Onasemnogene Abeparvovec-xioi (By injection)

Onasemnogene Abeparvovec-xioi (on-a-SEM-noe-jeen a-be-PAR-voe-vek - xioi)

Treats spinal muscular atrophy (progressive muscle wasting).

Brand Name(s):


There may be other brand names for this medicine.

When This Medicine Should Not Be Used:

This medicine is not right for everyone. Your child should not receive it if they had an allergic reaction to onasemnogene abeparvovec-xioi.

How to Use This Medicine:


  • Your doctor will prescribe your dose and schedule. This medicine is given through a needle placed in a vein. The medicine must be given slowly, so the needle will have to stay in place for at least 60 minutes.
  • A nurse or other health provider will give you this medicine.
  • Your doctor may give your child other medicines (including 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.

Drugs and Foods to Avoid:

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

  • 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.

Warnings While Using This Medicine:

  • Tell your doctor if your child has liver disease or heart disease.
  • This medicine may cause liver or heart problems.
  • 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.
  • Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments.

Possible Side Effects While Using This Medicine:

Call your doctor right away if you notice any of these side effects:

  • Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
  • Dark urine, pale stools, nausea, vomiting, loss of appetite, stomach pain, yellow skin or eyes
  • Chest pain, fast or irregular heartbeat, swelling of the feet and lower legs, trouble breathing
  • Unusual bleeding or bruising, nosebleeds

If you notice these less serious side effects, talk with your doctor:

  • Pain, itching, burning, swelling, or a lump under your skin where the needle is placed

If you notice other side effects that you think are caused by this medicine, tell your doctor

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

Last Updated: 11/6/2020

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