Description and Brand Names

Información sobre medicamentos proporcionada por: IBM Micromedex

US Brand Name

  1. Zolgensma


Onasemnogene abeparvovec-xioi injection is used to treat spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Spinal muscular atrophy is a rare neuromuscular disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to early death. Onasemnogene abeparvovec-xioi is an adeno-associated virus vector-based gene treatment that works by copying the gene needed for the human SMN protein.

This medicine is to be given only by or under the direct supervision of your doctor.

This product is available in the following dosage forms:

  • Suspension

Before Using

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:


Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.


Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of onasemnogene abeparvovec-xioi injection in newborns to children younger than 2 years of age. However, use is not recommended in premature infants. Safety and efficacy have been established.


No information is available on the relationship of age to the effects of onasemnogene abeparvovec-xioi injection in geriatric patients.


There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Drug Interactions

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other Interactions

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other Medical Problems

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

  • Heart disease or
  • Thrombocytopenia (low platelet count)—Use with caution. May make these conditions worse.
  • Liver disease—Use with caution. The effects may be increased because of the slower removal of the medicine from the body.

Proper Use

A nurse or other trained health professional will give your child this medicine in a medical facility. It is given through a needle placed into one of your child's veins. The medicine must be given slowly, so the needle will have to stay in place for at least 60 minutes.

Your doctor may give your child other medicines (eg, steroids) before and after the infusion to prevent unwanted effects.

Your doctor may also give your child a vaccine to prevent respiratory syncytial virus. Talk to your doctor if you have concerns.

This medicine is a virus vector-based gene treatment and may spread to other people through body waste (feces). Seal diapers in trash bags and throw them into the trash. Wash your hands with soap and water before and after handling your child's body waste. Follow these precautions during treatment with this medicine and for 1 month after the infusion.


It is very important that your doctor check your child's progress closely while receiving the medicine to make sure it is working properly. Blood and urine tests are needed to check for unwanted effects.

Check with your doctor right away if your child has pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, vomiting, or yellow eyes or skin. These could be symptoms of a serious liver problem.

This medicine may cause heart problems. Check with your doctor right away if your child has chest pain or discomfort, fast or irregular heartbeat, swelling of the feet and lower legs, or trouble breathing.

This medicine may cause your child to bruise or bleed more easily. Be extra careful to avoid injuries. Stay away from rough sports or other situations where you could be bruised, cut, or injured. Gently brush and floss your teeth. Be careful when using sharp objects, including razors and fingernail clippers.

Thrombotic microangiopathy (damage in the smallest blood vessels) may occur while you are using this medicine. Tell your doctor right away if your child have a decreased urine output, seizures, severe chest pain, sudden weakness in the arms or legs, or unusual bleeding or bruising.

Talk with your doctor before your child receives any vaccines during treatment with this medicine. The vaccination schedule should be adjusted as needed to fit in with the steroid treatment schedule.

Side Effects

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

  1. Clay-colored stools
  2. dark urine
  3. decreased appetite
  4. fever
  5. headache
  6. itching, skin rash
  7. loss of appetite
  8. nausea
  9. stomach pain or tenderness
  10. swelling of the feet or lower legs
  11. unusual tiredness or weakness
  12. vomiting
  13. yellow eyes or skin

Incidence not known

  1. Back pain
  2. blurred vision
  3. confusion
  4. decreased urine output
  5. dizziness
  6. dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position
  7. drowsiness
  8. seizures
  9. sudden weakness in the arms or legs
  10. sudden, severe chest pain
  11. sweating
  12. unusual bleeding or bruising

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.