Zolgensma (onasemnogene abeparvovec)

POLICY NUMBER

L.5.01.540

Zolgensma (onasemnogene abeparvovec-xioi)

DESCRIPTION

Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated viral 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.

POLICY

I. Designated and Approved Providers for Gene Therapy

Benefits for Gene Therapy will not be provided unless Medically Necessary subject to Medical Policy and a treatment plan submitted by the Network Provider and approved by the Company prior to the initiation of treatment. Gene Therapy services must be received from a Network Provider designated and approved by the Company where the Company has a contract in place with the Network Provider, either directly or through BlueCard®, for the specific Gene Therapy service.

No benefits will be provided unless the Member receives prior authorization through Case Management from Blue Cross & Blue Shield of Mississippi.

II.  Medically Necessary Gene Therapy

Zolgensma (onasemnogene abeparvovec-xioi) will be considered medically necessary when ALL of the following criteria are met:

1. Individual has a genetically confirmed diagnosis of spinal muscular atrophy (SMA) with documentation of bi-allelic mutations in the survival motor neuron 1 (SMN1) gene;

2. Individual has no more than 3 SMN2 copy numbers;

3. Individual has a baseline anti-AAV9 antibody titer ≤1:50;

4. Individual is ≤2 years of age at the time of infusion;

5. Individual has received, or will receive, systemic corticosteroids equivalent to oral prednisolone 1mg/kg/day starting 1-day prior to Zolgensma (onasemnogene abeparvovec-xioi) infusion and for a total of 30-days;

6. Individual will not receive concomitant SMN modifying therapy (e.g., Spinraza [nusinersen intrathecal injection]); AND

7. The prescriber will submit documentation to Blue Cross & Blue Shield of Mississippi Case Management of response to Zolgensma (onasemnogene abeparvovec-xioi) within 3 months following therapy as a follow-up to the prior approval request.

Length of Approval: 1 infusion per lifetime (weight-based) 

Repeat administration of Zolgensma is considered investigational as the safety and efficacy has not been evaluated.

Use of Zolgensma in patients with advanced SMA is considered investigational as the safety and efficacy has not been evaluated. Examples of advanced SMA include but are not limited to complete paralysis of limbs, permanent ventilator-dependence, etc.

POLICY EXCEPTIONS

Self-Insured Groups

For Federal Employee Program (FEP) subscribers, prior approval is required for gene therapy and cellular immunotherapy.

State Health Plan (State and School Employees) Participants

Hood Packaging: Gene therapy and all gene therapy related services are excluded from coverage effective 01/01/2025.

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

A. consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

B. appropriate with regard to standards of good medical practice; and

C. not solely for the convenience of the Member, his or her Provider; and

D. the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

POLICY HISTORY

10/15/2019: New policy added. Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

06/29/2020: Code Reference section updated to add new HCPCS code J3399, effective 07/01/2020.

07/01/2023: Policy Exceptions updated regarding State Health Plan (State and School Employees) Participants. Policy Guidelines updated to change "Nervous/Mental Conditions" to "Mental Health Disorders" and "Medically Necessary" to "medical necessity."

01/15/2025: Policy Exceptions updated to add the following for Hood Packaging: Gene therapy and all gene therapy related services are excluded from coverage effective 01/01/2025.

06/03/2025: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy description updated to change "Zolgensma (onasemnogene abeparvovec)" to "Zolgensma (onasemnogene abeparvovec-xioi)." Medically necessary criteria updated regarding a confirmed diagnosis of spinal muscular atrophy and to state that the individual will not receive concomitant SMN modifying therapy (e.g., Spinraza [nusinersen intrathecal injection]). Sources updated.

SOURCE(S)

Zolgensma prescribing information. Novartis Gene Therapies, Inc. February 2025. Last accessed April 2025.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

CPT copyright American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.