Onasemnogene abeparvovec

Onasemnogene abeparvovec is designated as an orphan drug by the US FDA on 9/30/2014, used in treatment of spinal muscular atrophy, manufactured / distributed by AveXis, Inc

How does this medication work?

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.

What are the limitations of use with this medication?

• 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.

How is this medication supplied?

• ZOLGENSMA is for single-dose intravenous infusion only.
• 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.

How should this medication be used?

• 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.

What tests are done to monitor treatment?

• 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.

Who should not use this medication (contraindications)?

None.

How much does this medication cost?

Novartis' Zolgensma, a one-time gene therapy for spinal muscular atrophy tops the list with a price tag of $2.125 million and is one of the most expensive medications for a single dose.

What should be watched monitored during the treatment?

• 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.

What are the side effects of this medication?

• The most common adverse reactions (incidence ≥ 5%) were elevated aminotransferases and vomiting.

What are the drug interactions I should be aware of?

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

Can this medication be used in children?

• 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.

• Dailymed label info on Onasemnogene abeparvovec
• FDA Onasemnogene abeparvovec

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