Now Approved!

A one-time-only gene therapy for the treatment of children less than 2 years old with spinal muscular atrophy (SMA).

ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA (pronounced zol-JEN-smuh) is given as a one-time infusion into the vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

Find helpful resources for caregivers and healthcare professionals here.

Now Approved!

A one-time-only gene therapy for the treatment of children less than 2 years old with spinal muscular atrophy (SMA).

ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA (pronounced zol-JEN-smuh) is given as a one-time infusion into the vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

Find helpful resources for caregivers and healthcare professionals here.

Support from the OneGene Program™

The OneGene Program is a dedicated resource brought to you by AveXis. This program is specifically designed to support and guide you and your family at every step during your journey with ZOLGENSMA. Contact the OneGene Program at 855-441-GENE (4363), Monday-Friday (8 AM-8 PM ET), to learn more.

ZOLGENSMA is changing SMA treatment with a one-time-only dose

ZOLGENSMA press release

Read the press release about ZOLGENSMA.

Read the press release
ZOLGENSMA letter to the SMA community

Read the letter from AveXis to the SMA community.

Read the letter
Resources for

HEALTHCARE PROFESSIONALS

Call the OneGene Program at 855-441-GENE (4363) to get connected with a ZOLGENSMA representative, Monday-Friday (8 AM-8 PM ET).

Resources for

CAREGIVERS & FAMILIES

Take the next step for your child

If you and your doctor decide that ZOLGENSMA is right for your child, work together to find a treatment center by emailing treatments@curesma.org.

Important Safety Information

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

  • Liver enzymes could become elevated and cause 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?

  • Liver enzymes could become elevated and cause 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 a patient experiences unexpected bleeding or bruising.

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 up to 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 the 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 AveXis at 833-828-3947.

Please see the Full Prescribing Information.