Onasemnogene Abeparvovec Side Effects

Medically reviewed by Drugs.com. Last updated on Dec 26, 2024.

Applies to onasemnogene abeparvovec: intravenous suspension.

Precautions

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.

This medicine may increase your risk of a tumor. Talk with your doctor if you have concerns about this.

Serious side effects

Along with its needed effects, onasemnogene abeparvovec 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 while taking onasemnogene abeparvovec:

For healthcare professionals

Applies to onasemnogene abeparvovec: intravenous kit.

General adverse events

The most commonly reported adverse reactions have included elevated aminotransferases and vomiting.^[Ref]

Hematologic

• Common (1% to 10%): Thrombocytopenia
• Frequency not reported: Transient decreases in platelet count
• Postmarketing reports: Thrombotic microangiopathy

During clinical trials, transient decreases in platelet counts, some meeting the criteria for thrombocytopenia, have been reported.

Respiratory

• Common (1% to 10%): Respiratory insufficiency^[Ref]

One patient presented with respiratory insufficiency 12 days after receiving this drug. The patient was found to have respiratory syncytial virus (RSV) and parainfluenza in respiratory secretions; episodes of hypotension were followed by seizures. Approximately 30 days following the infusion, leukoencephalopathy was found and the patient died after withdrawal of life support, 52 days after this drug was infused.^[Ref]

Cardiovascular

• Common (1% to 10%): Elevated troponin-I levels^[Ref]

Elevated cardiac troponin-I levels up to 0.176 mcg/L have been observed following infusion. The clinical importance of this finding is unknown; however, cardiac toxicity has been observed in animal studies.^[Ref]

Gastrointestinal

• Common (1% to 10%): Vomiting

Hepatic

• Very common (10% or more): Elevated aminotransferases (27.3%)^[Ref]

Elevated aminotransferases (ALT and/or AST) to greater than the upper limit of normal (ULN) occurred in 12 of 44 patients in clinical trials. Two patients had AST and ALT elevations of up to 48 x ULN; these patients were otherwise asymptomatic with normal total bilirubin. Following corticosteroid management, these abnormalities resolved. One patient with preexisting transaminase elevations (who received this drug with the recommended corticosteroid regimen) became jaundiced about 7 weeks after the infusion. Laboratory testing showed an AST level approximately 80 x ULN and ALT level approximately 45 x ULN. Liver biopsy showed acute massive degeneration of hepatocytes, and massive mixed inflammatory infiltrate (primarily CD8-positive T-lymphocytes). Following treatment with corticosteroids, the patient recovered to baseline.^[Ref]

Other

• Common (1% to 10%): Pyrexia

See also:

Frequently asked questions

• Is Zolgensma a cure for SMA?

Further information

Onasemnogene abeparvovec side effects can vary depending on the individual. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Note: Medication side effects may be underreported. If you are experiencing side effects that are not listed, submit a report to the FDA by following this guide.

Medical Disclaimer