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ZOLGENSMA is an essential,
one-time treatment option

Malachi has 2 copies of SMN2 gene
Treated at ~4 months old

The only one-time gene therapy that stops the progression of SMA

ZOLGENSMA® (onasemnogene abeparvovec-xioi) is the only treatment that addresses the genetic cause of spinal muscular atrophy (SMA) with just one dose, and is a type of medicine called gene therapy.

Children with SMA are born with a missing or nonworking SMN1 gene. In a single infusion, ZOLGENSMA replaces the function of the SMN1 gene, by delivering a new, working SMN gene to the motor neuron cells throughout a child’s body to keep the muscles working as they should. It’s called a gene therapy, because it places a gene in the body’s cells—ZOLGENSMA is engineered to keep continuously working in the body.

Since ZOLGENSMA is delivered by a vector made from a virus called adeno-associated virus 9 (or AAV9), the doctor will need to make sure that the child’s level of AAV9 antibodies falls within a certain range before administering gene therapy. (This type of virus is not known to make people sick.) The child’s antibody level will be determined with a blood test. If levels are too high, ask your doctor to monitor and retest—you may need to wait for levels of this antibody to lower before treatment. As with all decisions concerning your child’s health, talk to your doctor for more information.

More about SMA

SMA is a progressive and rare genetic disease that affects the motor neuron cells in the spinal cord and impacts the muscles used for breathing, eating, crawling, and walking.

ZOLGENSMA is a gene therapy for SMA that replaces the function of the missing or nonworking SMN1 gene with a new, working SMN gene. The new SMN gene helps the body’s cells—particularly the motor neuron cells—to keep producing SMN protein.

What causes SMA?

Missing or nonworking SMN1 gene

SMA is caused by a missing or nonworking survival motor neuron 1 (SMN1) gene. This is the primary gene responsible for producing the survival motor neuron (SMN) protein, which is critical to the health of motor neurons—the nerves that control our muscles. When the SMN1 gene is missing or is not working properly, the body cannot make enough SMN protein. Without enough SMN protein, some motor neuron cells throughout the body lose their ability to function and, as a result, die. When this happens, people with SMA may experience muscle weakness throughout the body, which can affect essential activities, such as walking, eating, and in some cases, breathing.

The role of the SMN2 backup gene

Like many genes, the SMN1 gene has a backup gene, called the survival motor neuron 2, or SMN2 gene. In people with SMA, when the SMN1 gene is missing or not working properly, the SMN2 gene can help produce SMN protein.

For people with SMA, the SMN2 gene is the main source of SMN protein production; however, it makes only about 10% of working SMN protein. ZOLGENSMA replaces the function of the missing or nonworking SMN1 gene.

How ZOLGENSMA works

ZOLGENSMA is a gene therapy that replaces the function of the SMN1 gene by delivering a new working SMN gene to the body’s cells. This allows the cells—particularly motor neuron cells—to produce SMN protein.

ZOLGENSMA is made up of a new, working SMN gene that is placed inside a delivery vehicle called a vector.

The vector that delivers the SMN gene is made from a virus called AAV9. To make the vector, the DNA of the virus is removed so that the new SMN gene can be put inside.

ZOLGENSMA travels throughout the body and delivers the new, working gene to motor neuron cells.

Vector with new, working SMN
gene travels through the body

By replacing the function of the SMN1 gene, ZOLGENSMA enables the production of SMN protein and helps preserve essential muscle function.

Motor neuron cells that would have died without treatment can survive and be maintained, stopping the progression of SMA.

After a single infusion, ZOLGENSMA replaces the function of the SMN1 gene to help the body produce SMN protein.

See how ZOLGENSMA works

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Important Safety Information

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

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
  • 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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

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

  • Infections before or after ZOLGENSMA infusion can lead to more serious complications. Caregivers and close contacts with the patient should follow infection prevention procedures. Contact the patient's doctor immediately if the patient experiences any signs of a possible 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 generally occur within the first two weeks after ZOLGENSMA infusion. 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.
  • There is a theoretical risk of tumor development with gene therapies such as ZOLGENSMA. Contact the patient’s doctor and Novartis Gene Therapies, Inc. (1-833-828-3947) if a tumor develops.

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 influenza and respiratory syncytial virus (RSV) is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration. Please consult the patient's doctor.

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 patient body waste for one 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 1-833-828-3947.

Please see the FullPrescribing Information.

Tap or scroll down to read full Important Safety Information and IndicationArrow icon

Important Safety Information

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

  • ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
  • 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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

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

  • Infections before or after ZOLGENSMA infusion can lead to more serious complications. Caregivers and close contacts with the patient should follow infection prevention procedures. Contact the patient’s doctor immediately if the patient experiences any signs of a possible 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 generally occur within the first two weeks after ZOLGENSMA infusion. 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.
  • There is a theoretical risk of tumor development with gene therapies such as ZOLGENSMA. Contact the patient’s doctor and Novartis Gene Therapies, Inc. (1-833-828-3947) if a tumor develops.

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 influenza and respiratory syncytial virus (RSV) is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration. Please consult the patient’s doctor.

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 patient body waste for one 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 1-833-828-3947.

Please see the Full Prescribing Information.

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The information provided in this site is intended only for audiences of the United States. This information does not take the place of talking to your healthcare professional about medical conditions and treatments. If you have questions about ZOLGENSMA after reading this information, ask your healthcare professional.

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