INDICATIONS AND USAGE
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:
Please click here for Full Prescribing Information.
DOSAGE AND ADMINISTRATION
ZOLGENSMA is for single-dose intravenous infusion only.
For patients with unremarkable findings, taper the corticosteroid dose over the next 28 days. Do not stop systemic corticosteroids abruptly. If liver function abnormalities persist, continue systemic corticosteroids (equivalent to oral prednisolone at 1 mg/kg/day) until findings become unremarkable, and then gradually taper the corticosteroid dose over the next 28 days. If liver function abnormalities continue to persist, consult a pediatric gastroenterologist or hepatologist.
DOSAGE FORMS AND STRENGTHS
ZOLGENSMA is a suspension for intravenous infusion, supplied as single-use vials.
ZOLGENSMA is provided in a kit containing 2 to 9 vials, as a combination of 2 vial fill volumes (either 5.5 mL or 8.3 mL). All vials have a nominal concentration of 2.0 × 1013 vector genomes (vg) per mL. Each vial of ZOLGENSMA contains an extractable volume of not less than either 5.5 mL or 8.3 mL.
Please click here for Full Prescribing Information.
WARNINGS AND PRECAUTIONS
Please click here for Full Prescribing Information.
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥ 5%) were elevated aminotransferases and vomiting.
To report SUSPECTED ADVERSE REACTIONS, contact Novartis Gene Therapies at 1-833-828-3947 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please click here for Full Prescribing Information.
DRUG INTERACTIONS
Where feasible, adjust a patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ZOLGENSMA infusion.
Please click here for Full Prescribing Information.
USE IN SPECIFIC POPULATIONS
Pediatric Use: Use of ZOLGENSMA in premature neonates before reaching full term gestational age is not recommended because concomitant treatment with corticosteroids may adversely affect neurological development. Delay ZOLGENSMA infusion until full-term gestational age is reached.
Please click here for Full Prescribing Information.
© 2022 Novartis Gene Therapies, Inc.
Bannockburn, IL 60015
01/2022 US-ZOL-21-0301 V2
Please click here for Full Prescribing Information.
Indication and Important Safety Information
Indication
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 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.
WARNINGS AND PRECAUTIONS
Thrombocytopenia
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 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
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.
ADVERSE REACTIONS
The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.
Please click here for Full Prescribing Information.