BlueCross BlueShield of Tennessee Medical Policy Manual

Onasemnogene Abeparvovec-xioi (Zolgensma®)

IMPORTANT REMINDER

We develop Medical Policies to provide guidance to Members and Providers.  This Medical Policy relates only to the services or supplies described in it.  The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy.  For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed.  If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.

DESCRIPTION

Onasemnogene abeparvovec-xioi (Zolgensma®) is a recombinant adeno-associated virus-based (AAV9-based) gene therapy.  It is designed to deliver a copy of the gene encoding the human SMN1 protein, or survival motor neuron1 protein into pediatric individuals less than 2 years of age with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene who have a diagnosis of Spinal Muscular Atrophy or SMA.

POLICY

• Onasemnogene abeparvovec-xioi for the treatment of Spinal Muscular Atrophy (SMA) is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)

• Onasemnogene abeparvovec-xioi for the treatment of other conditions/diseases is considered investigational. 

• Onasemnogene abeparvovec-xioi for repeat treatment or ante-partum use is considered investigational.

• Concurrent use with nusinersen and/or risdiplam is considered investigational. 

• Use of nusinersen and/or risdiplam after administration of onasemnogene abeparvovec-xioi is considered investigational.

Note: Please see Additional Information

MEDICAL APPROPRIATENESS

INITIAL APPROVAL

• Onasemnogene abeparvovec-xioi  is considered medically appropriate if ALL of the following conditions are met:

• • Diagnosis of spinal muscular atrophy based on the results of SMA newborn screening

  • Diagnosis of spinal muscular atrophy confirmed by genetic testing demonstrating bi-allelic mutations in the survival motor neuron 1 (SMN1) gene as stated below

  • • deletion of both copies of the SMN1 gene OR

    • compound heterozygous mutations of the SMN1 gene (defined below):

    • • pathogenic variant(s) in both copies of the SMN1 gene

      • pathogenic variant in 1 copy and deletion of the second copy of the SMN1gene.

  • Documentation of a genetic test confirms no more than 3 copies of the SMN2 gene.

• The patient is less than 2 years of age at the time of infusion of onasemnogene abeparvovec- xioi.

• Documentation of baseline laboratory assessments such as AST, ALT, total bilirubin, and prothrombin time.

• The patient does not have advanced spinal muscular atrophy (e.g., complete paralysis of limbs, permanent ventilator dependence).

• Baseline anti- adeno-associated virus serotype 9 (AAV9) antibody titers <1:50

RENEWAL CRITERIA

• Onasemnogene abeparvovec-xioi  is NOT considered medically appropriate for renewal

Note: Please see Additional Information

LENGTH OF AUTHORIZATION

Coverage will be provided as a one-time infusion and may not be renewed

APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS

BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.

ADDITIONAL INFORMATION 

For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).

REFERENCES

1.     BlueCross BlueShield Association. Evidence Positioning System. (8:2022). Treatment for Spinal Muscular Atrophy (5.01.28). Retrieved October , 2022 from https://www.evidencepositioningsystem.com/. (54 articles and/or guidelines reviewed)

2.     MICROMEDEX Healthcare Series. Drugdex Evaluations. (2021, November). Onasemnogene abeparvovec-xioi. Retrieved August 3, 2022 from MICROMEDEX Healthcare Series.

3.     Prior, T.W., Finanger, E. (2000, February [Updated 2016, December]). Spinal Muscular Atrophy. In: Adam, M. P., Ardinger, H. H., Pagon, R. A., et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2019. Bookshelf URL:

 https://www.ncbi.nlm.nih.gov/books/ .

4.     Zolgensma [package insert]. Bannockburn, IL; AveXis, Inc., March 2021. Retrieved July, 2021

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