Class Name(s):

• 26:12 Gene Therapy

Pronunciation:

• (pronunciation)

Brand Name(s):

• Zolgensma ^®

IMPORTANT WARNING:

Onasemnogene abeparvovec-xioi injection may cause liver damage. Tell your doctor if your child has had or ever had liver problems, including hepatitis (an infection that causes liver damage). If your child experiences any of the following symptoms, call your doctor immediately: yellowing of the skin or eyes, pain in the upper right part of the stomach, nausea, vomiting, sleepiness, confusion, dark yellow urine, or pale stools.

The doctor will order blood tests to monitor your child's liver before they receive onasemnogene abeparvovec-xioi injection and for at least 3 months afterward. Your doctor will prescribe a corticosteroid (a type of medication used to prevent swelling and inflammation) to take 1 day before receiving treatment and for at least 30 days afterward to help prevent liver damage. Call the doctor if your child misses a dose or vomits after taking a dose.

Talk to your doctor about the risks of receiving onasemnogene abeparvovec-xioi injection.

WHY is this medicine prescribed?

Onasemnogene abeparvovec-xioi injection is used to treat spinal muscular atrophy (an inherited condition that reduces muscle strength and movement) in certain infants and children less than 2 years old. Onasemnogene abeparvovec-xioi is in a class of medications called gene therapy. It works by replacing a defective gene which helps preserve muscle and nerve function.

HOW should this medicine be used?

Onasemnogene abeparvovec-xioi injection comes as a suspension (liquid) to be injected intravenously (into a vein) by a doctor or nurse in a healthcare setting. It is infused over 60 minutes as a single dose.

Onesemnogene abeparvovec-xioi injection may cause serious reactions during and after the infusion. A doctor or nurse will monitor your child during and after the infusion. Tell your doctor or nurse immediately if your child experiences any of the following symptoms: hives, itching, rash, vomiting, fast or racing heartbeat, shortness of breath, or fast breathing.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

Are there OTHER USES for this medication?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

What SPECIAL PRECAUTIONS should I follow?

Before receiving onasemnogene abeparvovec-xioi injection,

• tell your doctor or pharmacist if you are allergic to this medication, any part of this medication, or any other medications, foods or substances. Tell your doctor or pharmacist about the allergy and what symptoms you had.
• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products your child is taking or plans to take.
• tell your doctor if your child has or has ever low platelet counts, or heart or kidney problems.
• if your child is having surgery, including dental surgery, tell the doctor or dentist that they have received onasemnogene abeparvovec-xioi injection.
• check with the doctor to see if your child needs to receive any vaccinations. It is important to have all vaccines appropriate for their age before beginning treatment with onasemnogene abeparvovec-xioi injection. Do not have any vaccinations before or after treatment with onasemnogene abeparvovec-xioi injection without talking to the doctor.
• you should know that onasemnogene abeparvovec-xioi injection contains a virus that could spread and infect other people. Use good hand hygiene and wash your hands if you come in contact with your child's bodily fluids or waste (urine, feces, saliva). Put contaminated materials that contain bodily fluids or waste into a sealed plastic bag and throw away into the garbage for 1 month after the infusion.
• you should know that onasemnogene abeparvovec-xioi injection may cause serious adverse reactions. You should also know that an infection of any type can make these reactions more complicated or severe. Tell the doctor if your child has an infection or if they have or have ever had an infection that keeps coming back. Caregivers and close contacts should follow infection prevention practices, including frequent handwashing. If your child experiences any of the following symptoms during or after treatment, call the doctor immediately: cough, wheezing, runny nose, sore throat, or other signs of infection.

What SPECIAL DIETARY instructions should I follow?

Unless your doctor tells you otherwise, continue your normal diet.

What SIDE EFFECTS can this medicine cause?

Onasemnogene abeparvovec-xioi injection may cause side effects. If you experience any of these symptoms or those listed in the IMPORTANT WARNING or SPECIAL PRECAUTIONS sections, call your doctor immediately or get emergency medical treatment:

• unusual bruising or bleeding
• confusion, tiredness or sleepiness, weakness, dizziness, shortness of breath, unusual bruising or bleeding, seizures, or decreased urination

Onasemnogene abeparvovec-xioi injection may increase the risk of developing cancer. Talk to your doctor about the risks of receiving this medication.

Onasemnogene abeparvovec-xioi injection may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What should I do in case of OVERDOSE?

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at http://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

What OTHER INFORMATION should I know?

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to onasemnogene abeparvovec-xioi injection.

Onasemnogene abeparvovec-xioi injection may only be used in people with a certain genetic makeup. Your doctor will order a blood test to help decide if this medication is right for you.

Before having any laboratory test, tell your doctor and the laboratory personnel that you have received onasemnogene abeparvovec-xioi injection.

Keep a written list of all of the prescription and nonprescription (over-the-counter) medicines, vitamins, minerals, and dietary supplements you are taking. Bring this list with you each time you visit a doctor or if you are admitted to the hospital. You should carry the list with you in case of emergencies.

This report on medications is for your information only, and is not considered individual patient advice. Because of the changing nature of drug information, please consult your physician or pharmacist about specific clinical use.

The American Society of Health-System Pharmacists, Inc. represents that the information provided hereunder was formulated with a reasonable standard of care, and in conformity with professional standards in the field. The American Society of Health-System Pharmacists, Inc. makes no representations or warranties, express or implied, including, but not limited to, any implied warranty of merchantability and/or fitness for a particular purpose, with respect to such information and specifically disclaims all such warranties. Users are advised that decisions regarding drug therapy are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and the information is provided for informational purposes only. The entire monograph for a drug should be reviewed for a thorough understanding of the drug's actions, uses and side effects. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information is not a substitute for medical care.

AHFS^® Patient Medication Information™ Copyright, 2026. The American Society of Health-System Pharmacists^®, 4500 East-West Highway, Suite 900, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.

Selected Revisions: January 15, 2026.