ModifiedColorado Rocky Mountain Health PlansPolicy IEXD0059.11

Spinraza (nusinersen) — Medical Benefit Drug Policy (coverage criteria)

Medical benefit drug policy governing use and coverage criteria for Spinraza (nusinersen) for treatment of spinal muscular atrophy (SMA) under UnitedHealthcare Individual Exchange plans (excludes MA, NV, NY).

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicySpinraza (nusinersen) — Medical Benefit Drug Policy

Policy CodePolicy IEXD0059.11

Change TypeMaterial coverage criterion added (inpatient admission restriction)

Effective DateJan 1, 2026

Next Review DateN/A

Key ActionObtain prior authorization with documentation of diagnosis, SMN1/SMN2 copy number, baseline and follow-up motor assessments, and do not request a planned inpatient admission solely to administer Spinraza.

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 current information.

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.

Archived previous policy version IEXD0059.10.

FDA-approvedFDA status

≤4 loading dosesInitial auth limit

≤3 maintenance doses/12 mo

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1Material change count

IEXD0059.11Policy ID

Coverage Criteria for Spinraza (nusinersen)

Initial Therapy

Covered when ALL of the following are met

Initial Diagnosis: Diagnosis of spinal muscular atrophy by, or in consultation with, a neurologist with expertise in the diagnosis of SMA

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

Examples include homozygous deletion of exon 7 at locus 5q13 or compound heterozygous mutations

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

Baseline motor assessment: Submission of baseline exam using at least one of the following, based on age and motor ability: HINE‑2, HFMSE, RULM, or CHOP INTEND (baseline assessments may be omitted for patients <2 months of age to avoid delaying therapy)

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

Concomitant therapy exclusion: Patient is not receiving concomitant chronic SMN‑modifying therapy (e.g., risdiplam)

Administration and dosing: Spinraza to be administered intrathecally by, or under the direction of, healthcare professionals experienced in lumbar puncture; dosing within FDA‑approved labeling; provider does not request a planned inpatient admission solely for administration

Initial authorization limited to no more than four loading doses

Continuation Therapy

Covered when ALL of the following are met

Diagnosis and prior therapy: Diagnosis of spinal muscular atrophy by, or in consultation with, a neurologist with expertise in SMA; patient has previously received Spinraza therapy

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

Concomitant therapy exclusion: Patient is not receiving concomitant chronic SMN‑modifying therapy (e.g., risdiplam)

Demonstration of clinical benefit: Submission of recent medical records documenting positive clinical response from pretreatment baseline as shown by at least one of the following: HINE‑2, HFMSE, RULM, or CHOP INTEND with the specified point‑change thresholds or achievement/maintenance of new motor milestonesSee motor scale response thresholds (HINE‑2, HFMSE, RULM, CHOP INTEND)

Medical necessity criteria (evidence-aligned populations)

Covered when clinical evidence supports treatment consistent with trial populations and documented diagnostic criteria

Infantile‑onset (Type I) evidence: Coverage aligned to the ENDEAR trial population: symptomatic infants ≤7 months at first dose showed improved motor milestone response and reduced risk of death or permanent ventilation with nusinersen (responder definition based on HINE motor milestone improvements; 51% responders vs 0% control; 47% reduction in death or permanent ventilation; 63% reduction in death)

Later‑onset (Type II/III) evidence: Coverage aligned to the CHERISH trial population: children aged 2–12 years able to sit independently with HFMSE screening scores 10–54 experienced clinically meaningful improvement (mean ~3.9 point improvement; ≥3 points considered clinically important)HFMSE between 10 and 54 at screening

Maintenance dosing interval: Maintenance dosing in later‑onset SMA trials was every six months; evidence for greater dose intensity/frequency than CHERISH is lacking—maintenance regimens should be consistent with trial schedules (ENDEAR: maintenance every 4 months for infantile; CHERISH: maintenance every 6 months for later‑onset)

Administration setting (new requirement)

Revised coverage criteria include an added requirement regarding the administration setting.

Administration setting restriction: Provider must not request a planned inpatient admission for the sole purpose of administering Spinraza.

Material change effective 01/01/2026

Spinraza is considered not medically necessary for treatment of spinal muscular atrophy in individuals who do not have chromosome 5q mutations or deletions. It is also considered not medically necessary for patients receiving concomitant chronic SMN‑modifying therapy (for example, risdiplam/Evrysdi).

Current guidance does not support routine simultaneous use of two disease‑modifying SMA treatments (for example, gene replacement therapy plus nusinersen) outside of a controlled clinical trial. The 2024 European consensus update concluded that, in the absence of randomized evidence demonstrating superiority of combination therapy, combinatorial regimens cannot be recommended at this time; the policy therefore does not endorse concurrent disease‑modifying treatments and excludes concomitant chronic SMN‑modifying therapy.

This policy supersedes and archives the prior version IEXD0059.10. See the Policy History for the effective date of the revision and a summary of material changes.

Use of Spinraza for spinal muscular atrophy without confirmed chromosome 5q mutation or deletion is not covered. Concurrent treatment with other SMN‑modifying agents (for example, risdiplam) while receiving Spinraza is explicitly not supported by this policy and is considered not medically necessary.

The evidence base for later‑onset (Type II/III) SMA used maintenance dosing at six‑month intervals in published trials (for example, CHERISH); to date no randomized clinical trial has evaluated nusinersen at a dose intensity or frequency greater than that studied in CHERISH. Therefore, use of dosing intensity or frequency greater than those evaluated in randomized trials for later‑onset SMA is not supported by the available randomized evidence.

Coding and Outcome Thresholds

Applicable HCPCS CodesHCPCS

J2326

Injection, nusinersen, 0.1 mg

Applicable ICD-10 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

Covered HCPCS CodesHCPCSCovered

J2326

Injection, nusinersen, 0.1 mg

Motor scale response thresholds

HINE-2 response thresholdImprovement or maintenance of previous improvement of at least a 2-point increase in ability to kick (or maximal score) OR at least a 1-point increase in any other HINE-2 milestone; net positive improvement across milestones or achievement of new motor milestones

HFMSE response thresholdImprovement or maintenance of previous improvement of at least a 3-point increase in HFMSE score from pretreatment baseline

RULM response thresholdImprovement or maintenance of previous improvement of at least a 2-point increase in RULM score from pretreatment baseline

CHOP-INTEND response thresholdImprovement or maintenance of previous improvement of at least a 4-point increase in CHOP-INTEND score from pretreatment baseline

HFMSE meaningful change

Provider Requirements, Prior Authorization, and Denial Risk

Prior Authorization

Prior Authorization and Inpatient Admission Restriction

Prior authorization is required for Spinraza (nusinersen). Initial authorizations are limited to no more than four loading doses; reauthorization (continuation) authorizations are limited to no more than three maintenance doses (12 months total). Providers must include documentation supporting medical necessity as described below. Requests that include a planned inpatient admission solely for the purpose of administering Spinraza may be denied or restricted; do not request inpatient admission for sole administration.

Initial authorization: ≤ 4 loading doses
Reauthorization/continuation: ≤ 3 maintenance doses (covers 12 months)
Planned inpatient admission solely for administration may trigger denial or restriction

Prior Authorization

Prior Authorization and Administration Location Requirement

Coverage and the appropriate location of administration are governed by the insurer’s Medical Benefit Drug Policy and applicable benefit design. Spinraza must be administered intrathecally by, or under the direction of, clinicians experienced with lumbar puncture. Providers should follow the UnitedHealthcare Medical Benefit Drug Policy and any updated location-of-care requirements when requesting authorization.

Background on Spinal Muscular Atrophy and Spinraza

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease most often caused by biallelic pathogenic variants in the SMN1 gene; the number of SMN2 copies modulates disease severity. Spinraza (nusinersen) is an antisense oligonucleotide that modifies SMN2 splicing to increase full‑length SMN protein. Clinical trials, including ENDEAR (infantile‑onset) and CHERISH (later‑onset), demonstrated improved motor milestones and reductions in death or permanent ventilation in appropriately characterized trial populations; coverage under this policy is aligned with those evidence‑based populations and required diagnostic documentation.

Definitions and Outcome Measures

Definition — 5q spinal muscular atrophy (5q SMA)

Genetic causeSMA caused primarily by biallelic (homozygous) variants in the SMN1 gene

5q genetic confirmationMutation or deletion of genes on chromosome 5q, e.g., homozygous deletion of exon 7 at locus 5q13 or compound heterozygous mutations

Clinical implicationDocumentation of 5q SMN1 mutation/deletion is required for coverage; SMN2 copy number informs phenotype severity

Motor outcome measures — thresholds for response

HINE-2Response defined as >=2-point increase in ability to kick (or maximal score) OR >=1-point increase in other milestones, net positive improvement, or new milestone achievement

HFMSEResponse defined as >=3-point increase in HFMSE from pretreatment baseline or achievement of new motor milestones

Policy Revision History and Changes

2026-01-01coverage_criteria_revisionLatest

Revised coverage criteria to add a requirement that the provider must not request a planned inpatient admission solely for the purpose of administering Spinraza; supporting sections (Background, Clinical Evidence, References) were updated and the prior policy version IEXD0059.10 was archived.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicySpinraza (nusinersen) — Medical Benefit Drug Policy

Policy CodePolicy IEXD0059.11

Change TypeMaterial coverage criterion added (inpatient admission restriction)

Effective DateJan 1, 2026

Next Review DateN/A

SourceLink

On This Page

Reauthorization limited to no more than three maintenance doses per 12 months

Administration and dosing: Spinraza prescribed by, or in consultation with, a neurologist experienced in SMA; administered intrathecally by experienced providers; dosing within FDA‑approved labeling; provider does not request a planned inpatient admission solely for administration

To date no randomized trial supports higher intensity than CHERISH for later‑onset SMA

HFMSE meaningful change>= 3 points is considered a clinically important change in HFMSE (CHERISH secondary endpoint)

Evidence sourceCHERISH trial: mean improvement ~3.9 points vs control; proportion with >=3-point increase 57% nusinersen vs 26% control

ApplicabilityUsed to define clinical benefit for continuation/reauthorization in later-onset SMA (HFMSE screening range 10–54)

Intrathecal administration only (lumbar puncture) by experienced clinicians
Follow UnitedHealthcare Medical Benefit Drug Policy for location-of-care rules

Denial Risk

Explicit Non‑Covered Conditions

Spinraza is not considered medically necessary for certain situations. Do not request coverage when these exclusion conditions apply; include documentation if applicable.

SMA without chromosome 5q SMN1 mutations or deletions (e.g., no 5q SMN1 homozygous deletion/mutation) — not covered
Concomitant chronic treatment with other SMN-modifying agents (e.g., risdiplam/Evrysdi) — not covered

Documentation Required

Required Diagnostic and Baseline Documentation

Submit confirmatory diagnostic testing and baseline motor function documentation with prior authorization requests. Documentation must demonstrate SMN1 mutation/deletion status and include SMN2 copy number when available, and baseline motor assessments appropriate for age.

Genetic testing confirming SMN1 homozygous deletion or pathogenic mutation, or compound heterozygous SMN1 mutations
SMN2 copy number (recommended and important for prognosis)
Baseline motor function documentation (e.g., HINE‑2, CHOP‑INTEND, HFMSE, RULM) — include age‑appropriate baseline exams or justification if <2 months old

Note

Reference Member‑Specific Benefit Plan

When there is any conflict between this policy and the member’s benefit design, the member specific benefit plan document controls. Verify coverage and any applicable mandates in the individual member’s plan before submitting an authorization request.

Reference the member specific benefit plan document for final coverage determinations
Check for state or federal mandates that may affect coverage

Denial Risk

Concomitant Therapy Restriction

Concurrent chronic use of another SMN‑modifying therapy (for example, oral risdiplam/Evrysdi) is not allowed while the member is receiving Spinraza under this policy. Providers must document that the patient is not receiving concomitant chronic SMN‑modifying therapy at the time of request.

Do not co‑administer chronic SMN‑modifying therapies (e.g., risdiplam) with Spinraza
Documentation must attest the patient is not on concomitant chronic SMN‑modifying therapy

Step Therapy

Combinatorial Therapy Stance

There is no mandated step therapy requiring trial of another SMN‑modifying agent prior to Spinraza. Combinatorial (simultaneous) use of two disease‑modifying SMA treatments is not recommended outside of controlled clinical trials or formal expert‑led protocols due to insufficient evidence and cost‑effectiveness concerns.

No step therapy requirement to use Spinraza first or second
Combinatorial therapy (e.g., Spinraza plus gene therapy or risdiplam) is discouraged outside clinical trials; consider only within controlled research or expert consensus settings

RULMResponse defined as >=2-point increase in RULM from pretreatment baseline or achievement of new motor milestones

CHOP-INTENDResponse defined as >=4-point increase in CHOP-INTEND from pretreatment baseline or achievement of new motor milestones

Definition of the drug — Nusinersen (Spinraza)

Drug name (generic)Nusinersen

Brand nameSpinraza

Mechanism / indicationAn SMN2‑directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (pediatric and adult patients)

Permanent ventilation — policy definition

Operational definitionPermanent ventilation: >=16 hours ventilation/day continuously for >21 days in the absence of an acute reversible event or tracheostomy

Policy useTime to death or permanent ventilation was a primary endpoint in ENDEAR and informs coverage decisions regarding advanced respiratory dependence

Exclusion implicationPatients dependent on invasive ventilation or non-invasive ventilation beyond naps/nighttime are not eligible for initial therapy

Functional outcome measures for SMA — referenced updates

Updated outcome measuresRevised Upper Limb Module and other updated functional measures are referenced for SMA assessment (see Pera et al., 2019)

Minimal clinically important differencesPublished MCIDs in adults and children inform interpretation of score changes (Stolte et al., 2020)

Trial-aligned measuresHINE-2, HFMSE, RULM, CHOP-INTEND remain the referenced measures for baseline and response assessment in clinical trials and policy criteria

Consensus and guideline updates — cited sources

European consensus update (2024)2024 update: European consensus statement on gene therapy for SMA is cited (Kirschner et al., Eur J Paediatr Neurol 2024)

Best practices update (2024)SMA best practice recommendations for diagnosis considerations are cited (Schroth et al., Neurol Clin Pract 2024)

Working group guidanceAn international SMA working group updated best practice recommendations referenced in policy