CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Evrysdi (risdiplam) — Coverage Criteria for Spinal Muscular Atrophy

Criteria and prior authorization documentation requirements for coverage of Evrysdi for treatment of spinal muscular atrophy (SMA) for pediatric and adult patients.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEvrysdi (risdiplam) — Coverage Criteria for Spinal Muscular Atrophy

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateN/A

Next Review DateN/A

Key ActionObtain genetic confirmation of SMN1 deletion or mutation and baseline motor assessment with an accepted scale when requesting prior authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthsauthorization period

Types 1–3SMA types eligible

4 tiersdose tiers

FDA-approved onlyindications

Coverage Criteria for Evrysdi (risdiplam)

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Initial criteria: Member has type 1, type 2, or type 3 SMA; genetic documentation of 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote; member is not dependent on invasive ventilation or tracheostomy and not using non-invasive ventilation beyond naps/nighttime sleep; member meets one of the prior gene therapy criteria; member will not use Evrysdi and Spinraza concomitantly; member's daily dose does not exceed age/weight-specific maximums.

See dosing tiers for age/weight-specific maximums.

inv-02: Initial criterion regarding prior gene therapy

Member must meet one of the following regarding prior gene replacement therapy:

Gene therapy status

Worsening after gene therapy: HINE-2: decline of at least 2 points on kicking and 1 point on any other milestone (excluding voluntary grasp); HFMSE: decline of at least 3 points; CHOP-INTEND: decline of at least 4 points; MFM32: decline from baseline; BSID-III: inability to sit without support for more than 5 seconds on item 22.
These thresholds correspond to the specified exams based on member age and motor ability.

inv-03: Continuation Authorization - SMA

Authorization of 12 months may be granted for continued treatment when ALL of the following are met:

Continuation main criteria: Member has type 1, type 2, or type 3 SMA; member is not dependent on invasive ventilation or tracheostomy and not using non-invasive ventilation beyond naps/nighttime sleep; submission of medical records (most recent <1 month) documenting positive clinical response or stabilization from pretreatment baseline on at least one specified assessment; member will not use Evrysdi and Spinraza concomitantly; member's daily dose does not exceed age/weight-specific maximums.

Positive clinical response definitions follow per assessment.

Acceptable evidence of positive response: HINE-2: improvement or maintenance of >=2-point increase in kicking OR >=1-point increase in any other milestone (excluding voluntary grasp) OR net more improved than worsened milestones OR achievement/maintenance of new motor milestones; HFMSE: >=3-point increase or achievement/maintenance of new motor milestone; CHOP-INTEND: >=4-point increase or achievement/maintenance of new motor milestone; MFM32: increase from baseline correlating with clinically significant functional improvement; BSID-III: ability to sit without support for >=5 seconds after 12 months of treatment; OR documentation of stabilization or improvement for members treated after gene replacement therapy.

Meeting any one of these per member qualifies as a positive response.

Members who are dependent on invasive ventilation or tracheostomy, or who use non‑invasive ventilation beyond naps and nighttime sleep, are excluded from coverage for Evrysdi. Authorization requires documentation that the member is not ventilator dependent and does not exceed the specified non‑invasive ventilation use threshold.

Provider Actions and Prior Authorization Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Evrysdi (risdiplam). Requests will be reviewed for medical necessity and may be authorized for up to 12 months when all coverage criteria are met. Do not initiate or continue therapy without an approved prior authorization.

Authorization duration: up to 12 months

Denial Risk

Indication Restriction

Coverage is limited to the FDA‑approved indication: treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. All other uses are considered experimental/investigational and are not covered.

Denial Risk

Respiratory Dependence Exclusion

Requests may be denied if the member is dependent on invasive ventilation or tracheostomy, or if the member uses non‑invasive ventilation beyond naps and nighttime sleep.

Invasive ventilation or tracheostomy = exclusion
Non‑invasive ventilation beyond naps and nighttime sleep = exclusion

Documentation Required

Required Documentation for Prior Authorization

For initiation of therapy submit documentation of genetic confirmation and baseline motor assessment. For continuation submit recent assessment showing clinical response; special documentation is required for members treated after gene replacement therapy.

Initiation: genetic testing confirming SMN1 deletion or mutation
Initiation: baseline motor assessment using an age/ability‑appropriate scale (HINE‑2, HFMSE, CHOP‑INTEND, MFM32, or BSID‑III)
Continuation: most recent assessment (<1 month prior to request) demonstrating positive clinical response on an age/ability‑appropriate scale
Post‑gene therapy: if prescribed due to clinical worsening after gene replacement therapy (e.g., Zolgensma), provide documentation of impact of Evrysdi therapy (e.g., change in motor milestones)

Step Therapy

Step Therapy / Sequencing

Members must not be treated concurrently with Spinraza (nusinersen). No additional step‑therapy sequence is required, but prior gene replacement therapy status and response influence eligibility: members who have not previously received gene replacement therapy may be eligible; members who have received gene replacement therapy must demonstrate clinical worsening as specified.

Concurrent use of Evrysdi and Spinraza is prohibited
No explicit step therapy ordering required
If prior gene replacement therapy (e.g., Zolgensma): documentation of clinical worsening (see criteria) is required

Documentation Required

Post‑Gene Therapy Documentation

If Evrysdi is prescribed for clinical worsening after prior gene replacement therapy, submit documentation demonstrating decline from highest achieved or baseline on an appropriate motor scale (examples/specifications provided in criteria) and documentation of the observed impact of Evrysdi therapy for continuation requests.

Acceptable scales and decline thresholds: HINE‑2 (decline ≥2 points on kicking or ≥1 point on any other milestone excluding voluntary grasp), HFMSE (decline ≥3 points), CHOP‑INTEND (decline ≥4 points), MFM32 (decline from baseline), BSID‑III (inability to sit without support >5 seconds on item 22)
For continuation after gene therapy: documentation of impact of Evrysdi (e.g., motor milestone changes)

Background

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder caused by deletions or mutations in the SMN1 gene leading to loss of motor neurons and progressive weakness. Risdiplam (Evrysdi) is FDA‑approved for treatment of SMA in pediatric and adult patients to improve or stabilize motor function when patients meet the policy’s genetic confirmation and clinical criteria.

Definitions and Accepted Assessments

Genetic confirmation

RequirementDeletion or mutation at the SMN1 allele confirmed by genetic testing is required for initiation of therapy.

DocumentationProvide genetic testing results demonstrating 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote.

TimingGenetic confirmation must be included with the initiation prior authorization submission.

Motor assessment scales

Accepted scalesHammersmith Infant Neurological Exam Part 2 (HINE-2)

Accepted scalesHammersmith Functional Motor Scale Expanded (HFMSE)

Accepted scalesChildren's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND)

Accepted scalesMotor Function Measure 32 (MFM32)

Accepted scalesBayley Scales of Infant and Toddler Development - Third Edition (BSID-III)