CurrentBlue Cross Blue Shield - IowaPolicy 05.02.75

Zolgensma (onasemnogene abeparvovec-xioi) coverage

Defines medical necessity and prior authorization requirements for Zolgensma (one-time gene therapy for spinal muscular atrophy) for Blue Cross Blue Shield - Iowa members, including documentation, age limits, genetic criteria, and exclusions.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyZolgensma (onasemnogene abeparvovec-xioi) coverage

Policy CodePolicy 05.02.75

Change TypeNo material change

Effective DateSep 13, 2024

Next Review DateJun 1, 2025

Key ActionSubmit prior authorization with genetic test results, SMN2 copy number, anti-AAV9 ELISA titer, clinical records, and SMA specialist documentation to request one lifetime dose.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 lifetimeauthorization quantity limit

<2 yrsmaximum patient age

≤4SMN2 copy requirement

≤1:50AAV9 titer threshold

J3399HCPCS code

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Coverage Criteria

Initial Therapy (Covered when ALL of the following are met)

Zolgensma is covered when ALL listed criteria are satisfied:

Initial approval criteria: 1) Genetic confirmation of spinal muscular atrophy (SMA) with documented bi-allelic SMN1 mutations (deletions or point mutations). 2) Documentation of no more than four (≤4) copies of SMN2. 3) Patient is less than 2 years of age. 4) Patient does not have advanced SMA (examples include complete paralysis of limbs; invasive ventilatory support such as tracheostomy; or respiratory assistance ≥16 hours/day continuously for ≥14 days in the absence of an acute reversible illness). 5) Anti‑AAV9 antibody titer ≤1:50 by Enzyme‑linked Immunosorbent Assay (ELISA). 6) Patient has not previously received Zolgensma. 7) Medication is prescribed by or in consultation with a physician who specializes in SMA or neuromuscular disorders. 8) Patient is not currently being treated with nusinersen (Spinraza) or risdiplam (Evrysdi), or those therapies will be discontinued prior to Zolgensma administration.

Approval is for one dose per lifetime; authorization period up to 14 days ending on anticipated administration date or until the member turns 2 years of age, whichever occurs first.

Investigational / Not Medically Necessary

Investigational/Not covered indications: Zolgensma is considered investigational or not medically necessary for (including but not limited to): repeat treatment or ante‑partum use; concomitant use with nusinersen (Spinraza) or risdiplam (Evrysdi); symptomatic later‑onset SMA in patients older than 2 years; SMA without chromosome 5q mutations/deletions; SMA not due to bi‑allelic SMN1 mutations; patients with advanced SMA (e.g., complete limb paralysis, permanent ventilator dependence).

Zolgensma is not medically necessary for members who do not meet the criteria set forth in this policy.

The following uses are excluded or considered investigational under this policy and will not meet criteria for coverage: repeat administrations of Zolgensma (the safety and effectiveness of repeat dosing have not been evaluated), ante‑partum use, use in patients with advanced SMA (for example, complete paralysis of the limbs or permanent ventilator dependence), and concomitant use with nusinersen (Spinraza) or risdiplam (Evrysdi). The policy also considers use for symptomatic later‑onset SMA in patients older than 2 years, SMA without chromosome 5q mutations/deletions, or SMA not due to bi‑allelic SMN1 mutations to be investigational.

Zolgensma (abeparvovec‑xioi) is not medically necessary for indications that do not meet the policy criteria, including those specifically listed as investigational above.

Initial Therapy Requirements

Initial therapy requirements

Initial therapy nodes: See coverage criteria: genetic confirmation of bi‑allelic SMN1 mutation; documentation of ≤4 SMN2 copies; patient age <2 years; absence of advanced SMA as defined in policy; anti‑AAV9 ELISA titer ≤1:50; no prior Zolgensma; prescribed by or in consultation with an SMA/neuromuscular specialist; current nusinersen or risdiplam therapy must be discontinued prior to administration if applicable.

Approval is for one dose per lifetime; authorization period up to 14 days or until member turns 2 years of age, whichever occurs first.

Step Therapy

Step	Requirement	Notes
{"text":"1","status":""}\|{"text":"Member must not be currently treated with nusinersen (Spinraza) at time of Zolgensma administration","status":""}\|{"text":"If the member is receiving nusinersen, treatment must be discontinued prior to administration of Zolgensma; concomitant use is considered investigational.","status":""}
{"text":"2","status":""}\|{"text":"Member must not be currently treated with risdiplam (Evrysdi) at time of Zolgensma administration","status":""}\|{"text":"If the member is receiving risdiplam, treatment must be discontinued prior to administration of Zolgensma; concomitant use is considered investigational.","status":""}

Coding

Covered HCPCS CodesHCPCSCovered

J3399

Injection, onasemnogene abeparvovec-xioi, per treatment; up to 5x10^15 vector genomes

inv-06: AAV9 antibody titer (ELISA) — threshold value

AAV9 antibody titer (ELISA)Less than or equal to 1:50 (≤1:50) by ELISA binding immunoassay

Test typeEnzyme-linked Immunosorbent Assay (ELISA) binding immunoassay

Requirement timingResult must be documented as part of prior authorization submission

inv-07: SMN2 copy number — no more than 4 copies

SMN2 copy number requirementNo more than four copies (≤4 copies)

DocumentationSMN2 copy number must be provided with genetic testing confirming bi-allelic SMN1 mutation

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Approval will be for one dose per lifetime. Authorization period: 14 days ending on the date of the anticipated administration from the provider or until the member turns 2 years of age, whichever occurs first.

One dose per lifetime
Authorization period: 14 days ending on anticipated administration date or until member is 2 years old, whichever is first

Denial Risk

Triggers for Denial

Zolgensma (abeparvovec-xioi) will be denied when criteria for medical necessity are not met or when exclusion conditions apply.

Patient is older than 2 years of age at time of administration
Advanced SMA (e.g., complete limb paralysis, permanent ventilator dependence, invasive ventilatory support such as tracheostomy)

Site of Care

Note

Hospital outpatient dosing — single IV infusion per FDA label

For hospital outpatient administration, dosing must follow the FDA product label: a single intravenous infusion of 1.1 x 10^14 vector genomes per kg (single dose) as recommended by the FDA.

Administration setting: hospital outpatient.
Dose: single IV infusion per FDA dosing guidance (1.1 x 10^14 vg/kg).

Quantity Limits

inv-17: Zolgensma (onasemnogene abeparvovec-xioi) — 1 kit (one dose per lifetime)

Quantity limit1 kit per lifetime

Dose allowanceApproval is for one dose per lifetime

Authorization periodAuthorization period up to 14 days ending on anticipated administration date or until member turns 2 years of age, whichever is first

Definitions

inv-14: Advanced SMA — includes complete paralysis, permanent ventilator dependence, prolonged ventilatory support

Complete limb paralysisIncludes complete paralysis of limbs as a feature of advanced SMA

Permanent ventilator dependencePermanent ventilator dependence is included in the definition of advanced SMA

Prolonged ventilatory supportProlonged invasive or non-invasive respiratory support (e.g., ≥16 hours/day continuously for ≥14 days) is considered advanced SMA

Background

Zolgensma (onasemnogene abeparvovec‑xioi) is a one‑time gene replacement therapy that delivers a functional SMN1 gene to motor neurons to provide sustained SMN protein expression for spinal muscular atrophy. It is intended for pediatric patients under 2 years of age with bi‑allelic SMN1 mutations and is administered as a single intravenous infusion per FDA dosing guidance.