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Novartis Gene Therapies

  • Booth: 506

Novartis Gene Therapies, Inc. (formerly AveXis) is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurologic genetic diseases. Our initial gene therapy for spinal muscular atrophy (SMA) has been approved in more than 40 countries with additional approvals expected this year.

Exhibit intended for US healthcare professionals only. Colorado price disclosure short form is available for Colorado prescribers.

Please note that this booth is not staffed. For any questions, please contact Novartis Gene Therapies medical information.

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: Acute Serious Liver Injury

Acute serious liver injury and elevated aminotransferases can occur with ZOLGENSMA. Patients with pre-existing 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 observed at different time points 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 approximately 1 week after ZOLGENSMA 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 if clinically indicated.

Elevated Troponin-I
Transient 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 commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.

Please click here for Full Prescribing Information.

© 2021 Novartis Gene Therapies, Inc.
Bannockburn, IL 60015
09/2021  US-ZOL-21-0302

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