CurrentUnitedHealthcarePolicy CSPA2O24D0O79G

Zolgensma (Onasemnogene Abeparvovec-Xioi) (for Pennsylvania Only)

Defines UnitedHealthcare Community Plan medical benefit drug coverage for Zolgensma for treatment of spinal muscular atrophy (SMA) applicable to Pennsylvania members; describes medical necessity, limitations, and related administrative guidance.

Policy Summary

PayerUnitedHealthcare

PolicyZolgensma (Onasemnogene Abeparvovec-Xioi) (for Pennsylvania Only)

Policy CodePolicy CSPA2O24D0O79G

Change TypeNo material changes

Effective DateDec 1, 2024

Next Review Date

Key ActionObtain prior authorization with documentation confirming bi-allelic SMN1 mutation, age <2 years, anti-AAV9 titer ≤1:50, and the patient is not receiving routine concomitant SMN modifying therapy.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

one lifetimetreatment limit per patient

<2 yrsmaximum age for covered administration

J3399HCPCS code

≤1:50antibody titer requirement

≤45 daysauthorization time limit

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Coverage Criteria for Zolgensma (onasemnogene abeparvovec)

Medical necessity for Zolgensma (covered when ALL of the following are met)

Zolgensma is proven and medically necessary for one treatment per lifetime for SMA when ALL criteria below are met:

Main eligibility: Genetic confirmation: Homozygous deletion or mutation of SMN1 exon 7 at locus 5q13 OR submission of medical records confirming mutation or deletion of genes on chromosome 5q resulting in SMN1 loss.

Documented genetic testing or equivalent medical record evidence is required.

Clinical diagnosis: Diagnosis of SMA by either: (a) newborn screening consistent with SMA, or (b) symptomatic SMA diagnosed by a neurologist with expertise in SMA.

Provide newborn screen results or specialist documentation.

Prematurity consideration: For neonatal patients born prematurely, full-term gestational age must have been reached prior to administration.

Delay treatment until full-term gestational age in premature neonates.

Respiratory status: Patient is not dependent on invasive ventilation or tracheostomy.

Ventilation dependence is a basis for non-coverage or further review.

Age at treatment: Patient is less than 2 years of age at the time of treatment.age < 2 years

Consistent with FDA indication; authorization limits also apply.

Prescribing provider: Zolgensma is prescribed by a neurologist with expertise in the treatment of SMA.

Specialist documentation should be included with the request.

Anti-AAV9 antibody testing: Anti-AAV9 antibody titer is not elevated above 1:50.anti-AAV9 titer ≤ 1:50

Laboratory documentation of titer required.

Concomitant SMN therapy: Patient will not receive routine concomitant SMN modifying therapy (e.g., risdiplam, nusinersen); existing authorizations for such therapies will be terminated upon Zolgensma approval.

Concurrent routine SMN modifying therapy is not allowed.

Steroid prophylaxis: Patient will receive prophylactic prednisolone (or glucocorticoid equivalent) prior to and following receipt of Zolgensma in accordance with FDA labeling.

Follow FDA-approved dosing schedule for corticosteroid prophylaxis.

Prior Zolgensma exposure: Patient has never previously received Zolgensma (one lifetime treatment limit).one lifetime

Authorization will be issued for no more than one treatment per lifetime.

Route of administration: Administration will be intravenous in accordance with FDA-approved labeling.

Intrathecal administration is not covered and has known safety concerns.

Authorization duration: Authorization will be issued for no longer than 45 days from approval or until the patient reaches 2 years of age, whichever is first.authorization ≤ 45 days or until age 2

Authorization covers preparation and administration within this timeframe.

Indications and clinical considerations

Covered when consistent with FDA indication and expert guidance:

FDA indication: Patient is a pediatric patient less than 2 years of age with spinal muscular atrophy and bi-allelic SMN1 mutations (per FDA labeling).age < 2 years; bi-allelic SMN1 mutation

FDA approval information is provided for context; coverage requires benefit determination and documentation.

SMN2 copy number guidance: Document SMN2 copy number and use it to guide treatment decisions in presymptomatic patients; determination should be performed in an expert laboratory when possible.SMN2 copy number recorded

European and professional society guidance recommend using SMN2 copy number to inform urgency of treatment in presymptomatic infants.

Weight and age safety considerations: Patients with body weight greater than 13.5 kg have increased dose-related risks; consider alternative disease-modifying therapies, specialized protocols, or clinical trial settings for these patients.

Zolgensma is considered not proven or not medically necessary for the following scenarios: SMA without chromosome 5q mutations/deletions; treatment of symptomatic later-onset SMA in patients older than 2 years of age; and routine combination treatment with concomitant SMN modifying therapies. Requests that fall outside the listed coverage criteria will be reviewed on a case-by-case basis and may be denied if documentation does not support medical necessity.

Repeat dosing with Zolgensma is specifically addressed as a limitation of use: the safety and effectiveness of repeat administration have not been evaluated. Because evidence for additional doses is lacking, repeat administration is not supported by this policy and is a basis for non-coverage absent compelling supporting data.

Indications identified in the exclusions (for example, SMA without chromosome 5q mutations/deletions, symptomatic later-onset SMA in patients > 2 years of age, or routine combination therapy with other SMN-modifying agents) are considered not medically necessary. Requests for Zolgensma for these indications risk denial unless exceptional, well-documented justification is provided and reviewed.

Use of Zolgensma in patients with advanced SMA (for example, complete paralysis of limbs or permanent ventilator dependence) has not been evaluated and is not supported by available evidence. Such presentations may be a basis for denial because outcomes, safety, and benefit in this population are unproven.

Coding and Clinical Thresholds

HCPCS - DrugHCPCSCovered

J3399

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

ICD-10 - DiagnosesICD-10

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

Neutral / referenced product codesmixed

NDC/HCPCS not specified

Zolgensma product codes are referenced in the package insert but specific billing codes are not listed in this section.

inv-10: Weight threshold for special consideration

Weight threshold> 13.5 kg — patients above this weight have increased dose‑related risk and should only be treated in specific circumstances (consider alternative therapies or clinical trial settings)

Implication for dosing and monitoringDose is proportional to weight; patients >13.5 kg require more rigorous protocols with continuous safety and efficacy monitoring

ConsiderationFor patients >13.5 kg consider alternative disease‑modifying therapies or intrathecal approaches in research settings

inv-11: Target maximum time from diagnosis to treatment

Target maximum timeIdeally initiate treatment within 14 days of diagnosis to avoid delays, particularly in infants

Provider Actions, Authorization & Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Zolgensma (onasemnogene abeparvovec-xioi). Authorization duration: single lifetime treatment; authorization will be issued for no more than one treatment per lifetime and for no longer than 45 days from approval or until the patient reaches 2 years of age, whichever comes first.

Authorization: one lifetime treatment only
Authorization duration: up to 45 days from approval or until 2 years of age, whichever is first

Documentation Required

Required clinical documentation must be submitted with the prior authorization request to demonstrate that the patient meets coverage criteria.

Genetic confirmation of bi-allelic SMN1 mutation or homozygous deletion at chromosome 5q (laboratory report or medical record documentation).
SMN2 copy number (performed in an expert laboratory with quality control) for presymptomatic or early patients when available.

Background on Spinal Muscular Atrophy and Therapy

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletion or mutation of the SMN1 gene on chromosome 5q leading to motor neuron degeneration and progressive muscle weakness. Zolgensma is a one-time SMN1 gene replacement therapy delivered via an AAV9 vector designed to address the underlying genetic cause of SMA; clinical benefit is greatest when administered early and within the populations for which evidence exists.

Definitions and Key Terms

inv-22: Zolgensma (onasemnogene abeparvovec) — product definition

ProductZolgensma (onasemnogene abeparvovec) — a one‑time intravenous SMN1 gene replacement therapy delivered via AAV9 vector to treat the root cause of SMA by providing a functional SMN gene copy

AdministrationSingle intravenous infusion (per FDA labeling and clinical trials)

Treatment limitOne lifetime treatment per patient

inv-23: SMN2 copy number — clinical significance

Clinical significanceSMN2 copy number is the most important predictor of clinical severity and age of onset and should guide treatment decisions, especially in presymptomatic patients

Laboratory recommendationSMN2 copy number determination should be performed in an expert laboratory with quality control measures

Policy Summary

PayerUnitedHealthcare

PolicyZolgensma (Onasemnogene Abeparvovec-Xioi) (for Pennsylvania Only)

Policy CodePolicy CSPA2O24D0O79G

Change TypeNo material changes

Effective DateDec 1, 2024

Next Review Date

SourceLink

On This Page

European consensus advises treating >13.5 kg only in specific circumstances with rigorous monitoring.

Timing of treatment: Early initiation, ideally in the presymptomatic stage, is associated with markedly better outcomes; avoid delays and aim to initiate treatment promptly (ideally within 14 days of diagnosis).treatment initiated promptly (ideally ≤ 14 days)

European consensus and professional society algorithms emphasize rapid treatment initiation to maximize benefit.

Center and monitoring requirements: Treatment should be performed at centers with broad expertise in SMA assessment and management, with capacity for monitoring, managing gene therapy–related adverse events, and systematic data collection.

Centers should follow international standards and participate in disease-specific registries where available.

RationaleEarly initiation, ideally presymptomatic, is associated with markedly better outcomes

Operational noteAim to minimize time from diagnosis to treatment — prompt authorization and coordination recommended

Age at diagnosis and current age (patient must be less than 2 years of age).

Documentation of symptomatic status or newborn screening results as applicable.

Weight at time of request (for consideration of alternatives if >13.5 kg).

Respiratory status: documentation that patient is not dependent on invasive ventilation or tracheostomy.

Anti-AAV9 antibody titer (must not be elevated above 1:50).

Current and prior treatments for SMA (including any SMN modifying therapies such as nusinersen or risdiplam); if on concomitant therapy, documentation that routine combination therapy will be discontinued.

Documentation that Zolgensma will be prescribed/administered by a neurologist or center with expertise in SMA and has capacity for required monitoring and management of adverse events.

Plan for prophylactic corticosteroid administration (prednisolone or equivalent) per FDA labeling.

Any relevant baseline testing and motor function assessments and clinical notes detailing disease duration and motor status.

Denial Risk

Advanced Disease Not Evaluated

Use of Zolgensma in patients with advanced SMA has not been evaluated and may not be appropriate. For patients with complete limb paralysis or permanent ventilator dependence, clinicians should document consideration of the limited evidence and discuss palliative care and alternative approaches with caregivers.

Advanced disease (e.g., complete paralysis of limbs, permanent ventilator dependence) — safety and effectiveness not evaluated; provide explicit clinical justification if request is submitted for such patients.
If treated despite advanced disease, document thorough discussion of expected outcomes, high risk of severe disability, and consideration of palliative care.

Note

Alternatives for Higher-Risk Patients (>13.5 kg)

For patients whose body weight exceeds 13.5 kg or who are older than the primary trial populations, alternatives or enrollment in clinical trials should be considered due to increased dose-related risk and limited published evidence.

Patients > 13.5 kg: consider other disease-modifying therapies (e.g., nusinersen, risdiplam) or referral for clinical trial enrollment; if Zolgensma is requested, provide rationale and plan for enhanced safety monitoring and data collection.
Where Zolgensma is considered for >13.5 kg, documentation should include risk/benefit discussion, why alternatives are unsuitable, and plan for rigorous follow-up (ideally within a protocol or registry).

Use in decision‑makingConsensus and expert guidance recommend using SMN2 copy number to determine urgency of treatment (e.g., immediate treatment for ≤3 copies historically)

inv-24: SMN2 copy number — predictor of severity (alternate chunk refs)

Predictive valueHigher SMN2 copy number generally correlates with milder SMA phenotype; correlation is strong but not absolute (exceptions occur)

EpidemiologyPatients with 2–3 copies are more likely to present with severe (type I–II) disease; ~15% of cases have 4 copies and are less likely to have type I SMA

GuidanceSMN2 copy number should be used alongside clinical context to guide treatment timing and expectations

inv-25: Zolgensma (onasemnogene abeparvovec-xioi) — branded product note

Branded product noteZolgensma (onasemnogene abeparvovec-xioi) — an AAV vector‑based gene therapy indicated for treatment of pediatric patients less than 2 years of age with SMA with bi‑allelic SMN1 mutations (FDA indication)

Intended populationPediatric patients <2 years of age with bi‑allelic SMN1 mutations

Informational useFDA approval is informational only; coverage requires benefit determination and satisfaction of policy criteria