CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Evrysdi (risdiplam) — Coverage Criteria for Spinal Muscular Atrophy

Clinical coverage and prior authorization criteria for Evrysdi (risdiplam) to treat pediatric and adult patients with spinal muscular atrophy (SMA), including initiation and continuation requirements and dosing limits.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEvrysdi (risdiplam) — Coverage Criteria for SMA

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization with genetic confirmation of 5q SMA (SMN1 mutation/deletion) and required documentation of baseline or recent motor assessments.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthsauthorization period granted for initial and continuation approvals

Types 1-3SMA types eligible for coverage

4 tiersmaximum daily dose limits specified

Requiredgenetic confirmation required

Coverage Criteria for Evrysdi (risdiplam)

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Initial Therapy Criteria

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

Initial therapy criteria

Member has type 1, type 2, or type 3 SMA.
There is genetic documentation of 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote.
Member is not dependent on invasive ventilation or tracheostomy, and is not using non-invasive ventilation beyond naps and nighttime sleep.
Gene replacement therapy history
- Member has not previously received gene replacement therapy for SMA (e.g., Zolgensma).
- Member has previously received gene replacement therapy for SMA (e.g., Zolgensma) and has experienced worsening in clinical status since receiving gene replacement therapy as demonstrated by decline from highest score achieved or baseline on one of the following assessments: 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), or BSID-III (inability to sit without support for >5 seconds on item 22).
Member will not use Evrysdi (risdiplam) and Spinraza concomitantly.
Dose limits: Member's daily dose will not exceed age/weight limits: <2 months: 0.15 mg/kg; 2 months to <2 years: 0.2 mg/kg; ≥2 years and <20 kg: 0.25 mg/kg; ≥2 years and ≥20 kg: 5 mg.

Operational note

Continuation Therapy Criteria

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

Continuation therapy criteria

Member has type 1, type 2, or type 3 SMA.
Member is not dependent on invasive ventilation or tracheostomy, and is not using non-invasive ventilation beyond naps and nighttime sleep.
Submission of medical records (most recent assessment within 1 month prior to continuation request) documenting a positive clinical response from pretreatment baseline to Evrysdi therapy as demonstrated by at least one of the following assessment-specific criteria: HINE-2, HFMSE, CHOP-INTEND, MFM32, or BSID-III.
Assessment-specific continuation criteria
- HINE-2

All indications for Evrysdi (risdiplam) other than the FDA‑approved indication for treatment of spinal muscular atrophy (SMA) are considered experimental/investigational and not medically necessary.

Use of Evrysdi for any indication other than treatment of SMA in pediatric or adult patients is not medically necessary and will not be approved.

Dosing and Coding Limits

Evrysdi maximum daily dose by age/weight

<2 months of age0.15 mg/kg per day

2 months to <2 years of age0.2 mg/kg per day

>=2 years and <20 kg0.25 mg/kg per day

>=2 years and >=20 kg5 mg per day

Prior Authorization, Documentation, and Concomitant Therapy

Prior Authorization

Prior Authorization Required

Prior authorization is required. Genetic confirmation of 5q SMA (SMN1 mutation/deletion) and documentation that the member meets clinical eligibility criteria for treatment of SMA must be submitted to initiate review.

Authorization may be granted for up to 12 months when criteria are met.
Member must have type 1, 2, or 3 SMA with genetic documentation of 5q SMA (homozygous mutation, homozygous deletion, or compound heterozygote).
Member must not be dependent on invasive ventilation or on non‑invasive ventilation beyond naps/nighttime sleep.

Documentation Required

Required documentation — Initiation of therapy

For initiation of therapy, submit genetic confirmation of SMN1 deletion or mutation and baseline motor assessments to establish pretreatment status.

Genetic test confirming SMN1 deletion or mutation (5q SMA).
Baseline medical records showing at least one of the following motor assessments, appropriate to age and ability: HINE‑2, HFMSE, CHOP‑INTEND, MFM32, or BSID‑III.
Dose limits by age/weight must be followed: <2 months 0.15 mg/kg; 2 months to <2 years 0.2 mg/kg; ≥2 years & <20 kg 0.25 mg/kg; ≥2 years & ≥20 kg 5 mg.

Documentation Required

Required documentation — Continuation of therapy

For continuation of therapy, submit medical records within 1 month of the request documenting a positive clinical response from pretreatment baseline to Evrysdi therapy.

Recent (≤1 month) assessment using at least one of HINE‑2, HFMSE, CHOP‑INTEND, MFM32, or BSID‑III demonstrating clinical improvement or maintenance as specified in continuation criteria.
Examples of acceptable continuation evidence: HINE‑2 improvements (≥2‑point kick or ≥1‑point other milestones), HFMSE increase ≥3 points, CHOP‑INTEND increase ≥4 points, MFM32 increase correlating with functional improvement, BSID‑III ability to sit without support ≥5 seconds after 12 months.
For members who received prior gene replacement therapy (e.g., onasemnogene abeparvovec), submit documentation of stabilization or improvement with Evrysdi or evidence of clinical worsening after gene therapy and subsequent response to Evrysdi.
Dose limits by age/weight must continue to be followed for ongoing therapy.

Prior Authorization

Concomitant therapy and prior gene therapy

Evrysdi must not be used concomitantly with nusinersen (Spinraza). Additional restrictions apply for members with prior gene replacement therapy.

Concomitant use of Evrysdi and Spinraza is not permitted.
Members who previously received gene replacement therapy (e.g., Zolgensma) must either meet initial therapy criteria prior to restarting Evrysdi, or for continuation requests provide documentation of clinical worsening after gene therapy and subsequent stabilization or improvement on Evrysdi as specified in continuation criteria.
Authorization may be granted for members post‑gene therapy only if they meet the genetic and clinical criteria and have documentation supporting the need and response to Evrysdi.

Background on Spinal Muscular Atrophy and Therapy

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder typically caused by deletion or mutation of the SMN1 gene, resulting in loss of motor neurons and progressive muscle weakness. Evrysdi (risdiplam) is an oral, FDA‑approved therapy indicated to treat SMA across pediatric and adult populations and is intended to improve or stabilize motor function.

Definitions and Clinical Improvement Thresholds

Genetic confirmation of SMA

Required genetic test resultDeletion or mutation at the SMN1 allele confirmed by genetic testing (document 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote)

PurposeEstablish diagnosis of 5q spinal muscular atrophy for prior authorization

DocumentationInclude genetic test report in initiation submission medical records

Clinically significant improvement (examples)

HINE-2Improvement or maintenance of previous improvement: >=2-point increase in ability to kick (or maximal score), or >=1-point increase in any other HINE-2 milestone (excluding voluntary grasp)

HFMSEImprovement: >=3-point increase from baseline

CHOP-INTENDImprovement: >=4-point increase from baseline

MFM32Increase from baseline correlated with functional improvement (documented increase)

BSID-IIIAbility to sit unassisted >5 seconds on item 22 (or documented improvement in sitting ability)

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEvrysdi (risdiplam) — Coverage Criteria for SMA

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization with genetic confirmation of 5q SMA (SMN1 mutation/deletion) and required documentation of baseline or recent motor assessments.

SourceLink

On This Page

Improvement or maintenance of previous improvement of at least a 2-point (or maximal score) increase in ability to kick.

Improvement or maintenance of previous improvement of at least a 1-point (or maximal score) increase in any other HINE-2 milestone (e.g., head control, rolling, sitting, crawling, standing, or walking) excluding voluntary grasp.

Net positive improvement in number of HINE-2 motor milestones (more improved/maintained than worsened), or achievement and maintenance of new motor milestones that would otherwise be unexpected (e.g., sit or stand unassisted, walk).

HFMSE

Improvement or maintenance of previous improvement of at least a 3-point increase in HFMSE score.
Achievement and maintenance of any new motor milestone from pretreatment baseline that would otherwise be unexpected.

CHOP-INTEND

Improvement or maintenance of previous improvement of at least a 4-point increase in CHOP-INTEND score.
Achievement and maintenance of any new motor milestone from pretreatment baseline that would otherwise be unexpected.

MFM32: Member has experienced an increase in MFM32 score from baseline that correlates with a clinically significant functional improvement.

BSID-III: Member exhibited the ability to sit without support for at least 5 seconds after 12 months of treatment.

Post-gene therapy worsening: If Evrysdi was prescribed due to clinical worsening after gene replacement therapy (e.g., Zolgensma), there is documentation of stabilization or improvement in clinical status with Evrysdi therapy (e.g., impact on motor milestones).

Member will not use Evrysdi (risdiplam) and Spinraza concomitantly.

Dose limits: Member's daily dose will not exceed age/weight limits: <2 months: 0.15 mg/kg; 2 months to <2 years: 0.2 mg/kg; ≥2 years and <20 kg: 0.25 mg/kg; ≥2 years and ≥20 kg: 5 mg.