Modifiedsierra health and lifePolicy 2026D0059P

Spinraza (nusinersen) — Coverage Criteria for Spinal Muscular Atrophy

Clinical drug policy governing medical necessity criteria, documentation, and coding for Spinraza (nusinersen) therapy for patients with spinal muscular atrophy (SMA). Applies to providers requesting initial and continuation coverage under this payer policy.

Policy Summary

Payersierra health and life

PolicySpinraza (nusinersen) — Coverage Criteria for Spinal Muscular Atrophy

Policy CodePolicy 2026D0059P

Change TypeMaterial revision — added administrative admission restriction

Effective DateJan 1, 2026

Next Review DateN/A

Key ActionDo not request a planned inpatient admission solely for the purpose of administering Spinraza; obtain prior authorization with required documentation.

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 Spinraza.

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

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

J2326HCPCS code listed

4 loading / 3 maintenanceAuthorization limits

SMN1 biallelicGenetic requirement

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Mortality reduction (Type I)

Coverage Criteria for Spinraza (nusinersen)

inv-01: Initial Therapy

Covered when ALL of the following are met:

Initial Diagnosis and Provider: Diagnosis of spinal muscular atrophy (SMA) by, or in consultation with, a neurologist with expertise in SMA; Spinraza prescribed by, or in consultation with, a neurologist with expertise in the treatment of SMA; administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures

Genetic confirmation: Submission of medical records confirming mutation or deletion in chromosome 5q (e.g., homozygous deletion of exon 7 at locus 5q13) or compound heterozygous SMN1 mutation

Ventilation status: Patient is not dependent on invasive ventilation and not using non-invasive ventilation beyond naps and nighttime sleep

Baseline motor assessment: Submission of baseline exam using at least one of: HINE-2, HFMSE, RULM, or CHOP INTEND (baseline assessments for patients <2 months of age are not required to avoid delay)

Gene therapy history: Either patient has not previously received gene replacement therapy (e.g., onasemnogene abeparvovec) OR if previously received gene replacement therapy, submission of medical records documenting a clinically meaningful functional decline since gene therapy (e.g., loss of motor milestone)

Therapy exclusivity and dosing: Patient is not receiving concomitant chronic SMN-modifying therapy (e.g., risdiplam); nusinersen dosing must follow FDA-approved labeling; provider must not request a planned inpatient admission for the sole purpose of administering Spinraza; initial authorization limited to no more than 4 loading doses

inv-02: Continuation Therapy

Covered when ALL of the following are met:

Ongoing diagnosis and Provider: Diagnosis of spinal muscular atrophy (SMA) by, or in consultation with, a neurologist with expertise in SMA; Spinraza prescribed by, or in consultation with, a neurologist with expertise in the treatment of SMA; administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures

Prior Spinraza exposure: Patient has previously received Spinraza therapy

Ventilation status: Patient is not dependent on invasive ventilation and not using non-invasive ventilation beyond naps and nighttime sleep

No concomitant SMN-modifying therapy: Patient is not receiving concomitant chronic SMN-modifying therapy (e.g., Evrysdi/risdiplam)

inv-03: Not Medically Necessary

Not Medically Necessary (NMN): Use of Spinraza for spinal muscular atrophy without chromosome 5q mutations or deletions; concomitant treatment of SMA in patients receiving chronic SMN-modifying therapy (e.g., Evrysdi/risdiplam)

These uses are described in the policy as not proven or medically necessary and would trigger denial.

inv-04: Covered with criteria

Coverage is informed by clinical trial populations, dosing regimens, and professional guidance; criteria reflect populations studied and guideline recommendations.

General coverage criteria

Age and disease stage considerations: Includes infantile-onset (Type I) and later-onset (Type II/III) SMA consistent with trial evidence showing benefit in both populations.
ENDEAR supports infantile-onset; CHERISH and CS12 support later-onset.

Dosing and maintenance: Nusinersen dosing per trial and regulatory guidance with loading doses followed by maintenance dosing approximately every 6 months as evaluated in CHERISH and CS12; maintenance interval selected based on estimated CSF/spinal tissue half-life reflected in CHERISH protocol.

Documented maintenance interval every 6 months in later-onset SMA trials.

Treatment sequencing and combinations: Concurrent use of multiple disease-modifying therapies (e.g., nusinersen plus gene therapy or risdiplam) is not recommended outside controlled clinical trials; decisions about bridging, switching, or adding therapies should be individualized with documentation provided.

inv-05: Coverage Rationale — Administrative Criterion

Revised coverage rationale includes a new explicit administrative criterion:

Administrative coverage requirement: Provider does not request a planned inpatient admission for the sole purpose of administering Spinraza.

New material coverage criterion added 01/01/2026.

Spinraza (nusinersen) is not proven or medically necessary for treatment of spinal muscular atrophy when the patient does not have a confirmed mutation or deletion on chromosome 5q. The policy also considers Spinraza not medically necessary for concomitant treatment in patients who are receiving other chronic SMN‑modifying therapy (for example, Evrysdi/risdiplam).

The simultaneous use of two disease‑modifying SMA treatments (for example, combining gene replacement therapy with nusinersen or using two SMN‑modifying drugs concurrently) is not recommended outside controlled clinical trials or formal research settings because current evidence does not demonstrate superiority of combination therapy and concerns remain about additive biological effect and cost‑effectiveness.

A new administrative coverage requirement prohibits requesting a planned inpatient admission solely for the purpose of administering Spinraza. Requests that plan inpatient admission only to deliver nusinersen may be excluded from coverage under this policy.

Explicitly considered not medically necessary under this policy are: treatment of SMA without documented chromosome 5q mutation/deletion, and use of Spinraza as a concomitant therapy in patients already receiving chronic SMN‑modifying agents (e.g., Evrysdi/risdiplam). These conditions are exclusion triggers for authorization and may result in denial.

FDA approval information for Spinraza is provided for informational purposes only; FDA approval alone is not a basis for coverage under this policy.

This Medical Benefit Drug Policy is intended to assist in interpreting standard benefit plans and is informational. Coverage determinations must be made consistent with the member's specific benefit plan and any applicable federal or state mandates; in case of conflict, the member specific benefit plan governs.

Provider Requirements, Prior Authorization, and Documentation

Prior Authorization

Prior Authorization and Dose Limits

Prior Authorization Required. Prior authorization is required for both initial and continuation Spinraza (nusinersen) therapy. Initial authorizations are limited to no more than 4 loading doses; reauthorization/continuation approvals will be limited to specified maintenance dosing (e.g., no more than 3 maintenance doses over 12 months). Providers must verify and obtain prior authorization before initiating treatment.

Initial authorizations limited to ≤ 4 loading doses
Reauthorization limited to maintenance dosing (e.g., ≤ 3 maintenance doses / 12 months)

Denial Risk

Prior Authorization and Inpatient Admission Restriction

Authorization requests for planned inpatient admissions solely for the purpose of administering Spinraza may be restricted or denied. Providers should not request a planned inpatient admission for the sole purpose of intrathecal administration of Spinraza.

Relevant Codes and Billing

Applicable HCPCSHCPCSCovered

J2326

Injection, nusinersen, 0.1 mg.

Referenced Diagnosis CodesICD-10

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

Clinical Background and Rationale

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused predominantly by biallelic loss‑of‑function variants in SMN1, leading to degeneration of lower motor neurons and progressive muscle weakness. Phenotypic severity correlates with the number of copies of the paralogous gene SMN2, which produces reduced amounts of full‑length SMN protein; therapies that increase SMN protein (SMN‑modifying therapies) aim to restore motor neuron function.

Nusinersen (Spinraza) is an antisense oligonucleotide that modulates SMN2 splicing to increase full‑length SMN protein and has demonstrated improved motor milestones and survival in clinical trials of infantile‑onset and later‑onset SMA. Clinical assessment scales used in trials and for baseline/response measurement include instruments such as HFMSE, CHOP‑INTEND, RULM, and HINE‑2.

Key Definitions

SMN-modifying therapy — definition and examples

DefinitionSMN-modifying therapy: treatments intended to modify survival motor neuron (SMN) protein levels or function, including intrathecal nusinersen and oral risdiplam.

ExamplesNusinersen (intrathecal) and risdiplam (oral).

Policy restrictionConcomitant chronic use of another SMN-modifying therapy with nusinersen is not permitted under this policy (requests for concomitant treatment are considered not medically necessary).

Mechanism classIncludes antisense oligonucleotides and small-molecule SMN2 splicing modifiers that increase full-length SMN protein production.

Nusinersen (Spinraza) — drug definition and indication summary

DrugNusinersen (brand name Spinraza).

Policy Summary

Payersierra health and life

PolicySpinraza (nusinersen) — Coverage Criteria for Spinal Muscular Atrophy

Policy CodePolicy 2026D0059P

Change TypeMaterial revision — added administrative admission restriction

Effective DateJan 1, 2026

Next Review DateN/A

Key ActionDo not request a planned inpatient admission solely for the purpose of administering Spinraza; obtain prior authorization with required documentation.

SourceLink

On This Page

Demonstrated clinical response: Submission of medical records showing positive clinical response from pretreatment baseline on at least one measure: HINE-2 (e.g., ≥2-point increase in ability to kick or ≥1-point increase in other milestones, net positive improvement, or achievement/maintenance of unexpected new milestones); HFMSE (≥3-point increase or unexpected new milestone); RULM (≥2-point increase or unexpected new milestone); CHOP INTEND (≥4-point increase or unexpected new milestone)

Dosing and authorization limits: Nusinersen dosing must follow FDA-approved labeling; provider must not request a planned inpatient admission for the sole purpose of administering Spinraza; reauthorization limited to no more than 3 maintenance doses (12 months)

Consensus cautions about lack of evidence for additive benefit and cost-effectiveness concerns.

Planned inpatient admission requests solely for Spinraza administration may trigger denial

Administration should occur in appropriate outpatient or procedural settings when feasible

Documentation Required

Authorization and Coverage Rationale

Providers must verify authorization requirements and follow the coverage rationale in the member's benefit plan. Ensure documentation submitted supports the clinical criteria for initial or continuation therapy to facilitate approval.

Confirm prior authorization requirements with payer before service
Adhere to coverage rationale when preparing clinical justification

Note

Member-Specific Plan Governs

Member-specific benefit plan documents and applicable federal or state mandates govern coverage determination. In the event of any conflict between this policy and the member-specific benefit plan, the member-specific plan governs.

Reference the member-specific benefit plan and any applicable mandates for final coverage determination
This policy is informational and may be superseded by member plan terms

Note

Combination Therapy Stance

Combination or sequential use of multiple SMN-directed disease-modifying therapies (e.g., nusinersen, onasemnogene abeparvovec, risdiplam) is not recommended outside of controlled clinical trials due to lack of convincing evidence of superiority and concerns about additive mechanisms, safety, and cost-effectiveness. Temporary 'bridging' strategies may be considered in exceptional situations when a therapy is not immediately available, but simultaneous chronic concomitant SMN-modifying therapy is excluded for coverage.

Combinatorial therapy not recommended outside clinical trials
Bridging may be appropriate in select situations but simultaneous chronic concomitant SMN therapies are excluded from coverage
Requests for coverage when patient is receiving concomitant chronic SMN-modifying therapy (e.g., risdiplam) will be excluded

Step Therapy

No step therapy specifics are defined in this section. Providers should verify any plan-specific step therapy requirements separately in the member's benefit plan.

No step therapy requirements specified here; check member plan for any step edits

Documentation Required

Required Clinical Documentation

Required Clinical Documentation — Initial and continuation authorization requests must include comprehensive medical records as detailed in the policy (e.g., diagnosis and SMA type, SMN1/SMN2 copy number or chromosome 5q mutation testing, baseline and follow-up motor function assessments, prescribing clinician specialty, previous SMA treatments and responses, ventilation dependence status, dosing regimen). Failure to provide the required documentation may result in denial or delay.

Medical notes documenting diagnosis, SMA type, genetic testing (SMN1/SMN2 copy number or 5q mutation/deletion)
Baseline motor exams for initial therapy (HINE-2, HFMSE, RULM, CHOP INTEND as appropriate)
For continuation: documentation of clinical benefit from pretreatment baseline on HINE-2, HFMSE, RULM, or CHOP INTEND
Documentation that the member is not receiving concomitant chronic SMN-modifying therapy

Mechanism/targetAn antisense oligonucleotide directed at SMN2 to increase exon 7 inclusion and production of full-length SMN protein.

Indication summaryIndicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients; FDA approved December 23, 2016. Coverage requires confirmed chromosome 5q SMN1 mutation/deletion per policy criteria.

AdministrationIntrathecal injection administered by clinicians experienced in lumbar puncture; dosing follows FDA labeling with loading and maintenance doses as described in trials (loading on days 1, 29, 85 then maintenance every ~6 months).

HFMSE — motor function scale used in trials

DefinitionHFMSE (Hammersmith Functional Motor Scale Expanded): a validated motor function scale used as the primary outcome in later-onset (Type II/III) SMA trials such as CHERISH to assess change in motor function.

Use in trialsPrimary endpoint in CHERISH was change from baseline in HFMSE score at 15 months; CHERISH inclusion required HFMSE ≥10 and ≤54 at screening.

Clinically important changeA change of ≥3 points in HFMSE has been determined to be clinically important; CHERISH interim analysis showed a 5.9-point difference favoring nusinersen at 15 months (mean change 4.0 vs -1.9).

Medical Benefit Drug Policy — interpretation and member-specific documents

PurposeMedical Benefit Drug Policy: provides assistance in interpreting standard benefit plans for drug coverage and is informational, not medical advice.

Member-specific plan precedenceWhen deciding coverage, reference the member-specific benefit plan document; in the event of a conflict, the member-specific plan governs.

Medicare guidance noteIn the absence of a Medicare NCD or LCD, a Medicare Advantage Organization may create its own coverage determinations using objective evidence-based rationale.

LimitationPolicy does not constitute medical advice and should be used with independent clinical judgment; UnitedHealthcare may modify policies as necessary.