Modifiedculinary health fundPolicy N/A

Pdf Spinal Muscular Atrophy Gene Therapy Zolgensma Um Medical De Identified_Pdf

Defines prior-authorization, clinical eligibility, dosing, documentation, and exclusions for medical-benefit coverage of intravenous Zolgensma for patients with spinal muscular atrophy (SMA) with bi-allelic SMN1 mutations who are <2 years of age; approvals are one-time (per lifetime) single-dose with specific laboratory, genetic, and provider requirements.

Policy Summary

Payerculinary health fund

PolicyPdf Spinal Muscular Atrophy Gene Therapy Zolgensma Um Medical De Identified_Pdf

Policy CodePolicy N/A

Change TypeSelected revisions and annual updates (material)

Effective Date

Next Review Date

Key ActionPrior authorization is required; submit documentation (genetic test results, SMN2 copy number, baseline anti-AAV9 titer, recent labs, current weight) and verify prior receipt of Zolgensma/Itvisma; approvals are one-time single-dose and valid for 90 days to allow administration.

SourceLink

POLICY UPDATE CHANGES

Itvisma was added as a gene therapy that the patient should not have received previously; note allows physician confirmation if no claims history.

Approval duration changed from 30 days to 90 days.

Hemoglobin requirement changed from explicit range (8–18 g/dL) to hemoglobin within normal reference range.

Requirement for systemic corticosteroids (prednisolone 1 mg/kg/day starting 1 day prior and for 30 days) was removed.

Clarified that all approvals are provided for one-time (per lifetime) as a single dose and that dosing verification is required by Medical Director; verification in claims history required where available.

Note defining full-term gestational age as postmenstrual age (gestational + chronological) ≥ 39 weeks 0 days was added.

Coverage Summary

Scope: This policy defines prior-authorization, clinical eligibility, dosing, documentation, and exclusions for medical-benefit coverage of intravenous Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) in patients with bi-allelic SMN1 mutations who are < 2 years of age. Coverage stance: covered_with_criteria.

Coverage summary: Intravenous Zolgensma is authorized as a one-time (per lifetime) single-dose intravenous infusion at a dose of 1.1 x 10^14 vector genomes per kg (vg/kg) for eligible patients. Approvals are issued as a single dose and the approval duration is 90 days to allow procurement, preparation, and administration of the one-time kit/dose.

Key thresholds

AgePatient must be < 2 years of age

One-time doseOne-time (per lifetime) single dose — approve only once

Approval durationApproval duration is 90 days to allow procurement/preparation and administration

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

Dosing1.1 x 10^14 vector genomes per kg (vg/kg) IV as a single infusion

Weight documentation recencyCurrent body weight obtained within the past 14 days

Full-term gestational ageFull‑term defined as postmenstrual age ≥ 39 weeks 0 days

Initial Therapy Criteria (One-time Dose)

FDA-Approved Indication - Initial / One-time Dose Approval

Approve for a one-time (per lifetime) single dose if the patient meets ALL of the following (A, B, C, D, E, F, G, H, I, J, K, L, M, and N):

ALL of the following

Patient is < 2 years of age
criterion A
If the patient is a premature neonate, full term gestational age of 39 weeks and 0 days has been met
criterion B. Full-term can be defined as postmenstrual age (gestational age + chronological age) ≥ 39 weeks and 0 days
Patient has not received Zolgensma or Itvisma (onasemnogene abeparvovec-brve intrathecal injection) in the past
criterion C; [verification in claims history required]. If no claims history available, prescribing physician must confirm prior non-receipt

Continuation / Exclusions and Not Recommended Situations

Conditions Not Recommended for Approval

Coverage of Zolgensma is not recommended in the following situations:

ANY of the following

Patient has complete paralysis of all limbs (advanced SMA)
Limitation of use per PI; data lacking
Patient has permanent ventilator dependence
Limitation of use per PI; data lacking
Administration to individuals in utero
Not approved for in utero administration
Prior receipt of gene therapy

Applicable Codes

Zolgensma NDC kit numbers (by weight ranges / kit sizes)NDCCovered

71894-120-02	Kit for weight range 2.6 to 3.0 kg (Total vials per kit = 2)
71894-121-03	Kit for weight range 3.1 to 3.5 kg (Total vials per kit = 3)
71894-122-03	Kit for weight range 3.6 to 4.0 kg (Total vials per kit = 3)
71894-123-03	Kit for weight range 4.1 to 4.5 kg (Total vials per kit = 3)
71894-124-04	Kit for weight range 4.6 to 5.0 kg (Total vials per kit = 4)
71894-125-04	Kit for weight range 5.1 to 5.5 kg (Total vials per kit = 4)
71894-126-04	Kit for weight range 5.6 to 6.0 kg (Total vials per kit = 4)
71894-127-05	Kit for weight range 6.1 to 6.5 kg (Total vials per kit = 5)
71894-128-05	Kit for weight range 6.6 to 7.0 kg (Total vials per kit = 5)
71894-129-05	Kit for weight range 7.1 to 7.5 kg (Total vials per kit = 5)

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Provider Actions & Operational Requirements

Prior Authorization

Prior authorization required

Prior authorization is recommended for medical-benefit coverage; approvals are one-time (per lifetime) single-dose. All approvals and denials will be forwarded to the Medical Director.

Documentation Required

Required documentation

Provide patient-specific documentation to support the prior authorization request. Documentation may include chart notes, laboratory results, medical test results, claims records, prescription receipts, and other patient-specific information.

Genetic test results confirming biallelic SMN1 pathogenic variants
SMN2 copy number assay (quantitative assay if needed to distinguish 4 vs ≥5 copies)
Baseline anti‑AAV9 antibody titer (≤ 1:50)

Clinical Evidence & References

STR1VE / START / SPR1NTPrimary trials showing benefit in survival and motor milestones

1.1e14 vg/kgRecommended one-time IV dose (per lifetime)

Age <2Maximum patient age for coverage

Evidence: Primary efficacy trials including STR1VE, START, SPR1NT and related studies demonstrated benefits in survival and achievement of motor milestones in infants with biallelic SMN1 mutations. The policy cites the Novartis Zolgensma IV prescribing information (Feb 2025) as the prescribing information source for efficacy and dosing.

Safety and testing considerations from the prescribing information and trials include a boxed warning for acute serious liver injury and recommendations to perform baseline laboratory evaluations and anti-AAV9 antibody testing prior to infusion. Trials required baseline anti-AAV9 antibody titers of ≤ 1:50. The number of SMN2 gene copies influences disease severity and treatment recommendations and is used in eligibility determinations.

Background

Background: Zolgensma is an AAV-based, single-dose gene replacement therapy indicated for treatment of spinal muscular atrophy in patients < 2 years with bi-allelic SMN1 mutations. The recommended dosing is a one-time intravenous infusion of 1.1 x 10^14 vg/kg based on current weight.

Safety signals and monitoring: Zolgensma carries a boxed warning for acute serious liver injury and elevated aminotransferases can occur; therefore, baseline liver function testing, renal function assessment, complete blood count, and baseline anti-AAV9 antibody testing are required prior to infusion. Trials required baseline anti-AAV9 titers of ≤ 1:50 and laboratory thresholds (e.g., ALT/AST/total bilirubin/prothrombin time ≤ 2x ULN) are specified in the policy.

Role of SMN2 copy number: SMN2 gene copy number affects disease severity and is incorporated into treatment recommendations and eligibility criteria (e.g., eligibility pathways for ≤ three copies or specific criteria when four copies are present).

Definition: The policy defines one-time (per lifetime) single dose as Zolgensma administered once as a single intravenous infusion of 1.1 x 10^14 vg/kg in a patient's lifetime.

Definitions

Full-term gestational age (definition)Defined in policy as postmenstrual age (gestational age plus chronological age) ≥ 39 weeks 0 days

One-time (per lifetime) single dose (definition)

Revision History

01/28/2026addedLatest

Itvisma was added as a gene therapy that the patient should not have received previously; note allows physician confirmation if no claims history.

01/28/2026revisedLatest

Approval duration changed from 30 days to 90 days.

10/30/2024revised

Policy Summary

Payerculinary health fund

PolicyPdf Spinal Muscular Atrophy Gene Therapy Zolgensma Um Medical De Identified_Pdf

Policy CodePolicy N/A

Change TypeSelected revisions and annual updates (material)

Effective Date

Next Review Date

SourceLink

On This Page

ONE of

Patient has three or fewer SMN2 gene copies
criterion E.i; [documentation required]
OR
- Patient has four SMN2 gene copies
  criterion E.ii.a; [documentation required]
- Number of SMN2 gene copies determined by a quantitative assay capable of distinguishing between four and five or greater SMN2 copies
  criterion E.ii.b; [documentation required]

Liver function testing within past 30 days and ALL of

Baseline anti-AAV9 antibody titers are ≤ 1:50≤ 1:50
criterion F; [documentation required]
Alanine aminotransferase (ALT) ≤ 2 times upper limit of normal≤ 2x ULN
criterion G.i; [documentation required]
Aspartate aminotransferase (AST) ≤ 2 times upper limit of normal≤ 2x ULN
criterion G.ii; [documentation required]
Total bilirubin ≤ 2 times upper limit of normal≤ 2x ULN
criterion G.iii; [documentation required]. Patients with elevated bilirubin due to neonatal jaundice are acceptable
Prothrombin time ≤ 2 times upper limit of normal≤ 2x ULN
criterion G.iv; [documentation required]

Complete blood count within past 30 days and BOTH of

Renal function assessment within past 30 days with creatinine < 1.0 mg/dL< 1.0 mg/dL
criterion H; [documentation required]
White blood cell count ≤ 20,000 cells per mm3≤ 20,000 cells/mm3
criterion I.i; [documentation required]
Hemoglobin level is within the normal reference rangeWithin normal reference range
criterion I.ii; [documentation required]. Note: reference ranges vary by lab, age, and gender

Approvals are for a single-dose, per lifetime administration; continuation therapy or repeat dosing is not applicable because repeat administration has not been evaluated. Prior receipt of gene therapy (including Zolgensma or Itvisma) excludes coverage; verification in claims history is required where available, or the prescribing physician must confirm prior non-receipt if claims are not available.

Administration in utero is not approved and is specifically not covered.

Documentation Required

Verification of prior receipt and dosing

Verify whether the patient has previously received Zolgensma or Itvisma using claims history when available; if claims history is not available or does not show prior receipt, the prescribing physician must confirm the patient has not previously received Zolgensma or Itvisma. Verification of appropriate weight‑based dosing is required by the Medical Director.