CurrentmhnPolicy CP.PHAR.477

Risdiplam (Evrysdi) for Spinal Muscular Atrophy

Defines medical necessity criteria for initial and continued coverage of risdiplam (Evrysdi) for treatment of spinal muscular atrophy (SMA) across commercial, ICHRA/HIM, and Medicaid lines of business, including genetic, age/weight, baseline assessment, dosing, concurrent therapy exclusions, and renewal requirements.

Policy Summary

Payermhn

PolicyRisdiplam (Evrysdi) for Spinal Muscular Atrophy

Policy CodePolicy CP.PHAR.477

Change TypeRevisions & clarifications (2022–2026)

Effective Date08.07.20

Next Review Date

Key ActionSubmit genetic testing confirming SMA diagnosis and SMN2 copy number (no more than 4 copies) and baseline motor function scores.

SourceLink

POLICY UPDATE CHANGES

2Q 2026 annual review: clarified 'at least' 6 months of trial prior to treatment change per 2025 AAN SMA update; added Itvisma and clarified other items.

2Q 2025 annual review: removed requirement that documentation indicates presence of SMA symptoms for members with four SMN2 copies; added 5 mg tablet formulation for age ≥2 years and weight ≥20 kg.

RT4 update: added pediatric expansion indication for pre-symptomatic infants < 2 months old with dosing and criteria.

1Q 2022 annual review: revised continued therapy language to allow members to receive medication if initiated outside Centene benefit.

1Indication (SMA)

≤4

12Approval duration (months)

5 mgMax dose (≥2 yrs & ≥20 kg)

Coverage Summary

Policy CP.PHAR.477 (Status: CURRENT) covers risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) across Commercial, ICHRA/HIM and Medicaid lines of business when specified criteria are met. The policy requires genetic confirmation of SMA and documentation that the member has no more than ≤ 4 copies of the SMN2 gene. It specifies baseline motor function assessments and excludes concurrent use with Spinraza, Itvisma, or Zolgensma. (See Initial Approval Criteria and Continued Therapy sections.)

Risdiplam is available as an oral solution (60 mg risdiplam powder for constitution to 0.75 mg/mL) and as a 5 mg tablet formulation (tablet use requires Age ≥ 2 years and Weight ≥ 20 kg). Dosing and maximum dose limits are age- and weight-based per the policy (maximum 5 mg/day for patients ≥ 2 years and ≥ 20 kg).

Approvals are issued for a duration of 12 months with renewal contingent on meeting continued therapy response criteria (motor score improvement/maintenance thresholds and other renewal requirements).

High-level thresholds / stats

SMN2 gene copies≤ 4 copies (documentation confirming no more than 4 copies required)

Approval duration12 months

Maximum dose (age ≥2 yrs & ≥20kg)5 mg/day

Tablet formulation age/weightAge ≥ 2 years AND Weight ≥ 20 kg

Initial Therapy Criteria

Initial Approval Criteria - Spinal Muscular Atrophy

Provider must submit documentation supporting that member has met all approval criteria.

Genetic confirmation (one of)
- Homozygous deletions of SMN1 gene (e.g., absence of the SMN1 gene)
- Homozygous mutation in the SMN1 gene (e.g., biallelic mutations of exon 7)
- Compound heterozygous mutation in the SMN1 gene (e.g., deletion of SMN1 exon 7 and mutation of SMN1)
Prescriber specialty requirement: Prescribed by or in consultation with a neurologist

Continuation Therapy Criteria

Continued Therapy - Spinal Muscular Atrophy

Must meet all

Current treatment/prior initial criteria: Currently receiving medication for SMA with 1 to 4 copies of the SMN2 gene, or previously met initial approval criteriaSMN2 gene copies 1-4
Ventilation exclusion: Member does not require tracheostomy or invasive ventilation
Evidence of response (one of)
- For age < 2 years (one of)

Other Indications / Off-Label Guidance

Other diagnoses/indications

Must meet 1 or 2

Recent label change: If drug has undergone a recent (within 6 months) label change not yet reflected in this policy, follow formulary/non-formulary policies CP.CPA.190/ HIM.PA.33/ CP.PMN.255 or CP.CPA.190/ HIM.PA.103/ CP.PMN.16 as applicable
Not specifically listed use: If requested use is not specifically listed and criterion 1 does not apply, refer to off-label use policy CP.CPA.09 / HIM.PA.154 / CP.PMN.53 as applicable

Provider Actions

Documentation Required

Genetic testing documentation

Submit genetic testing confirming the diagnosis of SMA (SMN1 mutation/deletion) and documentation of SMN2 copy number (no more than 4 copies). Include baseline motor function scores with the request.

Prior Authorization

Prescriber specialty requirement

Prescription must be written by a neurologist or by a prescriber in consultation with a neurologist. Provide documentation of the neurologist involvement with the prior authorization request.

Applicable Codes & Product Formulations

Product availability / formulationmixed

Oral solution	60 mg risdiplam as a powder for constitution to provide 0.75 mg/mL solution
Tablet	5 mg tablet formulation

Clinical Evidence & Guidance

Evidence cited in the policy includes: 'Evrysdi Prescribing Information (Genentech, February 2025)' and the 'AAN 2025 SMA update'.

The AAN 2025 update included in the policy recommends that, unless there is an urgent indication (e.g., significant side effects, intolerance to route, significant disease progression, or loss of motor milestones), medication outcomes should be monitored for a minimum of 6–12 months before changing therapy.

The policy references the February 2025 Evrysdi Prescribing Information as the primary prescribing/dosing source and cites clinical trial and regulatory materials supporting dosing, formulation availability, and the pediatric expansion to pre-symptomatic infants where applicable.

Background

Risdiplam is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier that increases production of full-length SMN protein.

Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by mutations or deletions in the SMN1 gene; SMN2 copy number modulates disease severity, with higher SMN2 copy number generally associated with milder phenotypes.

Common motor function scales used for baseline assessment and monitoring in SMA include: CHOP-INTEND, HINE (Section 2 motor milestone score), HFMSE, RHS, RULM, ULM, and the 6-Minute Walk Test (6MWT).

Scale Definitions

CHOP-INTENDChildren's Hospital of Philadelphia Infant Test of Neuromuscular Disorder

HINEHammersmith Infant Neurological Examination (Section 2 motor milestone score)

HFMSEHammersmith functional motor scale expanded

RHS

Revision History

2Q 2026clarifiedLatest

Clarified 'at least' 6 months of trial prior to treatment change per 2025 AAN SMA update; added Itvisma as a newly approved one-time intrathecal therapy and clarified concurrent use examples and related documentation.

2Q 2025revised

Removed requirement that documentation indicates presence of SMA symptoms for members with four SMN2 copies; added 5 mg tablet formulation for age ≥ 2 years and weight ≥ 20 kg.

RT4revised

Policy Summary

Payermhn

PolicyRisdiplam (Evrysdi) for Spinal Muscular Atrophy

Policy CodePolicy CP.PHAR.477

Change TypeRevisions & clarifications (2022–2026)

Effective Date08.07.20

Next Review Date

Key ActionSubmit genetic testing confirming SMA diagnosis and SMN2 copy number (no more than 4 copies) and baseline motor function scores.

SourceLink

On This Page

Tablet formulation age/weight

Age requirement: Age ≥ 2 years
Weight requirement: Weight ≥ 20 kg

SMN2 copy documentation: Documentation of genetic testing confirming no more than 4 copies of SMN2 geneSMN2 gene copies ≤ 4

Baseline motor function documentation (one of)

Age < 2 years baseline: For age < 2 years: CHOP-INTEND score OR HINE Section 2 motor milestone score
Age ≥ 2 years baseline: For age ≥ 2 years: HFMSE score, RHS, ULM, RULM, or 6MWT distance

Ventilation exclusion: Member does not require tracheostomy or invasive ventilation

Concurrent therapy exclusion: Evrysdi is not prescribed concurrently with Spinraza, Itvisma, or Zolgensma

If switching from Spinraza, prescriber attestation of discontinuation required

Dose limits (must not exceed)

Age < 2 months dose: Age < 2 months: 0.15 mg/kg per day0.15 mg/kg/day
Age 2 months to < 2 years dose: Age 2 months to < 2 years: 0.2 mg/kg per day0.2 mg/kg/day
Age ≥ 2 years dosing options
- Age ≥2 yrs & weight <20 kg: Weight < 20 kg: 0.25 mg/kg per day0.25 mg/kg/day
- Age ≥2 yrs & weight ≥20 kg: Weight ≥ 20 kg: 5 mg per day5 mg/day
  Tablet formulation available

Initial Approval Criteria - Additional Initial Conditions

If currently on Spinraza: If the member is currently on Spinraza, documentation of prescriber attestation of Spinraza discontinuation upon initiation of Evrysdi
If history of Zolgensma or Itvisma
- Evidence of poor response: Provider must submit evidence of poor response to Zolgensma or Itvisma (e.g., sustained decrease in CHOP-INTEND or HFMSE score over a period of at least 6 months)
- Attestation of deterioration: Documentation of prescriber attestation of clinical deterioration

CHOP-INTEND improvement:

CHOP-INTEND: demonstrate score improvement or maintenance of previous score improvement of ≥ 4 points from baseline

HINE milestone improvement: HINE motor milestone score: demonstrate score improvement or maintenance of previous improvement in one or more categories AND improvement in more motor milestone categories than worsening

If first renewal since turning 2 years old

CHOP-INTEND or HINE option: For CHOP-INTEND, must demonstrate score improvement or maintenance of previous improvement of ≥ 4 points from baseline OR For HINE motor milestone score, demonstrate improvement or maintenance as specified≥ 4 points for CHOP-INTEND

If ≤ 2 years at therapy initiation and request is for subsequent renewal since turning 2

HFMSE/RHS/ULM/RULM improvement: For HFMSE, RHS, ULM or RULM: demonstrate score improvement or maintenance of previous score improvement from baseline submitted at first renewal since turning 2 years old
6MWT maintenance/improvement: For 6MWT distance: demonstrate improvement or maintenance of baseline distance

If > 2 years at therapy initiation (one of)

HFMSE or RHS improvement: HFMSE or RHS: demonstrate score improvement or maintenance of previous score improvement of ≥ 3 points from baseline≥ 3 points
ULM improvement: ULM: demonstrate score improvement or maintenance of previous improvements in ≥ 2 points from baseline≥ 2 points
RULM improvement: RULM: demonstrate score improvement or maintenance of previous improvements in ≥ 4 points from baseline≥ 4 points
6MWT maintenance/improvement: 6MWT distance: demonstrate improvement or maintenance of baseline distance

Alternative evidence of response: Member has not had a decline in motor function test score(s) from baseline AND medical justification demonstrates and supports that member is responding positively to therapy

Concurrent therapy exclusion: Evrysdi is not prescribed concurrently with Spinraza, Itvisma, or Zolgensma

Dose increase request limits (must not exceed)

Age < 2 months dose: Age < 2 months: 0.15 mg/kg per day0.15 mg/kg/day
Age 2 months to < 2 years dose: Age 2 months to < 2 years: 0.2 mg/kg per day0.2 mg/kg/day
Age ≥ 2 years dosing options
- Age ≥2 yrs & weight <20 kg: Weight < 20 kg: 0.25 mg/kg per day0.25 mg/kg/day
- Age ≥2 yrs & weight ≥20 kg: Weight ≥ 20 kg: 5 mg per day5 mg/day

Documentation Required

Baseline motor function documentation

For initial approval, submit baseline motor function assessment appropriate to the member's age: for age < 2 years submit CHOP-INTEND or HINE Section 2 motor milestone score; for age ≥ 2 years submit HFMSE, RHS, ULM, RULM, or 6-Minute Walk Test (6MWT) as applicable.

Prior Authorization

Concurrent therapy exclusion

Do not prescribe Evrysdi concurrently with Spinraza, Itvisma, or Zolgensma. If switching from Spinraza, include a prescriber attestation that Spinraza has been discontinued upon initiation of Evrysdi.

Documentation Required

Prior treatment failure documentation

If the member has a history of treatment with Zolgensma or Itvisma, submit evidence of poor response (for example, a sustained decrease in CHOP-INTEND or HFMSE score over a period of at least 6 months) and a prescriber attestation documenting clinical deterioration.

Prior Authorization

Dose verification

Document the member's age and weight and ensure the requested dose does not exceed the policy's age/weight-based maximums. Include weight and age with the prior authorization.

Maximum dose limits: <2 months: 0.15 mg/kg/day; 2 months to <2 years: 0.2 mg/kg/day; ≥2 years & weight <20 kg: 0.25 mg/kg/day; ≥2 years & weight ≥20 kg: 5 mg/day (maximum 5 mg/day).

Documentation Required

Renewal response documentation

At renewal, provide evidence of clinical response per the policy thresholds: for age <2 years CHOP-INTEND improvement or maintenance of prior improvement ≥4 points or HINE improvement as specified; for age ≥2 years meet the applicable scale thresholds (e.g., HFMSE/RHS ≥3 points improvement if >2 years at initiation; ULM ≥2 points; RULM ≥4 points; or maintenance/improvement of 6MWT distance). If motor scores are unchanged, include medical justification that documents clinical benefit.

Billing Rule

Approval duration

Approvals are granted for 12 months.

Revised Hammersmith Scale

RULMRevised Upper Limb Module

ULMUpper Limb Module

6MWT6-Minute Walk Test

Added pediatric expansion indication for pre-symptomatic infants < 2 months old with dosing and criteria.

1Q 2022clarified

Revised continued therapy language to allow members to continue medication if treatment was initiated outside the Centene benefit.