CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Evrysdi (risdiplam) for spinal muscular atrophy

Policy governing authorization criteria for Evrysdi (risdiplam) for treatment of pediatric and adult patients with spinal muscular atrophy (SMA), including required documentation, prescriber specialty, dosing limits, exclusions (concomitant use), and criteria for initial and continuation authorization (12-month approvals).

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEvrysdi (risdiplam) for spinal muscular atrophy

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionPrior authorization is required; submit genetic test documentation and relevant baseline or recent motor assessment records to initiate or continue therapy.

POLICY UPDATE CHANGES

No material changes to clinical or coverage criteria.

3Covered SMA types

12 moAuthorization duration

5Assessment tools

Coverage Summary

Coverage stance: covered_with_criteria for Evrysdi (risdiplam) for pediatric and adult spinal muscular atrophy (SMA). Scope: policy governs authorization for type 1, type 2, or type 3 SMA with 12-month authorizations for both initial and continuation therapy when all criteria are met. Required documentation includes genetic confirmation of SMN1 deletion/mutation and baseline or recent functional motor assessments. Prescriber requirement: must be prescribed by or in consultation with a physician who specializes in SMA. Dosing limits (daily maxima) are age/weight‑based and must not be exceeded. Concomitant use with nusinersen (Spinraza) is excluded. Patients previously treated with gene replacement therapy have an alternate path requiring documented clinical worsening or subsequent stabilization/improvement as specified.

Initial Therapy Criteria (Medical Necessity)

Initial Therapy - Medical Necessity Criteria

Authorization of 12 months may be granted when ALL of the following criteria are met:

ALL of the following

Member has type 1, type 2, or type 3 spinal muscular atrophy (SMA)
Genetic documentation of 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote
Member is not dependent on invasive ventilation or tracheostomy
Member is not dependent on use of non-invasive ventilation beyond naps and nighttime sleep
If member previously received gene replacement therapy
- Member has not previously received gene replacement therapy for SMA (e.g., Zolgensma)
- Previously received gene replacement therapy and worsening
  - HFMSE: decline of at least 3 points>= 3 point decline
  - HINE-2: decline of at least 2 points on kicking and 1 point on any other milestone (excluding voluntary grasp)>= 2 point decline on kicking; >= 1 point decline on other milestone
  - CHOP-INTEND: decline of at least 4 points>= 4 point decline
Member will not use Evrysdi and Spinraza concomitantly
Concomitant use is an exclusion
Dosing limit by age/weight (member daily dose must not exceed):
- <2 months: Members less than 2 months of age: 0.15 mg/kg0.15 mg/kg
- 2 months to <2 years: Members 2 months to less than 2 years of age: 0.2 mg/kg0.2 mg/kg
- >=2 years <20 kg: Members 2 years of age and older weighing less than 20 kg: 0.25 mg/kg0.25 mg/kg
- >=2 years >=20 kg: Members 2 years of age and older weighing 20 kg or more: 5 mg
Medication must be prescribed by or in consultation with a physician who specializes in treatment of spinal muscular atrophy
Prescriber specialty requirement

Continuation Therapy Criteria (Medical Necessity)

Continuation Therapy - Medical Necessity Criteria

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

ALL of the following

Member continues to have type 1, type 2, or type 3 SMA
Member is not dependent on invasive ventilation or tracheostomy
Member is not dependent on use of non-invasive ventilation beyond naps and nighttime sleep
Submission of medical records (chart notes, laboratory values) of the most recent assessment (less than 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 specified assessments
Acceptable demonstrations of positive clinical response (one of the following depending on assessment tool):
- HINE-2: Member exhibited improvement or maintenance of previous improvement of at least a 1-point (or maximal score) increase in any HINE-2 milestone (excluding voluntary grasp) OR at least a 2-point (or maximal score) increase in ability to kick>=1 point increase in milestone OR >=2 point increase in kicking
- HFMSE: Member exhibited improvement or maintenance of previous improvement of at least a 3-point increase in HFMSE score OR achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so>=3 point increase
- CHOP-INTEND: Member exhibited improvement or maintenance of previous improvement of at least a 4-point increase in CHOP-INTEND score OR achieved and maintained new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so>=4 point increase
Continuation dosing limit by age/weight (member daily dose must not exceed):
- <2 months: Members less than 2 months of age: 0.15 mg/kg0.15 mg/kg
- 2 months to <2 years: Members 2 months to less than 2 years of age: 0.2 mg/kg0.2 mg/kg
- >=2 years <20 kg: Members 2 years of age and older weighing less than 20 kg: 0.25 mg/kg0.25 mg/kg
- >=2 years >=20 kg: Members 2 years of age and older weighing 20 kg or more: 5 mg
Member will not use Evrysdi and Spinraza concomitantly
Concomitant use is an exclusion

Documentation & Provider Actions

Documentation Requirements

Submission of the following information is necessary to initiate the authorization review:

ALL of the following

Genetic test showing deletion or mutation at the SMN1 allele
Baseline assessment for initiation: For initiation: medical records of baseline assessment for at least one of the following assessment tools (based on patient age and motor ability): HFMSE, HINE-2, CHOP-INTEND, BSID-III, MFM32
Most recent assessment for continuation: For continuation: medical records of the most recent (less than 1 month prior to continuation request) assessment by at least one of the following: HINE-2, HFMSE, CHOP-INTEND, MFM32, BSID-III
Post-gene therapy documentation: For members prescribed Evrysdi due to clinical worsening after gene replacement therapy: documentation of the impact of Evrysdi therapy (e.g., impact on motor milestones)

Prior Authorization

Authorization required

Prior authorization is required to start or continue Evrysdi. Submit genetic testing documentation confirming deletion or mutation at the SMN1 allele and relevant baseline or recent motor assessment records as part of the authorization request.

Documentation Required

Assessment records required

Provide baseline assessment records at initiation using one of the accepted tools (HFMSE, HINE-2, CHOP-INTEND, BSID-III, or MFM32). For continuation, submit the most recent assessment performed less than 1 month before the request documenting the required improvements or stabilization per the policy (see specific thresholds for each tool).

Baseline (initiation): one of HFMSE, HINE-2, CHOP-INTEND, BSID-III, MFM32
Continuation: most recent assessment <1 month prior demonstrating specified improvement or stabilization

Documentation Required

Prescriber specialty requirement

Evrysdi must be prescribed by, or in consultation with, a physician who specializes in the treatment of spinal muscular atrophy. Documentation of prescriber specialty or consultation should be included in the request.

Billing Rule

Dosing limits enforced

Member daily dose must not exceed the age- and weight-specific maxima listed in the policy. Dosing above the specified limits (e.g., 0.15 mg/kg for <2 months; 0.2 mg/kg for 2 months to <2 years; 0.25 mg/kg for ≥2 years & <20 kg; 5 mg for ≥2 years & ≥20 kg) may jeopardize authorization or payment.

Denial Risk

Concomitant therapy exclusion

Claims may be denied if Evrysdi is used concomitantly with Spinraza. Concomitant use of Evrysdi and Spinraza is an exclusion in the policy and must be avoided.

Clinical Thresholds & Assessment Metrics

Thresholds

HFMSE decline (worsening after gene therapy)>= 3 point decline

CHOP-INTEND decline (worsening after gene therapy)>= 4 point decline

HINE-2 decline (worsening after gene therapy) - kicking>= 2 point decline on kicking

HINE-2 decline (worsening after gene therapy) - other milestones>= 1 point decline on any other milestone (excluding voluntary grasp)

HINE-2 improvement (continuation)>= 1 point increase (or maximal score) in milestone OR >= 2 point increase in ability to kick

HFMSE improvement (continuation)>= 3 point increase

CHOP-INTEND improvement (continuation)>= 4 point increase

BSID-III sitting milestone (continuation)Ability to sit without support for at least 5 seconds (item 22) after 12 months

Acceptable assessment tools include HFMSE, HINE-2, CHOP-INTEND, BSID-III, and MFM32. The policy maps score changes to worsening versus improvement: specific numeric declines on these tools (e.g., HFMSE ≥3, CHOP-INTEND ≥4, HINE-2 declines on kicking or other milestones, BSID-III sitting inability) indicate clinical worsening after gene therapy, whereas defined increases or attainment/maintenance of motor milestones on these same tools indicate a positive clinical response for continuation.

Background & Clinical Evidence

Background: Evrysdi (risdiplam) is FDA‑approved for treatment of SMA in pediatric and adult patients. The policy requires genetic confirmation of SMN1 deletion or mutation and functional motor assessments (one of HFMSE, HINE-2, CHOP-INTEND, BSID-III, MFM32) to establish baseline and to document response. Patients who previously received gene replacement therapy follow a specified alternate path requiring documented clinical worsening to initiate or documentation of stabilization/improvement to continue.

Evidence: Evrysdi [package insert], Genentech, Inc; February 2024; and Duong T. et al., Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study. Neurol Ther 2023.

Definitions

SMN1: Survival Motor Neuron 1 gene; deletion or mutation confirms 5q SMA.

HFMSE: Hammersmith Functional Motor Scale Expanded — an assessment of motor function used to establish baseline and measure change.

HINE-2: Hammersmith Infant Neurological Exam Part 2 — a milestone-based infant neurological exam used for baseline and follow-up.

CHOP-INTEND: Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders — an infant motor function scale used for baseline and monitoring.

BSID-III: Bayley Scales of Infant and Toddler Development Third Edition — includes motor items such as the item for sitting without support.

MFM32: Motor Function Measure 32 — a 32-item motor function assessment used to quantify functional change.

OpenPayer

Evrysdi (risdiplam) for spinal muscular atrophy

Coverage Summary

Initial Therapy Criteria (Medical Necessity)

Continuation Therapy Criteria (Medical Necessity)

Documentation & Provider Actions

Clinical Thresholds & Assessment Metrics

Applicable Codes

Background & Clinical Evidence

Definitions

Revision History