ZOLGENSMA (onasemnogene abeparvovec-xioi)

ZOLGENSMA (onasemnogene abeparvovec-xioi)

OFFICE ADMINISTRATION [medical benefit]

Indications for Prior Authorization:

• Indicated in 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

Patients must meet the following criteria for the indication(s) above

• Patient is less than 2 years of age, AND
• Diagnosis of spinal muscular atrophy (SMA) with bi-allelic mutations in the SMN1 gene reported as at least one of the following: homozygous deletion, homozygous mutation, or compound heterozygous mutation (as confirmed by chart note documentation and genetic testing), AND
• Patient has three or fewer survival motor neuron 2 (SMN2) gene copies, AND
• Patient has started or will receive systemic corticosteroids equivalent to oral prednisolone at a dose of 1 mg/kg per day starting 1 day prior to Zolgensma® infusion and for a total of 30 days, AND
• Documentation of baseline laboratory tests demonstrating anti-AAV9 antibody titers are ≤ 1:50, AND
• The following laboratory assessments have been completed, or will be performed, prior to administration of Zolgensma® (i, ii, and iii):
  • Baselines liver function testing (e.g. aspartate aminotransferase, alanine aminotransferase, total bilirubin, prothrombin time), AND
  • Platelet counts, AND
  • Troponin-I levels, AND
• Prescribed by or in consultation with a neurologist and/or physician who specializes in the management of spinal muscular atrophy and/or neuromuscular disorders, AND
• Patient has not received Zolgensma® in the past, AND
• Zolgensma® will not be used in combination with Spinraza®, AND
• Zolgensma® will not be used in premature neonates, AND
• Patient does not have advanced SMA A (e.g., complete paralysis of limbs, permanent ventilator dependence)

Dosing:

• If criteria met, approve one dose (kit) of Zolgensma based on kg weight (documentation required) per the cited NDC in the table below:

Table 1.  Dose of Zolgensma Based on Availability.

•  Total dose does not exceed 1.1 x 10¹⁴ vector genomes (vg) per kg

Approval:

• One time infusion per lifetime