Currentculinary health fundPolicy N/A

Itvisma (onasemnogene abeparvovec-brve intrathecal) for spinal muscular atrophy — Coverage Criteria

Defines medical necessity, prior authorization, dosing, and eligibility criteria for Itvisma gene therapy for patients with spinal muscular atrophy; applies to medical benefit reviews and prescribers managing SMA patients.

Policy Summary

Payerculinary health fund

PolicyItvisma (onasemnogene abeparvovec-brve intrathecal) for spinal muscular atrophy — Coverage Criteria

Policy CodePolicy N/A

Change TypeRevised clinical threshold

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization for a one-time intrathecal dose and submit required documentation including genetic test results, AAV9 titer, and relevant laboratory tests.

SourceLink

POLICY UPDATE CHANGES

Hemoglobin requirement changed from a specific numeric range (8–18 g/dL) to 'within the normal reference range' with a note that reference ranges vary by lab, age, and gender.

≥2 to <18Age range eligible under FDA-approved indication

1.2×10^14Recommended dose

One-timeApproval frequency

≤1:50AAV9 antibody requirement

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Liver/coagulation thresholds

Coverage Criteria for Itvisma (Intrathecal)

Initial (FDA-Approved) Therapy

Approve for a one-time single dose if the patient meets ALL of the following

FDA-Approved Indication - Composite Criteria: A) Patient is ≥ 2 years to < 18 years of age; AND B) Patient has not received Itvisma or Zolgensma (onasemnogene abeparvovec-xioi) in the past (verification in claims history required; if claims unavailable, prescribing physician confirmation is acceptable); AND C) Genetic test confirming diagnosis of spinal muscular atrophy with pathogenic SMN1 variants (documentation required); AND D) Patient meets ONE of the following: (i) two or three SMN2 gene copies (documentation required); OR (ii) BOTH (a) four SMN2 gene copies (documentation required) AND (b) SMN2 copy number determined by a quantitative assay capable of distinguishing four versus five or more copies; AND E) Baseline anti-AAV9 antibody titer ≤ 1:50 (documentation required); AND F) Liver function testing within 30 days with ALL of the following: (i) ALT ≤ 2x ULN; AND (ii) AST ≤ 2x ULN; AND (iii) total bilirubin ≤ 2x ULN; AND (iv) prothrombin time ≤ 2x ULN; AND G) Renal function assessment within 30 days with creatinine < 1.0 mg/dL (documentation required); AND H) Complete blood count within 30 days with BOTH: (i) WBC ≤ 20,000 cells/mm3; AND (ii) hemoglobin within the laboratory reference range (age- and gender-dependent) (documentation required); AND I) Negative for hepatitis B; AND J) Negative for hepatitis C; AND K) HIV negative; AND L) If receiving or previously received Spinraza (nusinersen), prescribing physician confirms further Spinraza therapy will be discontinued; AND M) If receiving or previously received Evrysdi (risdiplam), prescribing physician confirms further Evrysdi therapy will be discontinued; AND N) Medication prescribed by a physician who has consulted with or specializes in SMA/neuromuscular disorders; AND O) If criteria A through N are met, approve one dose of Itvisma (one-time per lifetime) 1.2 x 10^14 vector genomes intrathecal (verification required).

All elements A through O must be met for approval

Coverage of Itvisma is not recommended in the situations listed below. These scenarios should generally be considered contraindications to approval unless new, robust evidence emerges supporting benefit.

1) Complete paralysis of all limbs. There are insufficient data to show benefit in patients with complete limb paralysis. 2) Permanent ventilator dependence. Patients who require ongoing, permanent mechanical ventilatory support are not recommended for coverage given lack of evidence of benefit. 3) Prior receipt of gene therapy. Itvisma has not been studied in individuals who previously received gene therapy; prior gene therapy is a basis for noncoverage. 4) Circumstances not listed in the Recommended Authorization Criteria. Cases outside the specified authorization criteria are not recommended for approval; criteria will be updated as new published data become available.

Laboratory & Eligibility Thresholds / Key Values

AAV9 antibody titer — eligibility

ThresholdBaseline anti-AAV9 antibody titer ≤ 1:50

DocumentationTiter must be documented in the medical record prior to dosing

TimingBaseline test within 30 days of proposed dosing recommended

Liver and coagulation laboratory thresholds

ThresholdALT, AST, total bilirubin, and prothrombin time ≤ 2 times the upper limit of normal

DocumentationLiver and coagulation tests must be performed within the past 30 days and results documented

Components

Prior Authorization, Documentation, and Denial Risks

Prior Authorization

Prior Authorization Required

Prior authorization is required for Itvisma. Approvals are one-time (per lifetime) for a single intrathecal dose when the patient meets the policy criteria. Approval duration is 30 days to allow preparation and administration of the single dose. Prescribing physician must have consulted with or specialize in the condition. If claims history is available, verification of prior receipt of Itvisma or Zolgensma is required. All reviews will be forwarded to the Medical Director for evaluation. Some clients have Embarc Benefit Protection; when applicable the Medical Director will coordinate with eviCore to complete that portion of the review.

Approval: one-time (per lifetime), single dose; authorization duration = 30 days
Prescriber: physician who has consulted with or specializes in the condition
Claims verification: confirm no prior Itvisma or Zolgensma when claims history available

Documentation Required

Documentation is required to support all elements of the requested Itvisma therapy. Documentation may include chart notes, laboratory results, medical test results, claims records, prescription receipts, and other patient-specific information. All documentation must include patient identifiers.

Clinical Background

Spinal muscular atrophy (SMA) is an autosomal recessive, progressive neuromuscular disorder caused by biallelic pathogenic variants in the SMN1 gene, resulting in reduced SMN protein levels and loss of lower motor neurons. Disease severity is modified by the number of copies of the paralogous gene SMN2, with higher SMN2 copy number generally correlating with milder phenotypes.

This policy addresses use of Itvisma (an AAV vector–based gene therapy) administered by intrathecal injection for treatment of SMA in the FDA‑approved population. The therapy is a one‑time, per‑lifetime administration intended to deliver functional SMN gene copies to the central nervous system and potentially alter the disease course; eligibility criteria include age, genetic confirmation of SMA, SMN2 copy assessment, baseline anti‑AAV9 titer, laboratory thresholds, and discontinuation of other SMA disease‑modifying therapies prior to treatment.

Key Definitions

Itvisma — therapy description and indication

Generic nameOnasemnogene abeparvovec-brve

Brand nameItvisma

Description and indicationAn adeno-associated virus vector-based gene therapy indicated for treatment of spinal muscular atrophy with confirmed SMN1 mutation in patients ≥ 2 years of age via intrathecal injection

SMN1 and SMN2 — genetic definitions and role

SMN1Gene in which biallelic pathogenic variants (deletions or mutations) cause spinal muscular atrophy

SMN2Paralogous gene whose copy number modulates SMA severity (higher SMN2 copy number generally associated with milder disease)

Policy Summary

Payerculinary health fund

PolicyItvisma (onasemnogene abeparvovec-brve intrathecal) for spinal muscular atrophy — Coverage Criteria

Policy CodePolicy N/A

Change TypeRevised clinical threshold

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization for a one-time intrathecal dose and submit required documentation including genetic test results, AAV9 titer, and relevant laboratory tests.

SourceLink

On This Page

Includes alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, and prothrombin time

Creatinine — renal function threshold

ThresholdSerum creatinine < 1.0 mg/dL

TimingRenal function assessment within 30 days of dosing is required and must be documented

DocumentationResult must be included in prior authorization or medical record

White blood cell count — laboratory threshold

WBC thresholdWhite blood cell count ≤ 20,000 cells per mm3

TimingComplete blood count must be obtained within the past 30 days and documented

Related requirementCBC must also include hemoglobin within the normal reference range

Hemoglobin — operational definition

RequirementHemoglobin level must be within the laboratory normal reference range (varies by lab, age, and gender)

Change notePolicy revised: previous numeric range (8–18 g/dL) replaced with 'within the normal reference range'

DocumentationHemoglobin result must be included in CBC obtained within 30 days

Genetic test results confirming SMN1 pathogenic variants (e.g., deletion, compound heterozygous mutation) [documentation required]
SMN2 copy number documentation (two or three copies OR four copies with quantitative assay capable of distinguishing ≥5 copies) [documentation required]
Baseline anti-AAV9 antibody titer result (≤ 1:50) [documentation required]
Recent liver function tests within 30 days, including ALT ≤ 2x ULN [documentation required]
Other labs as applicable: creatinine, CBC as may be required for baseline evaluation
Verification of prior therapies via claims history or prescriber attestation (no prior Itvisma/Zolgensma)

Documentation Required

Concomitant Therapy Discontinuation

Concomitant therapy with other SMA-targeted treatments must be discontinued prior to approval. Confirm the patient is not currently receiving nusinersen (Spinraza) or risdiplam (Evrysdi) and provide documentation of discontinuation or that these therapies will be stopped before Itvisma administration.

Discontinue Spinraza (nusinersen) prior to Itvisma administration — provide documentation/attestation
Discontinue Evrysdi (risdiplam) prior to Itvisma administration — provide documentation/attestation
If claims history is unavailable, prescriber attestation that concomitant SMA therapies have been discontinued is required

Denial Risk

Conditions Likely to Trigger Denial

Coverage is not recommended in certain scenarios; these may be grounds for denial. Examples include complete paralysis of all limbs, permanent ventilator dependence, or prior receipt of gene therapy. Coverage is also not recommended for circumstances not listed in the Recommended Authorization Criteria.

Complete paralysis of all limbs — not recommended for approval
Permanent ventilator dependence — not recommended for approval
Prior receipt of gene therapy (including prior Itvisma/Zolgensma) — not recommended for approval
Requests that do not meet the Recommended Authorization Criteria — not recommended for approval

SMN2 copy number is used in eligibility: 2–3 copies OR exactly 4 copies confirmed by a quantitative assay able to distinguish 4 vs ≥5 copies