Onasemnogene abeparvovec-xioi  (Zolgensma®)

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.

 

Patient Weight Range (kg)

Dose Volume (mL)

Zolgensma Kit Configuration

 

NDC Number

5.5 mL vial

8.3 mL vial

Total Vials per Kit

2.6 to 3.0 16.5 0 2 2 71894-120-02
3.1 to 3.5 19.3 2 1 3 71894-121-03
3.6 to 4.0 22.0 1 2 3 71894-122-03
4.1 to 4.5 24.8 0 3 3 71894-123-03
4.6 to 5.0 27.5 2 2 4 71894-124-04
5.1 to 5.5 30.3 1 3 4 71894-125-04
5.6 to 6.0 33.0 0 4 4 71894-126-04
6.1 to 6.5 35.8 2 3 5 71894-127-05
6.6 to 7.0 38.5 1 4 5 71894-128-05
7.1 to 7.5 41.3 0 5 5 71894-129-05
7.6 to 8.0 44.0 2 4 6 71894-130-06
8.1 to 8.5 46.8 1 5 6 71894-131-06
8.6 to 9.0 49.5 0 6 6 71894-132-06
9.1 to 9.5 52.3 2 5 7 71894-133-07
9.6 to 10.0 55.0 1 6 7 71894-134-07
10.1 to 10.5 57.8 0 7 7 71894-135-07
10.6 to 11.0 60.5 2 6 8 71894-136-08
11.1 to 11.5 63.3 1 7 8 71894-137-08
11.6 to 12.0 66.0 0 8 8 71894-138-08
12.1 to 12.5 68.8 2 7 9 71894-139-09
12.6 to 13.0 71.5 1 8 9 71894-140-09
13.1 to 13.5 74.3 0 9 9 71894-141-09
> or = 13.6 kg Refer to the medical director for approval of specific NDCs
  •  Total dose does not exceed 1.1 x 1014 vector genomes (vg) per kg
Approval:
  • One time infusion per lifetime

Last review date: July 16, 2019

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