ModifiedColorado Rocky Mountain Health PlansPolicy 2025D0079K

Zolgensma (onasemnogene abeparvovec-xioi) coverage

Medical benefit drug policy governing one-time gene replacement therapy Zolgensma for treatment of spinal muscular atrophy (SMA); applies to covered members under the payer's medical benefit (Community and Commercial plan language present).

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyZolgensma (onasemnogene abeparvovec-xioi) coverage

Policy CodePolicy 2025D0079K

Change TypeRevised (added inpatient admission denial risk)other editorial updates

Effective DateDec 1, 2025

Next Review Date

Key ActionPrior authorization required; authorization limited to one lifetime treatment and provider must not request a planned inpatient admission solely for administration.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria; added criterion requiring the provider does not request a planned inpatient admission for the sole purpose of administering Zolgensma.

Updated Background, Clinical Evidence, and References sections to reflect the most current information.

Archived previous policy version 2024D0079J.

onetreatment per lifetime authorized when criteria met

J3399HCPCS code listed for Zolgensma

<2 yrsmaximum patient age for coverage

≤1:50maximum anti-AAV9 antibody titer allowed

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Coverage Criteria

Initial therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Genetic confirmation: Submission of medical records confirming mutation or deletion of SMN1 (e.g., homozygous deletion of exon 7 or compound heterozygous mutation)

Diagnosis of symptomatic SMA by a neurologist with SMA expertise OR diagnosis based on newborn screening plus medical records showing 4 copies or less of SMN2

Age and prematurity: Patient is less than 2 years of age; for premature neonates, full-term gestational age must be reached before infusion< 2 years

Respiratory status: Patient is not dependent on invasive ventilation or on non-invasive ventilation beyond use for naps and nighttime sleep

Provider qualifications: Zolgensma prescribed by a neurologist with expertise in the treatment of SMA

Concomitant therapy: Patient is not to receive routine concomitant SMN modifying therapy; current authorizations for SMN modifying therapy will be terminated upon Zolgensma approval

Anti-AAV9 titer: Patient does not have an elevated anti-AAV9 antibody titer above 1:50<= 1:50

Steroid prophylaxis: Patient will receive prophylactic prednisolone (or glucocorticoid equivalent) prior to and following Zolgensma per FDA labeling

Route and lifetime limit: Zolgensma will be given intravenously per FDA labeling; patient has never received Zolgensma; authorization limited to one treatment per lifetime and for no longer than 45 days from approval or until patient is 2 years of age, whichever is firstone lifetime treatment

Provider must not request a planned inpatient admission for the sole purpose of administering Zolgensma

Clinical coverage considerations and prioritization — Coverage and treatment urgency considerations described by expert groups and FDA information

Coverage and treatment urgency considerations described by expert groups and FDA information:

Presymptomatic infants identified by newborn screening: Treatment decisions should be guided primarily by SMN2 copy number; determination of SMN1 and SMN2 copy numbers is urgent and required; infants with symptoms or SMN2 copy number <= 3 should have disease-modifying treatment initiated without delay; distinguishing between 4 and 5 copies is importantSMN2 copy number <=3

SMN2 copy number determination must be performed in an expert laboratory with quality control

FDA indicated population: FDA indicates Zolgensma for pediatric patients less than 2 years of age with bi-allelic SMN1 mutations; FDA approval is informational and payer may apply coverage criteriaAge <2 years

FDA approval alone is not a basis for coverage

Older/heavier patients:

The policy considers the following uses not proven or not medically necessary: treatment of pre-symptomatic patients identified by newborn screening who have more than 4 copies of SMN2; treatment of symptomatic later-onset SMA in patients older than 2 years; treatment of SMA cases that do not have chromosome 5q (SMN1) mutations or deletions; and routine combination treatment with concomitant SMN modifying therapies.

The safety and effectiveness of Zolgensma in patients with advanced disease have not been evaluated. Examples of advanced SMA cited in the evidence include complete limb paralysis and permanent ventilator dependence. In addition, the 2024 European expert consensus recommends that intravenous gene replacement therapy for older and heavier patients be performed only under rigorous protocols with continuous monitoring and states that treatment of patients above 21 kg cannot be recommended.

Coverage does not extend to several scenarios where benefit is unproven: pre-symptomatic newborns with >4 SMN2 copies, symptomatic later‑onset SMA in individuals >2 years of age, SMA lacking chromosome 5q (SMN1) mutation/deletion, or routine combination therapy with other SMN modifying agents. Furthermore, repeat administration (re-dosing) of Zolgensma is not supported by evaluated evidence and therefore is not an accepted routine practice.

Repeat administration of onasemnogene abeparvovec-xioi (Zolgensma) has not been evaluated for safety or effectiveness. Because evidence for re-dosing is lacking, repeat administration is not supported within this policy.

Coding

Applicable HCPCSHCPCS

J3399

Injection, Onasemnogene abeparvovec-xioi, per treatment, up to 5x10 15 vector genomes

Applicable ICD-10ICD-10

G12.0	Infantile spinal muscular atrophy, type I [Werdnig-Hoffmann]
G12.1	Other inherited spinal muscular atrophy
G12.8	Other SMAs and related syndromes
G12.9	Spinal muscular atrophy, unspecified

Anti-AAV9 antibody titer — <= 1:50

Anti-AAV9 antibody titerPatient must not have an elevated anti-AAV9 antibody titer above 1:50

Measurement thresholdThreshold specified as <= 1:50

Consequence of exceeding thresholdRequests with antibody titer above 1:50 are denial triggers

Age / Weight limits

FDA age limitCoverage limited to pediatric patients less than 2 years of age (patient must be <2 years)

Premature neonatesFor premature neonates, infusion should be delayed until full-term gestational age is reached

Provider Actions & Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Authorization is limited to one treatment per lifetime and will be issued for no longer than 45 days from approval or until the patient reaches 2 years of age, whichever is first.

Authorization: one lifetime treatment; duration ≤ 45 days from approval or until age 2, whichever comes first.
Prior authorization required for administration consistent with payer medical benefit drug policy.

Denial Risk

Triggers for Denial

Requests will be denied when the patient does not meet key eligibility or safety criteria.

Patient ≥ 2 years of age at time of request.
Patient dependent on invasive ventilation or tracheostomy.

Background

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder most often caused by biallelic loss or mutation of the SMN1 gene. Clinical severity is strongly associated with the number of copies of the paralog gene SMN2, which is used to guide urgency of treatment decisions in presymptomatic infants. Zolgensma is a one‑time adeno‑associated virus (AAV9) mediated gene replacement therapy intended to deliver a functional SMN1 transgene; treatment decisions—especially for newborn‑screened infants—should incorporate SMN2 copy number determination and expert neuromuscular evaluation.

Definitions

SMN1/SMN2

SMN1 definitionSMN1 is the survival motor neuron 1 gene; biallelic loss (e.g., homozygous deletion) causes spinal muscular atrophy (SMA)

SMN2 definitionSMN2 is a paralog of SMN1; the number of SMN2 copies correlates with SMA severity and clinical phenotype

Clinical relevanceDetermination of SMN1 and SMN2 copy number is required for diagnosis and treatment decision-making, especially in newborn screening contexts

SMN2 copy number

DefinitionNumber of copies of the SMN2 gene in a patient; associated with disease phenotype, progression, and outcomes

Use in treatment decisionsSMN2 copy number guides urgency of treatment in presymptomatic infants (low copy number → rapid initiation)

Initial Therapy Criteria

Initial administration

Single-dose IV gene therapy: One-time intravenous administration of Zolgensma per FDA labeling

Authorization limited to one treatment per lifetime; do not request planned inpatient admission solely for administration

Initial therapy guidance for NBS-identified infants — Initial treatment decisions and urgency

Initial treatment decisions and urgency

Newborns identified by NBS: For infants identified by newborn screening, confirmatory diagnostic testing must include determination of SMN1 and SMN2 copy number and documentation of current motor function, age at symptom onset, and symptom severity; treat urgently if symptoms are present or SMN2 copy number is <= 3; distinguish between 4 and 5 SMN2 copies per US consensusSMN2 copy number <=3; distinction 4 vs 5 copies

SMN2 copy number testing should be performed in an expert laboratory with quality control

Step Therapy

Step	Requirement / Notes
No prior-step requirement specified
Approval will terminate existing authorizations for routine concomitant SMN modifying therapy (e.g., risdiplam, nusinersen); patient must not receive routine concomitant SMN modifying therapy
Provider must not request a planned inpatient admission for the sole purpose of administering Zolgensma

Consideration	Guidance / Rationale
Older or heavier patients
European expert panel advises treating heavier and older patients very cautiously because risk increases with dose (proportional to weight/age); consider alternative disease-modifying treatments or intrathecal onasemnogene abeparvovec if evidence supports acceptable efficacy-safety
Combination therapy
Panel does not recommend combinatorial use of gene therapy with other disease-modifying treatments outside controlled clinical trials due to lack of convincing evidence of superiority; consider controlled trials as gold standard

Quantity Limits

Onasemnogene abeparvovec-xioi — per treatment (single dose)

HCPCS code (billing)J3399 — Injection, Onasemnogene abeparvovec-xioi, per treatment, up to 5x10^15 vector genomes

Quantity limitPer treatment (single dose); authorization limited to one lifetime treatment

Authorization periodAuthorization issued for no more than 45 days from approval or until patient is 2 years of age, whichever is first

Site of Care

Billing Rule

Administer per FDA labeling; avoid planned inpatient admission solely for administration

Administer Zolgensma per FDA labeling in an appropriate outpatient/infusion setting and do not request a planned inpatient admission solely for administration.

Follow FDA‑approved intravenous administration and peri‑infusion corticosteroid (prednisolone) prophylaxis

Note

Do not request planned inpatient admission solely for administration (site‑of‑care consideration)

Provider must not request a planned inpatient admission solely for administration of Zolgensma; site‑of‑care requests and inpatient admission planning may affect coverage decisions and should be avoided.