Zolgensma® is an FDA approved treatment for spinal muscular atrophy (SMA). It is a type of treatment referred to as gene therapy or gene replacement therapy.

About Zolgensma®

  • Zolgensma® (onasemnogene abeparvovec-xioi), marketed by Novartis Gene Therapies, is FDA-approved for patients with all forms and types of SMA who are under two years of age at the time of dosing
  • Zolgensma® is given through an intravenous (IV) infusion that takes one hour. It is a one-time treatment
  • Zolgensma® is an SMN-enhancing therapy that works by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of an SMN gene. A virus, AAV9, carries the replacement gene into the body. This virus delivers the new DNA to the cells
  • As with all treatments, timing is critical. Infants dosed with Zolgensma® as soon after diagnosis as possible had better results than those who waited to begin treatment
  • Sites across the U.S. are dosing individuals with Zolgensma®. A number of these sites can be found on our map locator tool
  • To find insurance policies and payer information for Zolgensma®, please click here
  • Check out Novartis’ caregiver brochure to learn about important information for caregivers of Zolgensma® patients
  • For the most up-to-date prescribing information, see here




Single stranded SMN1 DNA via adeno-associated virus (AAV9) vector
Mechanism SMN1 functional replacement with SMN1 DNA episome and own promotor
Approved age Less than two years old
Dose 1.1 × 10^14 vector genomes per kilogram (vg/kg) of body weight
How given Slow intravenous infusion over 60 minutes
How often One time only
Body distribution Blood stream – systemic
Boxed warning Cases of sudden liver failure with fatal outcomes have been reported Acute serious liver injury, and elevated liver aminotransferase (enzyme) levels can occur with Zolgensma®. Patients with preexisting liver impairment may be at higher risk for complications. Prior to receiving Zolgensma®, liver function must be assessed by examination and laboratory testing of all patients. Corticosteroids must be given to all patients before and after Zolgensma® infusion. Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated.
Warnings and precautions If any of the following signs occur after Zolgensma® infusion, consult your healthcare provider immediately: fever, lethargy, decreased feeding, decreased urination, seizures, or easy bruising or bleeding.

1. Acute serious liver injury, acute liver failure, and elevated liver enzyme levels can occur. Acute serious liver injury and acute liver failure, including fatal cases, have been reported with Zolgensma® use. These effects are mitigated by administering systemic corticosteroids to all patients prior to giving Zolgensma® and continued after Zolgensma® dose as indicated below. Patients with preexisting liver impairment or acute hepatic (liver) viral infection may be at higher risk for serious liver injury/liver failure.

2. Systemic Immune response may occur if Zolgensma® is given to a patient with an active infection. Avoid giving Zolgensma® . when an infection of any kind is suspected.

3. Low platelet count or thrombocytopenia may be transient and was typically seen in the first 2 weeks after Zolgensma®. Platelets prevent bleeding.

4. Thrombotic microangiopathy (TMA) has been reported to occur within the first two weeks after Zolgensma® infusion. TMA is associated with low platelet count, microangiopathic hemolytic anemia (bleeding) and kidney injury.

5. Elevated Troponin-I has been observed and should be monitored for at least three months after dose.

Adverse reactions Elevated liver enzymes in the blood and vomiting
Interactions Adjust vaccination schedule to accommodate concomitant corticosteroid administration.
Monitoring Prior to Zolgensma® infusion:

  • Assess for illness, and do not give Zolgensma® if concurrent infection is suspected until the infection has resolved
  • Obtain Anti-AAV9 antibodies testing
  • Assess liver function (exam and blood testing for liver enzymes, albumin, clotting
  • Obtain blood for creatinine, complete blood count, and troponin-I


After Zolgensma® infusion:

  • Monitor blood tests for three months after dose as follows:
  • Liver function weekly for the first month and during the corticosteroid taper period, then every other week for another month and as indicated
  • Platelet count weekly for the first month, then every other week for the second and third months until returns to baseline
  • Troponin-I level weekly for the first month, then monthly for the second and third months until returns to baseline
Associated treatment
  • One day prior to Zolgensma® infusion, begin prednisolone by mouth or feeding tube one mg/kg per day, or equivalent, for 30 days while monitoring as above, followed by tapering dose as clinically indicated. Avoid others who are ill while on corticosteroid treatment. If you see signs of possible infection, contact your healthcare provider immediately
  • RSV prophylaxis treatment is recommended. Temporary viral shedding after Zolgensma® occurs primarily through stooling. For one month after Zolgensma® dose, wash hands well after changing diaper or after contact with stool. Seal disposable diapers in disposable trash bags and then discard into regular trash
Prescribing information See here

Learn more about gene replacement therapy:

History of Zolgensma®

Beginning in 2010, Cure SMA made a series of grants to Nationwide Children’s Hospital to study gene therapy. The adeno-associated virus (AAV9) vector with a single stranded SMN1 DNA was determined to be an effective way to transport the SMN1 gene into cells where the DNA forms an independent episome with its own promotor and makes SMN protein.

Following extensive preclinical investigation, Zolgensma® was evaluated in two clinical trials of symptomatic infantile onset SMA. The first clinical trial (START) was a single center, open label with dose escalation after the first three patients received low dose. An additional 12 patients received a high dose. Outcomes demonstrated improved motor function and improved event free survival in the high dose cohort. A multicenter open-label clinical trial verified the efficacy of Zolgensma®. Zolgensma® was FDA approved for use in infants less than two years old on May 24, 2019.

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