The Steroid Question Everyone Has After Novartis
With Novartis’ Itvisma bringing gene replacement therapy to adults and children age 2 and older with Spinal Muscular Atrophy, one important question is gaining attention: why are steroids required with the treatment? Itvisma, also known as onasemnogene abeparvovec-brve, uses an AAV vector to deliver functional genetic instructions for producing survival motor neuron, or SMN, protein. Because the body can mount an immune and inflammatory response after receiving an AAV-based gene therapy, the FDA requires systemic corticosteroids around Itvisma treatment to help reduce the risk of serious liver injury. The FDA-approved prescribing information carries a boxed warning for acute serious liver injury and instructs healthcare professionals to evaluate liver function before treatment and continue monitoring it for at least three months afterward.
For Itvisma, corticosteroid treatment begins 24 hours before the one-time intrathecal injection. The FDA-approved regimen calls for a systemic corticosteroid equivalent to oral prednisolone at 1 mg/kg per day for 30 days, including the day Itvisma is administered. If liver examinations and laboratory results are satisfactory after those 30 days, the steroid is gradually tapered over the following 28 days rather than stopped suddenly. If liver abnormalities remain, corticosteroid treatment may need to continue longer before the taper begins, with the treatment team adjusting the plan when necessary. The steroids are therefore not being used to treat SMA muscle weakness itself; they are an important safety measure surrounding the immune and liver response associated with gene therapy.
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