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Onasemnogene abeparvovec (Zolgensma®)

Commercial Policy
Version Date: January 2023

Product(s):

  • onasemnogene abeparvovec (Zolgensma®) intravenous infusion for administration by a healthcare professional

FDA Approved Use:

  • For treatment of patients less than 2 years of age with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene
    • Limitations of use: Not for repeat administration or for use in advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence)

Criteria for Medical Necessity:

The restricted product(s) may be considered medically necessary when the following criteria are met:

  1. The patient is less than 2 years of age; AND
  2. The patient has a diagnosis of spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene [medical record documentation required]; AND
  3. The diagnosis has been confirmed by genetic testing consisting of one of the following [medical record documentation required]:
    1. Homozygous deletion of SMN1 exon 7; OR
    2. Compound heterozygosity for SMN1 exon 7 deletion and small mutation; AND
  4. The patient has four or fewer copies of the survival motor neuron 2 (SMN2) gene [medical record documentation required]; AND
  5. The patient does not have advanced SMA (e.g., complete paralysis of limbs, or permanent ventilator dependence defined as invasive ventilation [tracheostomy] or at least 16 hours of respiratory assistance per day continuously for at least 14 days in the absence of an acute, reversible illness or a perioperative state) [medical record documentation required]; AND
  6. The patient has had laboratory testing confirming anti-adeno-associated virus serotype 9 (AAV9) antibody titer ≤ 1:50 [medical record documentation required]; AND
  7. The prescriber is a board-certified neurologist or pediatric neurologist who is experienced in the diagnosis and management of SMA and practices in a research academic setting [medical record documentation required]; AND
  8. For members with North Carolina benefits/coverage seeking care within North Carolina, the provider is in the Blue Premier health system network; AND
  9. The patient has not received prior treatment with onasemnogene abeparvovec-xioi (Zolgensma®) or other gene replacement therapy for SMA [medical record documentation required]; AND
  10. The patient will not be treated concurrently with risdiplam (Evrysdi™) and/or nusinersen (Spinraza®), and any existing authorizations will be closed upon approval of onasemnogene abeparvovec-xioi (Zolgensma®); AND
  11. The requested dose is within FDA labeled dosing for the requested indication [medical record documentation required].

Duration of Approval: 30 days (one-time, single-dose treatment per lifetime)

**Please note, for certain identified gene and cellular therapies such as onasemnogene abeparvovec-xioi (Zolgensma®), when coverage is available and the individual meets medically necessary criteria, distribution from a specialty pharmacy provider due to cost (distribution channel restriction) may be required in order for coverage to be provided. Please contact BCBS NC to coordinate this therapy.

FDA Label Reference

MedicationIndicationDosingHCPCSMaximum Units*
onasemnogene
abeparvovec-xioi
(Zolgensma®)
intravenous (IV) infusion		SMA in patients < 2 years old with biallelic
mutations in the SMN1 gene		Single-dose one-time IV infusion of
1.1 x 1014 vector genomes (vg) per kg
of body weight without retreatment		J33991

*Maximum units allowed for duration of approval

References:

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information:

Criteria and treatment protocols are reviewed annually by the Blue Cross NC P&T Committee, regardless of change. This policy is reviewed in Q4 annually.

January 2023: Criteria change: Changed SMN2 gene copy requirement from three or fewer copies to four or fewer copies.

June 2021: Criteria change: Medical policy formatting change. Policy notification given 4/16/2021 for effective date 6/16/2021.

*Further historical criteria changes and updates available upon request from Medical Policy and/or Corporate Pharmacy.

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