CurrentCareSourcePolicy N/A

Zolgensma (onasemnogene abeparvovec-xioj) — Prior-Authorization Coverage Criteria

Defines prior-authorization coverage criteria for Zolgensma gene therapy for pediatric patients with spinal muscular atrophy (SMA) and outlines site-of-service, billing, and benefit type details for affected members.

Policy Summary

PayerCareSource

PolicyZolgensma (onasemnogene abeparvovec-xioj) — Prior-Authorization Coverage Criteria

Policy CodePolicy N/A

Change TypeRevised — material clinical/operational updates

Effective DateMar 1, 2024

Next Review DateN/A

Key ActionSubmit prior authorization with documentation of genetically confirmed biallelic SMN1 mutation, SMN2 copy number (2–4), required baseline labs and anti-AAV9 titer <1:50; therapy must be prescribed by or in consultation with a neurologist.

SourceLink

POLICY UPDATE CHANGES

Updated age to 2 years old and younger and clarified SMN2 copy number requirement to 2 to 4 copies.

Added exclusion for prior Zolgensma administration and concomitant use of Evrysdi; removed childhood vaccination requirement and baseline symptom measurement.

<2 yrsmaximum member age for initial approval

2–4SMN2 copy number required

1.1e14 vg/kgallowed single IV dose

Prior Auththerapy status

Outpatientsite of service

Coverage Criteria for Zolgensma

inv-01: Initial Therapy

Covered when ALL of the following are met for initial authorization:

Initial Authorization

Age: Member is less than two years of age<2 years
Diagnosis of SMA confirmed by genetic/newborn testing showing homozygous SMN1 deletion, compound heterozygous SMN1 mutations, or homozygous SMN1 mutation
Genetic/newborn testing documentation required in chart notes
SMN2 copies: Member has 2 to 4 copies of SMN22-4 copies

Policy Summary

PayerCareSource

PolicyZolgensma (onasemnogene abeparvovec-xioj) — Prior-Authorization Coverage Criteria

Policy CodePolicy N/A

Change TypeRevised — material clinical/operational updates

Effective DateMar 1, 2024

Next Review DateN/A

SourceLink

On This Page

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Prescriber: Medication prescribed by or in consultation with a neurologist

Baseline testing: Baseline labs: liver function tests (AST, ALT, total bilirubin, prothrombin time), platelet counts, troponin-I, and anti-AAV9 antibody titer <1:50anti-AAV9 <1:50

Document results in chart prior to administration

Gestational age (if premature): If premature neonate, gestational age is ≥ 35 weeks>= 35 weeks

Exclusions: No active viral infection; no signs of aspiration; no advanced SMA (e.g., complete limb paralysis or permanent ventilator dependence); no prior treatment with Zolgensma; not concomitantly using Spinraza or Evrysdi (discontinuation required)

Dosage: Single intravenous dose of 1.1 x 10^14 vector genomes per kg1.1 x 10^14 vg/kg

If all requirements are met, approval for 1 month

Operational note: Prior authorization required (billing code J3399); benefit type is Medical with outpatient site of service

inv-02: Reauthorization / Continuation

Reauthorization: Zolgensma will not be reauthorized for continuous use

Single-dose therapy only

CareSource does not cover Zolgensma for indications not explicitly listed in this policy. The policy excludes members who have had prior treatment with Zolgensma and excludes requests for concurrent use with Spinraza (nusinersen) or Evrysdi (risdiplam).

Requests will also be excluded when clinical contraindications described in this policy are present, including active viral infection, advanced SMA (for example, complete limb paralysis or permanent ventilator dependence), or signs of aspiration. Providers should refer to the specific initial-authorization criteria for accepted indications and to the Off‑Label policy for other diagnoses.

Zolgensma (onasemnogene abeparvovec-xioj) is considered not medically necessary when used to treat conditions that are not listed in this document. For any indication outside those specified here, providers should follow the insurer’s Off‑Label policy.

Initial Therapy Requirements

inv-14: Initial Therapy

Initial Therapy: Single IV dose 1.1 x 10^14 vg/kg for pediatric SMA patients <2 years with confirmed biallelic SMN1 mutation and 2-4 copies of SMN2; prescriber must be or consult neurologist; required baseline labs and anti-AAV9 titer <1:50; exclude prior Zolgensma and concurrent Spinraza/Evrysdi

Approval for 1 month if criteria met

Continuation / Reauthorization Criteria

inv-15: Continuation Therapy

Continuation: No reauthorization: single administration only; not authorized for continuous use

Provider Actions, Documentation, and Denial Conditions

Prior Authorization

Prior Authorization Required

Prior authorization is required before Zolgensma (onasemnogene abeparvovec-xioj) is administered. Billing code: J3399. Benefit type: Medical. Site of service: Outpatient.

J3399 — Zolgensma (onasemnogene abeparvovec-xioj)
Benefit type: Medical
Site of service: Outpatient

Prior Authorization

Prescribing Requirements

Zolgensma must be prescribed by or in consultation with a neurologist. Zolgensma must not be given concomitantly with Spinraza (nusinersen) or Evrysdi (risdiplam); these therapies must be discontinued prior to Zolgensma administration.

Prescriber: neurologist or in consultation with neurologist
Discontinue Spinraza and Evrysdi prior to Zolgensma

Documentation Required

Documentation Requirements

Documentation supporting the diagnosis and eligibility must be included in the chart notes. Required documentation includes genetic or newborn testing confirming SMA with biallelic SMN1 mutation and SMN2 copy number, baseline laboratory testing, and antibody testing.

Genetic/newborn testing confirming SMA with biallelic SMN1 mutation (e.g., homozygous deletion, compound heterozygous mutation, or biallelic exon 7 mutation)
SMN2 copy number documented (2–4 copies)
Baseline liver function tests (AST, ALT, total bilirubin, prothrombin time), platelet count, troponin-I
Baseline anti‑AAV9 antibody titer (< 1:50)
Gestational age documented if premature (≥ 35 weeks)

Denial Risk

Denial Conditions

Requests will be denied if the member is older than 2 years of age or if required genetic confirmation or SMN2 copy number documentation is absent or inconsistent with criteria. Other denial conditions include prior treatment with Zolgensma, presence of advanced SMA (e.g., complete limb paralysis or permanent ventilator dependence), active viral infection, or failure to discontinue Spinraza or Evrysdi.

Member older than 2 years (> 24 months)
No documentation of biallelic SMN1 mutation
SMN2 copy number not documented or outside 2–4 copies
Prior treatment with Zolgensma
Advanced SMA (complete paralysis of limbs, permanent ventilator dependence)
Active viral infection
Concomitant use of Spinraza or Evrysdi (not discontinued)

Billing and Coding

Covered HCPCS Billing CodeHCPCSCovered

J3399

BILLING CODE, Zolgensma (onasemnogene abeparvovec-xioj)

Anti-AAV9 antibody titer requirement

Required titerAnti-AAV9 antibody titer must be < 1:50

PurposeBaseline testing required prior to Zolgensma administration

DocumentationResult must be documented in chart notes as part of baseline labs (AST, ALT, total bilirubin, prothrombin time, platelets, troponin-I)

Step Therapy and Prescribing Constraints

Requirement	Details
Medication must be prescribed by or in consultation with a neurologist;
Spinraza (nusinersen) and Evrysdi (risdiplam) must be discontinued prior to Zolgensma administration; concomitant use is not allowed.

Quantity Limits

Zolgensma — Single dose quantity limit

DoseSingle intravenous administration at 1.1 x 10^14 vector genomes (vg) per kg

Quantity limitOne dose per course; Zolgensma is single-dose therapy and will not be reauthorized

Approval durationIf criteria met, approval granted for 1 month

Allowed Site of Service

Note

Outpatient site of service allowed

Administration is allowed in the outpatient setting. Ensure billing and authorization reflect outpatient benefit and site of care.

Site of service allowed: Outpatient

Definitions

SMN1 biallelic mutation definition

DefinitionBiallelic SMN1 mutation = homozygous deletion of SMN1 or compound heterozygous SMN1 mutations resulting in loss of function

Examples listedHomozygous deletion (absence of SMN1 gene); compound heterozygous mutation (e.g., deletion of SMN1 exon 7 on one allele and mutation on the other); homozygous mutation of SMN1 exon 7

DocumentationGenetic/newborn testing confirming the mutation must be included in chart notes

SMN2 copy number requirement

Required copy numberMember must have 2 to 4 copies of SMN2 for coverage

RoleSMN2 copy number is used as a modifier of SMA severity and eligibility

DocumentationSMN2 copy number must be documented in the medical record or genetic testing results

Background

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by loss-of-function biallelic mutations in the SMN1 gene leading to motor neuron degeneration and progressive muscle weakness. Disease severity is modified by the number of SMN2 gene copies; this policy requires documentation of 2 to 4 copies of SMN2 for coverage.

Zolgensma is an adeno-associated virus (AAV)-based, single-dose intravenous gene-replacement therapy indicated for pediatric patients with genetically confirmed SMA due to biallelic SMN1 mutation. Coverage under this policy is limited to patients younger than 2 years of age with required baseline testing (including liver tests, platelet count, troponin‑I, and anti‑AAV9 antibody titer < 1:50) and exclusion of contraindications such as active viral infection, advanced SMA, prior Zolgensma treatment, or concurrent Spinraza/Evrysdi use.