Onasemnogene Abeparvovec-xioi (Zolgensma®)
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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 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.
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.
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 does not have advanced SMA, including but not limited to any of the following
- 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
Baseline lab documentation required for PA submission (see Documentation section).
Authorization limited to one total dose; deny if previously received gene therapy.
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
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
- 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
Provider Actions & Prior Authorization Requirements
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)
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
- Report documenting SMN2 copy number ≤3
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
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
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
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
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
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
Applicable Codes & Identifiers
| 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
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).
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.
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