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The following article is content provided by our sponsor Novartis Gene Therapies, Inc. The views and opinions expressed in the content below are not the views and opinions of HCP SMA News Today or its parent company, BioNews Inc.


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 The mechanism of action for ZOLGENSMA targets the genetic root cause of SMA—deletions or mutations in the SMN1 gene—with a one time-dose, and is designed to replace the function of the SMN1 gene with a fully functional copy of a human SMN gene.1 ZOLGENSMA is also designed to enable rapid and continuous expression of functional SMN protein.1,2

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Indication and Important Safety Information


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 (SMN1) gene.

Limitations of Use

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

Important Safety Information

BOXED WARNING: Serious Liver Injury and Acute Liver Failure

Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury, acute liver failure, and elevated aminotransferases can also 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. Administer systemic corticosteroid 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.  If acute serious liver injury or acute liver failure is suspected, promptly consult a pediatric gastroenterologist or hepatologist.


Systemic Immune Response

Patients with underlying active infection, either acute or chronic uncontrolled, could be at an increased risk of serious systemic immune response.  Administer ZOLGENSMA to patients who are clinically stable in their overall health status (e.g., hydration and nutritional status, absence of infection). Postpone ZOLGENSMA in patients with infections until the infection has resolved and the patient is clinically stable.


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 infusion and on a regular basis for at least 3 months afterwards.

Thrombotic Microangiopathy

Cases of thrombotic microangiopathy (TMA) were reported to occur generally within the first two weeks after ZOLGENSMA infusion.  TMA can result in life-threatening or fatal outcomes.  Obtain baseline creatinine and complete blood count before ZOLGENSMA infusion.  Following infusion, monitor platelet counts closely as well as other signs and symptoms of TMA.  Consult a pediatric hematologist and/or pediatric nephrologist immediately to manage as 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. Consider consultation with a cardiologist if troponin elevations are accompanied by clinical signs or symptoms.


The most commonly 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; 2023.
  2. Mendell JR, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Eng J Med. 2017;377(18):1713-1722.

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Bannockburn, IL 60015                                                                       03/2023 US-ZOL-22-0081  V3

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