Itvisma®

Itvisma® 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.

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About Itvisma®

  • Itvisma® (onasemnogene abeparvovec-brve), marketed by Novartis, is FDA-approved for patients with all forms and types of SMA who are two years of age or older at the time of dosing.
  • Itvisma® is given through an intrathecal bolus injection over approximately 1 to 2 minutes.
  • Itvisma® 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, AAV, carries the replacement gene into the body. This virus delivers the new DNA to the cells.
  • For Cure SMA's outreach to public and private insurers regarding Itvisma® coverage, click here.
  • Learn more about Cure SMA's insurance resources available to you here.
  • Learn more about the Itvisma® clinical trials here and here.
  • As sites across the U.S. start dosing individuals with Itvisma® they can be found on our map locator tool.
  • For the most up-to-date prescribing information, see here.

Overview:

Description Single stranded SMN1 DNA via adeno-associated virus (AAV) vector
Mechanism SMN1 functional replacement with SMN1 DNA episome and own promotor
Approved age Two years of age or older
Dose 1.2 × 10^14 vector genomes (vg)
How given Intrathecal bolus injection over 1 to 2 minutes
How often One time only
Body distribution Cerebral spinal fluid (Central only)
Boxed warning The most common adverse reactions that occurred in patients treated with Itvisma® were upper respiratory tract infection, fever, upper gastrointestinal symptoms, increased liver enzymes, headache, dizziness, pain in extremity, low platelet counts, and sensory disturbance.
Warnings and precautions If any of the following signs occur after Itvisma® injection, consult your healthcare provider immediately: fever, lethargy, decreased feeding, decreased urination, seizures, or easy bruising or bleeding.

1.) Hepatotoxicity, with elevated ALT and/or AST levels, can occur. Patients with preexisting hepatic impairment or acute hepatic viral infection may be at higher risk of liver injury. In order to mitigate potential aminotransferase elevations, administer systemic corticosteroid before and after Itvisma® injection. Immune-mediated hepatotoxicity may require adjustment of the corticosteroid treatment regimen, including longer duration, increased dose, or prolongation of the corticosteroid taper.

2.) Low platelet counts (thrombocytopenia) can occur. Platelet counts are expected to return to baseline two weeks following Itvisma® injection.

3.) Peripheral sensory neuropathy has occurred with Itvisma® administration. Signs and symptoms may include numbness, tingling, prickling, or pain in the arms, hands, legs and/or feet, with onset seen at approximately three weeks post-injection in clinical studies.  Patients and caregivers should promptly notify their physician if such symptoms occur.

4.) Thrombotic microangiopathy (TMA) may occur with Itvisma® administration. TMA is characterized by thrombocytopenia (low platelet count), microangiopathic hemolytic anemia (bleeding), and acute kidney injury.

5.) Troponin I levels may increase without clinical symptoms. Consider cardiac evaluation and consult a cardiologist if needed.

6.) There is a theoretical risk of tumorigenicity due to integration of AAV vector DNA into the genome.  Random integration of recombinant AAV-vector DNA into human DNA has been reported with AAV gene therapies like Itvisma®. Long-term studies in animals show rare, clonal integration events can occur, especially in the liver, raising theoretical concerns for high-dose treatments. Long-term studies assessing cancer risk in humans are limited.
Adverse reactions Upper respiratory tract infection, upper gastrointestinal symptoms, pyrexia (fever), and headache. 
Interactions Adjust vaccination schedule to accommodate concomitant corticosteroid administration.
Monitoring Prior to Itvisma® injection:

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

 

After Itvisma® injection:

  • Monitor liver function (AST, ALT, total bilirubin) weekly for the month after Itvisma® injection and during the corticosteroid taper period (over the next 28 days or longer if needed).  
  • If the patient is clinically stable with unremarkable findings (normal clinical exam, total bilirubin, and ALT and AST levels below 2 × ULN) at the end of the corticosteroid taper period, continue to monitor liver function every other week for another month.  
  • Monitor platelet counts weekly for the first month and as clinically indicated until platelet counts return to baseline. 
Associated treatment One day prior to Itvisma® 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. If you see signs of possible infection, contact your healthcare provider immediately
Prescribing information See here

Learn more about gene replacement therapy:

History of Itvisma®

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.

Itvisma® builds upon the progress of Zolgensma® in that it is the same gene therapy product. Phase III clinical trials, the STEER study and the STRENGTH study, tested the therapy in older patients via intrathecal injection.

Itvisma® (onasemnogene abeparvovec-brve), a gene therapy for spinal muscular atrophy (SMA), was approved by the FDA in November 2025 for individuals with SMA aged 2 and older.

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