ZOLGENSMA^® (onasemnogene abeparvovec-xioi) by Novartis

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  PHYSICIAN RESOURCES ZOLGENSMA Treatment Guide

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  PHYSICIAN RESOURCES ZOLGENSMA Prescription and Patient Consent Forms

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  PHYSICIAN RESOURCES Early Diagnosis of SMA

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  PATIENT PROFILES Lucy's Patient Story

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  PATIENT PROFILES Brady's Patient Story

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  PATIENT PROFILES Maisie’s Patient Story

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  PATIENT PROFILES Adalyne’s Patient Story

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  PATIENT RESOURCES A Caregiver’s Guide to ZOLGENSMA

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  PATIENT RESOURCES Facts About SMA

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  PATIENT RESOURCES Post Treatment Plan for Caregivers

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 AND ADMINISTRATION

 

ZOLGENSMA is for single-dose intravenous infusion only.

 

• Due to the increased risk of serious systemic immune response, postpone ZOLGENSMA in patients with concurrent infections until resolved. Clinical signs of symptoms of infection should not be evident at the time of administration. 
• 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. 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.

 

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CONTRAINDICATIONS

 

None.

 

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WARNINGS AND PRECAUTIONS

 

• Thrombocytopenia: Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were observed within the first two weeks after ZOLGENSMA infusion. 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

 

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

 

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

 

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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