Dosing and infusion information for a treatment for SMA

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ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a gene therapy for the treatment of patients with spinal muscular atrophy (SMA) who are less than 2 years of age.1 ZOLGENSMA is the first one time only treatment for SMA.1,2 ZOLGENSMA is administered through intravenous (IV) infusion over 60 minutes. Baseline testing for anti-AAV9 antibodies, liver function, creatinine, complete blood count (including hemoglobin and platelet count), and troponin-l is required for every ZOLGENSMA patient. 24 hours prior to ZOLGENSMA infusion, patients begin an oral prednisolone regimen that continues for 30 days. After 30 days, the recommended corticosteroid regimen varies based on the patient’s liver function assessment. Do not stop corticosteroids abruptly. After infusion, lab tests and monitoring will need to be continued on a regular basis over a 3-month period or longer.1 For more details, please see the accompanying Full Prescribing Information.

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ZOLGENSMA, is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMNI) gene.

Limitations of Use

The safety and effectiveness of repeat administration or the use in patients with advanced SIVA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated with ZOLGENSMA.

Important Safety Information

BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure

Acute serious liver injury, acute liver failure, and elevated aminotransferases can occur with ZOLGENSMA, Patients with preexisting liver impairment may be at higher risk. Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing (e.g., hepatic aminotransferases (aspartate aminotransferase (AST) and alanine aminotransferase (ALT)], total bilirubin, and prothrombin time). Administer a systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion.



Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were typically observed within the first two weeks after ZOLGENSMA infusion. Monitor platelet counts before ZOLGENSMA intusion and on a regular basis for at least 3 months afterwards.

Thrombotic Microangiopathy

Cases of thrombotic microangiopathy (TA) were reported approximately 1 week after ZOLGENS MA infusion. Obtain baseline creatinine and complete blood count before ZOLGENSMA infusion. Following infusion, monitor for thrombocytopenia as well as other signs and symptoms of TMA. Consult a pediatric hematologist and/or pediatric nephrologist immediately to manage it clinically indicated.

Elevated Troponin-I

Increases in cardiac troponin-I levels were observed following ZOLGENSMA infusion. Monitor troponin-I before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards.


The most common/v observed adverse reactions (incidence >5%) in clinical studies were elevated aminotransferases and vomiting.

Please see Full Prescribing Information.


  1. ZOLGENSMA [prescribing information]. Bannockburn, IL: Novartis Gene Therapies, Inc; 2022.
  2. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality [press release]. Bethesda, MD: US Food and Drug Administration, May 24, 2019. approves innovative gene therapy treat pediatric patients spinal muscular atrophy rare-disease. Accessed May 5, 2022.

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