps to starting treatment at ZOLGENSMA.com.

If you have questions about ZOLGENSMA, talk to your doctor today.

Close the transcript

Take the next step for your child

Before treatment

Pretreatment testing to determine if your child qualifies for ZOLGENSMA

ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). It is given as a one-time infusion into a vein. Now that you and your child’s doctor have chosen ZOLGENSMA, there are a few steps that need to be taken to determine if your child qualifies for ZOLGENSMA.


Lab tests that need to be completed

Checkbox

Confirm a diagnosis of SMA through a genetic test

Before ZOLGENSMA can be given, a diagnosis of SMA has to be confirmed. If needed, your doctor will order a genetic test to confirm the SMN1 gene is missing or nonworking.

Checkbox

Your doctor should perform an AAV9 antibody test

An adeno-associated virus 9 (AAV9) antibody test measures the amount of anti-AAV9 antibodies in your child’s blood. If your child’s immune system has built up a certain level of anti-AAV9 antibodies, he or she may not qualify for ZOLGENSMA right away. If your child’s anti-AAV9 antibodies are too high, your doctor has the option to retest. Speak with your doctor about the AAV9 antibody test.

Checkbox

Your doctor should perform blood tests to check your child's liver function and obtain creatinine, complete blood count (including hemoglobin and platelet count), and troponin-I.

Your doctor should perform blood tests to check your child’s liver function and to establish baseline levels for other markers in the blood. These measurements before dosing will help your doctor and care team monitor your child’s levels after dosing. These tests may happen while waiting for approval or as you get closer to treatment. Your doctor will give you a prescription for an oral corticosteroid to start the day before your child’s infusion.


Forms that need to be submitted

Have your doctor submit a ZOLGENSMA Prescription Form and a Patient Consent Form

While waiting for test results, your doctor may submit a ZOLGENSMA Prescription Form and/or a Patient Consent Form to the OneGene Program. This starts the process to assess insurance coverage and authorization requirements and helps you learn about the patient support the OneGene Program offers. You will need to sign the Patient Consent Form to access all of the support provided by the OneGene Program.

Once the Prescription Form and/or Patient Consent Form is completed, a representative from the OneGene Program will call you to discuss the patient support available to you.


Connect with the OneGene Program

The OneGene Program, a team of highly trained and dedicated people, is a resource brought to you by Novartis Gene Therapies. It is designed to support and guide you and your family at every step during treatment. After the ZOLGENSMA Prescription Form and/or Patient Consent Form is received, a representative from the OneGene Program will call you to discuss the variety of patient resources that are available to support your family.

Learn how the OneGene Program can help your family when you call 855-441-GENE (4363), Monday-Friday (8 AM-8 PM ET).


Meet the team helping you

Some of the resources the OneGene Program provides:

A Patient Resource Manager icon

A Patient Resource Manager is your main point of contact on behalf of Novartis Gene Therapies, providing expert guidance and answering questions about ZOLGENSMA as your child goes through the treatment journey.

A Case Coordinator icon

A Case Coordinator acts as a go-between to connect you to your Patient Resource Manager and assists doctors with insurance processes. Other support includes verification of insurance benefits and coordination of financial assistance programs for eligible patients.

Your doctor’s office will follow up with you about the status of your insurance coverage approval for ZOLGENSMA.


Prepare for treatment day

If your child is approved for treatment with ZOLGENSMA, your doctor and care team will help ensure that you know exactly what to expect on the day of treatment and how to prepare. Additionally, your Patient Resource Manager will work with you to understand how he or she can best support your family on treatment day.


Get blood tests completed

Blood drop icon

If not completed already, your doctor should perform blood tests to check your child’s liver function and to establish baseline levels for other markers in the blood. These measurements before dosing will help your doctor and care team monitor your child’s levels after dosing. Your doctor will give you a prescription for an oral corticosteroid to start the day before your child’s infusion.


Watch for viral respiratory infection

Temperature icon

Viral respiratory infection before or after ZOLGENSMA infusion can lead to more serious complications. Contact your doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.


Start oral corticosteroid

Calendar icon

A course of an oral corticosteroid* should be started the day before infusion with ZOLGENSMA. This helps manage elevated liver enzyme reactions to ZOLGENSMA by the body’s immune system.

*The specific treatment course for each patient will be determined by the treating doctor. The treatment course is based on several clinical factors and the judgment of the doctor. Caregivers should discuss specific treatment recommendations with their treating doctor.


Confirm treatment date

Doctor icon

Confirm your child’s infusion date, time, and location with your doctor. Determine your family’s transportation plan and get directions to the treatment center, including the address and available parking areas if you’re driving. Ask the treatment center how many family members can be with you and your child on the day of infusion.


If you have any additional questions prior to treatment day, you can reach out to your doctor and care team or your Patient Resource Manager.

Treatment day

On the day of treatment, your child will be infused with ZOLGENSMA

You should give your child the second dose of the oral corticosteroid on the day of infusion as prescribed to help manage reactions to ZOLGENSMA by the body’s immune system. The actual infusion will take 60 minutes. However, ask your doctor or care team for additional details on the schedule for the day. Remember to talk to your doctor and care team about any family members you would like to have with you on treatment day. If possible, you may have the option of having your Patient Resource Manager present on the day of treatment to support your family.


Planning for post-treatment care

Post-treatment plan

Before you leave the hospital, talk with your doctor about post-treatment follow-up and additional monitoring. You will continue to give your child the corticosteroid as prescribed by your doctor. Your doctor will monitor your child’s liver function for at least 3 months after infusion through weekly clinical exams and blood tests for the first month and every other week for the second and third months. Your doctor will determine when to gradually reduce the dose of the corticosteroid and when to stop it.

  • You should contact your doctor immediately if a dose of the corticosteroid is missed or vomited up
  • Talk to your doctor about any side effects

In addition to liver function, your doctor will monitor other markers through blood tests, which may require weekly appointments:

  • Weekly platelet counts for the first month and then every other week for the second and third months until platelet counts return to baseline
  • Weekly troponin-I for the first month and then monthly for the second and third months until troponin-I level returns to baseline

After treatment

After treatment with ZOLGENSMA

Once your child has been treated with ZOLGENSMA, it is important to learn how to continue managing your child’s SMA. This includes speaking with a neuromuscular specialist, creating an extended healthcare team, and identifying additional therapies that may be needed.


Support and care after treatment

Support that goes further

Your support continues after treatment with ZOLGENSMA. Next steps will include:

  • Following up with your doctor to evaluate your child’s progress
  • Speaking with your neuromuscular specialist to review services (like physical and occupational therapy, working with a nutritionist, and meeting with a pulmonologist) to determine what kind of supportive care may be best for your child following treatment
  • Sharing information about SMA and ZOLGENSMA with your pediatrician

Continuing your child’s SMA care

While ZOLGENSMA replaces the function of your child’s missing or nonworking SMN1 gene, your child still has SMA. That’s why it is important to work with your doctor and care team to review supportive care needs. Your child may continue to show signs and symptoms of SMA now or in the future. These may include difficulty swallowing or breathing or muscle weakness. Call your doctor if you see these or any other signs or symptoms. Additional therapies, accommodations, and support may be needed to help manage your child’s SMA and guide his or her ongoing development.

The OneGene Program will also reach out periodically after treatment to offer support and answer questions.

Learn more about SMA after ZOLGENSMA

Download A Caregiver’s Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi)

A Caregiver’s Guide to ZOLGENSMA

What causes SMA? How does ZOLGENSMA work in the body? What are the results of the clinical studies? How do you start treatment with ZOLGENSMA? Get answers to these questions and many more with this in-depth guide created just for caregivers.

Download the guide

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
  • Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

  • Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

What do I need to know about vaccinations and ZOLGENSMA?

  • Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
  • Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?

Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?

The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 833-828-3947.

Please see the Full Prescribing Information.