Modifiedculinary health fundPolicy N/A

Spinal Muscular Atrophy - Gene Therapy - Zolgensma Utilization Management Medical Policy

Defines prior authorization, clinical and laboratory criteria, dosing, exclusions, documentation and approval duration for one-time intravenous Zolgensma administration for patients with bi-allelic SMN1 mutations who are under 2 years of age.

Policy Summary

Payerculinary health fund

PolicySpinal Muscular Atrophy - Gene Therapy - Zolgensma Utilization Management Medical Policy

Policy CodePolicy N/A

Change TypeMODIFIED

Effective Date

Next Review Date

Key ActionPrior authorization recommended; provide required genetic, lab, weight and documentation and obtain Medical Director verification for weight-based dosing; approvals are one-time (per lifetime) single dose with 30-day duration.

SourceLink

POLICY UPDATE CHANGES

Itvisma was added as gene therapy that the patient should not have received in the past.

Hemoglobin requirement changed to 'within the normal reference range' from a fixed numeric range (8-18 g/dL).

Clarified that approvals are one-time (per lifetime) single dose and that Medical Director verification is required for weight-based dosing.

Added definition that full-term gestational age can be defined as postmenstrual age ≥ 39 weeks and 0 days.

Removed prior requirement that systemic corticosteroids (prednisolone 1 mg/kg/day) be started 1 day prior and continued 30 days.

1Dose per lifetime permitted

≤2yAge limit for coverage

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Required testing windows (labs/weight)

MultipleNDC kit sizes provided

Coverage Summary

FDA-Approved Indication - Spinal Muscular Atrophy - Treatment (Initial approval)

Approve one-time (per lifetime) single dose if ALL of the following are met:

ALL of the following

Patient is < 2 years of age< 2 years
If the patient is a premature neonate, full term gestational age of 39 weeks and 0 days has been metFull-term gestational age (postmenstrual) ≥ 39 weeks and 0 days
Full-term gestational age can be defined as postmenstrual age (gestational age plus chronological age) being equal to ≥ 39 weeks and 0 days
Patient has not received Zolgensma or Itvisma (onasemnogene abeparvovec-brve intrathecal injection) in the past
Verification in claims history required when available; if no claim or claims history unavailable, prescribing physician must confirm patient has not previously received Zolgensma or Itvisma
Genetic test confirming diagnosis of spinal muscular atrophy with bi-allelic pathogenic variants in SMN1 genebi-allelic SMN1 pathogenic variants
Documentation required; pathogenic variants may include homozygous deletion, compound heterozygous mutation, or other rare mutations
ONE of
- Patient has three or fewer SMN2 gene copies≤ 3 SMN2 copies
  Documentation required
- OR BOTH of
  - Patient has four SMN2 gene copies4 SMN2 copies
    Documentation required
  - Number of SMN2 gene copies determined by a quantitative assay capable of distinguishing between four and five or greater SMN2 copiesassay distinguishes 4 vs ≥5 copies
    Documentation required
Baseline anti-AAV9 antibody titers are ≤ 1:50≤ 1:50
Documentation required
Liver function testing within past 30 days and ALL of
- Alanine aminotransferase (ALT) ≤ 2 times upper limit of normal≤ 2 x ULN
  Documentation required
- Aspartate aminotransferase (AST) ≤ 2 times upper limit of normal≤ 2 x ULN
  Documentation required
- Total bilirubin ≤ 2 times upper limit of normal≤ 2 x ULN
  Documentation required; patients with elevated bilirubin due to neonatal jaundice are acceptable
- Prothrombin time ≤ 2 times upper limit of normal≤ 2 x ULN
Complete blood count within past 30 days and BOTH of
- White blood cell count ≤ 20,000 cells per mm3≤ 20,000 cells/mm3
  Documentation required
- Hemoglobin level within the normal reference rangeWithin normal reference range (age/gender/lab dependent)
  Documentation required; reference ranges vary by laboratory, age and gender
Renal function assessment within past 30 days with creatinine < 1.0 mg/dLcreatinine < 1.0 mg/dL; within past 30 days
Documentation required
If patient currently receiving or has received nusinersen (Spinraza), prescribing physician confirms further Spinraza therapy will be discontinued
If patient currently receiving or has received risdiplam (Evrysdi), prescribing physician confirms further Evrysdi therapy will be discontinued
Medication is prescribed by a physician who has consulted with or who specializes in management of spinal muscular atrophy and/or neuromuscular disorders
Current patient body weight has been obtained within the past 14 daysweight documented within past 14 days
Documentation required
Dosing: one-time (per lifetime) single intravenous infusion of 1.1 x 10^14 vector genomes (vg)/kg based on current patient weight (kg)1.1 x 10^14 vg/kg; single lifetime dose
Verification required by Medical Director; Zolgensma provided as customized kit per weight (refer to NDC kit table)

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title

FDA-Approved Indication - Spinal Muscular Atrophy - Treatment (Initial approval)

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Coverage is covered_with_criteria for a one-time (per lifetime) single intravenous dose of Zolgensma (onasemnogene abeparvovec) for patients < 2 years of age with genetically confirmed spinal muscular atrophy caused by bi-allelic SMN1 pathogenic variants. Required documentation includes genetic confirmation, SMN2 copy number, baseline anti-AAV9 titer ≤ 1:50, liver/renal/CBC results within the specified testing windows, and current weight obtained within the past 14 days. Dosing is a single IV infusion of 1.1 x 10^14 vg/kg based on current weight; approvals are one-time (per lifetime) single dose and the authorization duration is 30 days to allow preparation and administration.

Provider Actions & Authorization

Prior Authorization

Prior authorization required

Prior authorization recommended for medical benefit coverage; approvals provided for one-time (per lifetime) single dose; approval duration is 30 days to allow preparation/administration.

Approval duration: 30 days
One-time (per lifetime) single dose

Documentation Required

Documentation required for clinical and lab criteria

Provide genetic test confirming bi-allelic SMN1 pathogenic variants, SMN2 copy number documentation, baseline anti-AAV9 antibody titer, liver/renal/complete blood count results within required timeframes, current body weight within 14 days, and documentation that the medication is prescribed by a physician who has consulted with or who specializes in management of spinal muscular atrophy and/or neuromuscular disorders.

Genetic test confirming bi-allelic SMN1 pathogenic variants

Applicable Codes

Zolgensma kit NDCs (by weight ranges)NDCCovered

71894-120-02	Kit for weight 2.6 to 3.0 kg
71894-121-03	Kit for weight 3.1 to 3.5 kg
71894-122-03	Kit for weight 3.6 to 4.0 kg
71894-123-03	Kit for weight 4.1 to 4.5 kg
71894-124-04	Kit for weight 4.6 to 5.0 kg
71894-125-04	Kit for weight 5.1 to 5.5 kg
71894-126-04	Kit for weight 5.6 to 6.0 kg
71894-127-05	Kit for weight 6.1 to 6.5 kg
71894-128-05	Kit for weight 6.6 to 7.0 kg
71894-129-05	Kit for weight 7.1 to 7.5 kg

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Zolgensma is supplied as customized kits mapped to patient weight ranges (NDC kit table). Kit selection is based on calculated weight-based dosing and requires Medical Director verification of appropriate weight-based dosing; claims verification or physician confirmation is used when claims are unavailable.

Clinical Evidence & Trial Data

Key evidence includes the open-label clinical trials START and STR1VE (assessing symptomatic infants) and additional studies STR1VE‑EU and SPR1NT (Phase I/III open‑label single‑arm studies). Prescribing information is Novartis Zolgensma PI, February 2025.

Key thresholds

Age< 2 years

Full-term gestational age≥ 39 weeks and 0 days (postmenstrual)

Baseline anti-AAV9 titer≤ 1:50

ALT/AST/Total bilirubin/PT≤ 2 times upper limit of normal

Creatinine< 1.0 mg/dL

White blood cell count≤ 20,000 cells per mm3

Background

Zolgensma is an AAV‑based gene therapy indicated for spinal muscular atrophy due to bi‑allelic SMN1 mutations in patients < 2 years of age. Efficacy was demonstrated in open‑label trials including START and STR1VE (single‑dose IV administration) and additional trials such as STR1VE‑EU and SPR1NT provide supportive data. The recommended administration is a single intravenous infusion at 1.1 x 10^14 vg/kg. Safety concerns include a Boxed Warning for acute serious liver injury and acute liver failure; therefore baseline liver function, renal function, complete blood count, and anti‑AAV9 antibody titers are required prior to infusion. Hemoglobin acceptance was updated to be within the normal reference range, and reference ranges vary by laboratory, age, and gender.

Definitions: Full-term gestational age (postmenstrual) — gestational age plus chronological age being equal to ≥ 39 weeks and 0 days. One-time (per lifetime) single dose — a single administration of Zolgensma at 1.1 x 10^14 vg/kg for the patient's lifetime; no repeat dosing covered.

Revision History

2026-01-28addedLatest

Itvisma was added to the list of gene therapies that the patient must not have previously received; note clarified that if no claim for Zolgensma or Itvisma is present (or claims history unavailable), the prescribing physician must confirm no prior receipt.

2026-01-28revisedLatest

Hemoglobin requirement changed from a fixed numeric range (8–18 g/dL) to 'hemoglobin level within the normal reference range'; added note that reference ranges vary by laboratory, age and gender.

Policy Summary

Payerculinary health fund

PolicySpinal Muscular Atrophy - Gene Therapy - Zolgensma Utilization Management Medical Policy

Policy CodePolicy N/A

Change TypeMODIFIED

Effective Date

Next Review Date

SourceLink

On This Page

Documentation required

SMN2 copy number documentation (including quantitative assay if 4 copies)

Baseline anti-AAV9 antibody titer (≤ 1:50)

Liver function tests within past 30 days (ALT, AST, total bilirubin, prothrombin time ≤ 2x ULN)

Renal function within past 30 days (creatinine < 1.0 mg/dL)

Complete blood count within past 30 days (WBC ≤ 20,000 cells/mm3; hemoglobin within normal reference range)

Current body weight documented within past 14 days

Prescribing physician specialty consultation/documentation

Documentation Required

Verification of prior gene therapy and prior Zolgensma/Itvisma receipt

Verify claims history for prior receipt of Zolgensma or Itvisma when available; if no claim is present or claims history is not available, the prescribing physician must confirm the patient has not previously received Zolgensma or Itvisma.

Documentation Required

Medical Director verification for dosing

Medical Director verification is required for appropriate weight-based dosing and kit selection for the one-time (per lifetime) IV dose of 1.1 x 10^14 vg/kg based on current patient weight.

Billing Rule

Single lifetime dose

All approvals are for a one-time (per lifetime) single dose; dosing is provided as customized kits tied to specific NDC kit sizes per patient weight. Approval duration is 30 days to allow preparation and administration of the kit/dose.

Denial Risk

Conditions not recommended for approval

Claims are not recommended for approval for patients with complete paralysis of all limbs, permanent ventilator dependence, administration in utero, prior receipt of gene therapy, and for circumstances not listed in the recommended authorization criteria.

HemoglobinWithin normal reference range (age/gender/lab dependent)

Dosing1.1 x 10^14 vg/kg as one-time (per lifetime) single intravenous infusion

Weight documentation windowCurrent body weight obtained within past 14 days

Lab testing windowRequired labs obtained within past 30 days

2025-10-01revised

Clarified that approvals are one-time (per lifetime) single dose and approval duration set to 30 days to allow preparation/administration; verification required by Medical Director for appropriate weight-based dosing; dosing documentation/verification language standardized.

2025-10-01revised

Premature neonate requirement moved and clarified with definition that full-term gestational age commences at 39 weeks and 0 days (postmenstrual age ≥ 39 weeks and 0 days).

2024-10-30removed

Removed prior requirement that systemic corticosteroids (prednisolone 1 mg/kg/day starting 1 day prior and continued 30 days) be started.