ZOLGENSMA® (onasemnogene abeparvovec-xioi) by Novartis



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Indications & Usage

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:

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

 

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Dosage & Administration

DOSAGE AND ADMINISTRATION

 

ZOLGENSMA is for single-dose intravenous infusion only.

 

  • The recommended dosage of ZOLGENSMA is 1.1 × 1014 vector genomes (vg) per kg of body weight.
  • Administer ZOLGENSMA as an intravenous infusion over 60 minutes.
  • Starting one day prior to ZOLGENSMA infusion, administer systemic corticosteroids equivalent to oral prednisolone at 1 mg/kg of body weight per day for a total of 30 days.
  • At the end of the 30-day period of systemic corticosteroid treatment, check liver function by clinical examination and by laboratory testing.

 

For patients with unremarkable findings, taper the corticosteroid dose over the next 28 days. If liver function abnormalities persist, continue systemic corticosteroids (equivalent to oral prednisolone at 1 mg/kg/day) until findings become unremarkable, and then taper the corticosteroid dose over the next 28 days. Consult expert(s) if patients do not respond adequately to the equivalent of 1 mg/kg/day oral prednisolone.

 

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.

 

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Contraindications

CONTRAINDICATIONS

 

None.

 

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Warnings & Precautions

WARNINGS AND PRECAUTIONS

 

  • Thrombocytopenia: Monitor platelet counts before ZOLGENSMA infusion, and weekly for the first month and then every other week for the second and third month until platelet counts return to baseline.
  • Thrombotic Microangiopathy (TMA): If clinical signs, symptoms and/or laboratory findings occur, consult a pediatric hematologist and/or pediatric nephrologist immediately to manage as clinically indicated.
  • Elevated Troponin-I: Monitor troponin-I before ZOLGENSMA infusion, and weekly for the first month and then monthly for the second and third month until troponin-I level returns to baseline.

 

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Adverse Reactions

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.

 

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Drug Interactions

DRUG INTERACTIONS

 

Where feasible, adjust a patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ZOLGENSMA infusion.

 

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Use in Specific Populations

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.

 

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© 2021 Novartis Gene Therapies, Inc.
Bannockburn, IL 60015

10/2021 US-ZOL-21-0301

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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

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.



WARNINGS AND PRECAUTIONS

Thrombocytopenia
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.

 

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.