Currentblue cross blue shield - tennesseePolicy N/A

Onasemnogene Abeparvovec-xioi (Zolgensma®)

Defines coverage and prior authorization criteria for a single total dose of onasemnogene abeparvovec-xioi (Zolgensma) for treatment of pediatric spinal muscular atrophy (SMA) in members, including required documentation, laboratory and genetic tests, exclusion criteria, and prescriber specialty.

Policy Summary

Payerblue cross blue shield - tennessee

PolicyOnasemnogene Abeparvovec-xioi (Zolgensma®)

Policy CodePolicy N/A

Change Typeclarified

Effective DateJun 30, 202615d

Next Review Date

Key ActionSubmit prior authorization with medical records documenting baseline liver function, platelet count, troponin I, creatinine, genetic testing showing bi-allelic SMN1 pathogenic variants, and documentation of SMN2 copy number (<=3).

SourceLink

POLICY UPDATE CHANGES

Policy states authorization of one dose total and lists explicit clinical and laboratory criteria including SMN2 copy number, SMN1 bi-allelic pathogenic variants, age and disease onset limits, AAV9 titer threshold, and exclusions for advanced disease and prior gene therapy.

1Dose authorized

<=3SMN2 copy allowance

1:50AAV9 antibody titer threshold

<2 yrsAge limit for coverage

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Coverage Summary

Coverage stance: covered_with_criteria for a single total dose of onasemnogene abeparvovec-xioi (Zolgensma) when all authorization criteria are met for pediatric SMA patients under 2 years of age with genetically confirmed bi-allelic SMN1 pathogenic variants. Other indications and uses outside these criteria are considered experimental/investigational and not medically necessary. Effective date: 6/30/2026. Scope: defines coverage and prior authorization criteria for one total dose of Zolgensma including required documentation, laboratory and genetic tests, exclusion criteria, and prescriber specialty.

Coverage Thresholds

SMN2 copy number<= 3 copies

AAV9 antibody titer<= 1:50 (ELISA binding immunoassay)

Age at treatment< 2 years

Disease onsetOnset before 6 months of age

Respiratory assistance exclusion>=16 hours/day continuously for 14+ days (excluding acute reversible illness and perioperative ventilation)

Dose authorized1 total dose

Authorization / Medical Necessity Criteria (Initial Therapy)

Authorization / Medical Necessity Criteria

Authorization of one dose total may be granted when ALL of the following criteria are met:

Member has no more than 3 copies of SMN2.<=3 copies

Member has a genetically confirmed diagnosis of SMA, with documentation of bi-allelic pathogenic variants in the survival motor neuron 1 (SMN1) gene (deletions or point mutations).bi-allelic SMN1 pathogenic variants

Member experienced onset of disease before 6 months of age.onset before 6 months

Member does not have advanced SMA, including but not limited to any of the following

Unproven / Exclusions

Exclusions and non-covered indications: prior receipt of gene therapy (including Zolgensma, Itvisma, or other gene therapy) is excluded; use in patients with advanced SMA has not been evaluated and is not covered; repeat administrations (multiple doses) have not been evaluated and are not covered; members with a serious concomitant illness (e.g., severe liver or kidney disease, symptomatic cardiomyopathy) are excluded; members with an active infectious process (e.g., viral, bacterial, or febrile illness) prior to treatment are excluded. Advanced SMA is defined to include complete paralysis of limbs, invasive ventilatory support (tracheostomy), or prolonged respiratory assistance as described below.

Not eligible / Exclusions

Member has not received Zolgensma, Itvisma, or other gene therapy previously.No prior Zolgensma/Itvisma/other gene therapy

Authorization limited to one total dose; prior gene therapy is an exclusion.

Member has advanced SMA (NOT eligible), including but not limited to any of the following

Complete paralysis of limbs

Provider Actions & Prior Authorization Requirements

Prior Authorization

Prior authorization submission required

Prior authorization is required. Submit PA with medical records documenting baseline liver function, platelet count, troponin I, creatinine, genetic testing showing bi-allelic SMN1 pathogenic variants, and documentation of SMN2 copy number (≤3).

Baseline LFTs, platelet count, troponin I, creatinine (laboratory reports)
Genetic testing showing bi-allelic SMN1 pathogenic variants
Documentation of SMN2 copy number (≤3)

Documentation Required

Genetic testing documentation

Provide genetic test results demonstrating bi-allelic pathogenic variants in SMN1 and documentation confirming no more than 3 copies of SMN2.

Genetic testing confirming deletions or point mutations in both copies of SMN1

Applicable Codes & Identifiers

Identifiers / internalmixed

ID_CHS_2025a

Internal policy identifier

Clinical Evidence & References

Evidence sources: Zolgensma package insert, February 2025; supportive compendia including MICROMEDEX (2026) and Lexi-Comp (2026).

Background

Background: Zolgensma (onasemnogene abeparvovec-xioi) is an FDA-approved gene-replacement therapy indicated for pediatric patients less than 2 years of age with spinal muscular atrophy due to bi-allelic SMN1 mutations. Use in patients with advanced SMA and the safety/effectiveness of repeat administrations have not been evaluated. All other indications are considered experimental/investigational and not medically necessary.

Definitions: Advanced SMA — includes complete paralysis of limbs, invasive ventilatory support (tracheostomy), or respiratory assistance ≥16 hours/day continuously for 14 or more days (excluding acute reversible illness or perioperative ventilation). Bi-allelic pathogenic variants in SMN1 — deletions or point mutations confirmed in both copies of the SMN1 gene by genetic testing.

Revision History

2026-06-30policy_effective

Policy effective date set to 6/30/2026 (Internal ID: ID_CHS_2025a); authorization limited to one total dose with required documentation including baseline liver function, platelet count, troponin I, creatinine, genetic testing confirming bi-allelic SMN1 pathogenic variants, and SMN2 copy number (<=3).

2026-06-30criteria_clarifiedLatest

Clarified criteria: authorization of one dose may be granted when ALL criteria met including: age <2 years; disease onset before 6 months; genetically confirmed bi-allelic SMN1 pathogenic variants; no more than 3 copies of SMN2; exclusion of advanced SMA (complete limb paralysis, invasive ventilatory support/tracheostomy, or respiratory assistance >=16 hours/day continuously for 14+ days excluding acute reversible illness/perioperative ventilation); no active infection; AAV9 antibody titer <=1:50 by ELISA; no serious concomitant illness; vaccinations up to date; baseline labs assessed and to be monitored (liver function, platelet count, troponin I, creatinine); discontinue nusinersen or risdiplam prior to administration; no prior receipt of Zolgensma, Itvisma, or other gene therapy. Medical Policy Manual Approved Rev: Do Not Implement until 6/30/26.

Policy Summary

Payerblue cross blue shield - tennessee

PolicyOnasemnogene Abeparvovec-xioi (Zolgensma®)

Policy CodePolicy N/A

Change Typeclarified

Effective DateJun 30, 202615d

Next Review Date

SourceLink

On This Page

Complete paralysis of limbs

Invasive ventilatory support (tracheostomy)

Respiratory assistance for 16 or more hours per day (including non-invasive respiratory support) continuously for 14 or more days in the absence of acute reversible illness (excluding perioperative ventilation).>=16 hours/day continuously for 14+ days

Member is less than 2 years of age.<2 years

Member does not have an active infectious process (e.g., viral, bacterial, or febrile illness) prior to treatment.

Member has an anti-adeno-associated virus 9 (AAV9) antibody titer less than or equal to 1:50 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay.<=1:50 (ELISA)

Member does not have a serious concomitant illness (e.g., severe liver or kidney disease, symptomatic cardiomyopathy).

Member's vaccination status will be up to date prior to Zolgensma administration.

Liver function, platelet count, troponin I, and creatinine levels have been assessed at baseline and will be monitored after Zolgensma administration as clinically appropriate.Baseline labs documented

Baseline lab documentation required for PA submission (see Documentation section).

If the member is on nusinersen (Spinraza) or risdiplam (Evrysdi), it will be discontinued prior to administration of the requested drug.

Member has not received Zolgensma, Itvisma, or other gene therapy previously.No prior Zolgensma/Itvisma/other gene therapy

Authorization limited to one total dose; deny if previously received gene therapy.

Invasive ventilatory support (tracheostomy)

Use of Zolgensma in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated and is considered an exclusion from coverage.

Report documenting SMN2 copy number ≤3

Documentation Required

Baseline laboratory assessment

Document baseline liver function tests, platelet count, troponin I, and creatinine prior to administration and plan for monitoring these parameters after Zolgensma administration as clinically appropriate.

Baseline LFTs
Baseline platelet count
Baseline troponin I
Baseline creatinine
Plan for post-administration monitoring

Documentation Required

Vaccination status

Ensure the member's vaccinations are up to date prior to Zolgensma administration and document vaccination status for the prior authorization.

Documentation of up-to-date vaccinations prior to treatment

Documentation Required

Prescriber specialty requirement

Medication must be prescribed by or in consultation with a physician who specializes in the treatment of spinal muscular atrophy; document prescriber specialty or consultation.

Prescribing physician specialty (SMA specialist) or documentation of specialist consultation

Denial Risk

Single-dose limitation and prior gene therapy exclusion

Authorization is limited to one total dose. Requests will be denied if the member has previously received Zolgensma, Itvisma, or other gene therapy.

One total dose only
Exclude and deny if prior gene therapy received

Documentation Required

Infectious illness exclusion

Do not administer Zolgensma if the member has an active infectious process (e.g., viral, bacterial, or febrile illness). Document absence of active infection prior to treatment to avoid denial or delay.

Document no active infectious process prior to administration

Documentation Required

Concurrent SMA therapy discontinuation

If the member is receiving nusinersen (Spinraza) or risdiplam (Evrysdi), discontinue that therapy prior to Zolgensma administration and document discontinuation.

Documentation of discontinuation of nusinersen or risdiplam prior to treatment