Onasemnogene Abeparvovec

Reviewed on 5/17/2022

What Is Onasemnogene Abeparvovec and How Does It Work?

Onasemnogene Abeparvovec is a prescription medication used for the treatment of spinal muscular atrophy in children aged above 2 years.

  • Onasemnogene Abeparvovec is available under the following different brand names: Zolgensma, onasemnogene Abeparvovec-xioi

What Are Dosages of Onasemnogene Abeparvovec?

Pediatric dosage

Suspension for IV infusion

  • Provided in a kit containing 2 to 9 vials, as a combination of 2 vial fill volumes (either 5.5 mL or 8.3 mL)
  • All vials have a nominal concentration of 2 x 10^13 vector genomes (VG) per mL
  • Each vial contains an extractable volume of not less than either 5.5 mL or 8.3 mL

Spinal Muscular Atrophy Type 1

Pediatric dosage

  • Children below 2 years: Safety and efficacy not established
  • Children above 2 years
  • Administered as a one-time, single IV infusion through a venous catheter
  • 1.1 x 1014 vector genomes per kilogram (VG/kg) of body weight

Dosage Considerations – Should be Given as Follows: 

  • See “Dosages”

What Are Side Effects Associated with Using Onasemnogene Abeparvovec?

Common side effects of the Onasemnogene Abeparvovec include:

  • elevated aminotransferases and
  • vomiting.

Serious side effects of the Onasemnogene Abeparvovec include:

  • hives,
  • difficulty breathing,
  • swelling of the face, lips, tongue, or throat,
  • jaundice (yellowing of the skin or eyes),
  • easy bruising,
  • unusual bleeding, or
  • purple or red spots under the skin.

Rare side effects of the Onasemnogene Abeparvovec include:

  • none 

This is not a complete list of side effects and other serious side effects or health problems that may occur as a result of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

What Other Drugs Interact with Onasemnogene Abeparvovec?

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

  • Onasemnogene Abeparvovec has severe interactions with the following drugs:
    • adenovirus types 4 and 7 live, oral
    • BCG vaccine live
    • cholera vaccine
    • dengue vaccine
    • influenza virus vaccine quadrivalent, intranasal
    • measles (rubeola) vaccine
    • measles mumps and rubella vaccine, live
    • measles, mumps, rubella, and varicella vaccine, live
    • rotavirus oral vaccine, live
    • rubella vaccine
    • smallpox (vaccinia) vaccine, live
    • typhoid polysaccharide vaccine
    • typhoid vaccine live
    • varicella virus vaccine live
    • yellow fever vaccine
    • zoster vaccine live
  • Onasemnogene Abeparvovec has serious interactions with no other drugs.
  • Onasemnogene Abeparvovec has moderate interactions with at least 31 other drugs.
  • Onasemnogene Abeparvovec has minor interactions with no other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your health care professional or doctor for additional medical advice, or if you have health questions or concerns.

What Are Warnings and Precautions for Onasemnogene Abeparvovec?


  • None

Effects of drug abuse

  • None

Short-Term Effects

  • See “What Are Side Effects Associated with Using Onasemnogene Abeparvovec?”

Long-Term Effects

  • See “What Are Side Effects Associated with Using Onasemnogene Abeparvovec?”


  • Transient decreases in platelet count, some meeting criteria for thrombocytopenia, observed after infusion; monitor platelet counts before infusion and regularly afterward (weekly for the first month; once in 2 weeks for next 2 months)
  • Increases in cardiac troponin-I levels observed after infusion; clinical importance of these findings is unknown; cardiac toxicity observed in animal studies; monitor troponin-I before infusion and then for at least 3 months afterward (weekly for the first month; once in 2 weeks for the next 2 months); consider consulting a cardiologist if troponin elevations are accompanied by clinical signs or symptoms (e.g., heart rate changes, cyanosis, tachypnea, respiratory distress)
  • Acute liver injury, acute liver failure, or elevated AST/ALT
    • Acute serious liver injury, acute liver failure, and elevated aminotransferases can occur
    • Hepatotoxicity (which may be immune-mediated), generally manifested as elevated ALT/AST levels and at times as acute serious liver injury or acute liver failure, has been reported
    • Immune-mediated hepatotoxicity may require adjustment of the corticosteroid treatment regimen, including longer duration, increased dose, or prolongation of the corticosteroid taper
    • Patients with preexisting liver impairment or acute hepatic viral infection may be at higher risk of acute serious liver injury/acute liver failure
    • Patients with ALT/AST, or TB levels (except due to neonatal jaundice) more than two times the ULN have not been studied in clinical trials
    • If acute serious liver injury or acute liver failure is suspected, consult a pediatric gastroenterologist or hepatologist
  • Thrombotic microangiopathy
    • Thrombotic microangiopathy (TMA) was reported for approximately 1 week after infusion, characterized by thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney injury; concurrent immune system activation (eg, infections, vaccinations) was identified in some cases
    • Monitor platelet counts and signs/symptoms of TMA (e.g., hypertension, bruising, seizures, decreased urine output)
    • If these signs and symptoms occur in the presence of thrombocytopenia, further diagnostic evaluation for hemolytic anemia and renal dysfunction is recommended
    • If clinical signs, symptoms, and/or laboratory findings consistent with TMA occur, consult a pediatric hematologist and/or pediatric nephrologist immediately to manage TMA as clinically indicated
  • Drug interaction overview
    • Where feasible, adjust vaccination schedule to accommodate concomitant corticosteroid administration before and following onasemnogene Abeparvovec infusion
    • Certain vaccines (e.g., MMR, varicella) are contraindicated for patients on a substantially immunosuppressive steroid dose (i.e., above 2 weeks of prednisone 2 mg/kg/day or 20 mg/day or equivalent)
    • Seasonal RSV prophylaxis is not precluded
    • Refer to CDC vaccination guidelines for immunosuppressed patients

Pregnancy and Lactation

  • There are no data regarding use in pregnant women
  • No animal reproductive studies conducted
  • Lactation
    • Unknown if secreted in human milk

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