CurrentBlue Cross Blue Shield - IowaPolicy N/A

Evrysdi_Medical_Policy

Defines prior authorization, initiation and continuation criteria, prescriber requirements, quantity limits, dosing guidance, and excluded uses for Evrysdi (risdiplam) for Wellmark members.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyEvrysdi_Medical_Policy

Policy CodePolicy N/A

Change TypeRevised (April 2025 review)

Effective DateSep 9, 2022

Next Review Date

Key ActionSubmit genetic testing confirming SMN1 deletion/mutation, baseline motor assessments (HINE-2, CHOP-INTEND, HFMSE, MFM32, or BSID-III), and documentation of respiratory status; for continuation submit most recent assessments within 90 days showing clinical benefit.

POLICY UPDATE CHANGES

Policy reviewed and revised April 2025; current effective date listed as September 9, 2022 with review in April 2025.

12 monthsApproval duration

HINE-2, CHOP-INTEND, HFMSE, MFM32, BSID-IIIAccepted motor assessments

Permanent invasive ventilationExcluded population

Quantity limitsDosing constraints

Coverage Summary

Evrysdi (risdiplam) is covered with criteria for FDA-approved indications for pediatric and adult spinal muscular atrophy (SMA) when the policy’s approval criteria are met. The policy defines the covered population as Wellmark members with genetically confirmed SMA (SMN1 deletion or mutation) diagnosed with SMA type 1, 2, or 3, excluding members who require permanent invasive ventilation. The purpose of the policy is to ensure appropriate selection of patients for Evrysdi therapy based on FDA labeling, clinical guidelines and clinical studies, and to define prior authorization, initiation and continuation criteria, prescriber requirements, quantity limits, dosing guidance, and excluded uses for Evrysdi.

Policy summary

Approval duration12 months

Accepted motor assessmentsHINE-2, CHOP-INTEND, HFMSE, MFM32, BSID-III

Excluded populationUse in patients requiring permanent invasive ventilation (investigational/not medically necessary)

Dosing constraintsQuantity limits specified (5 mg tablet: 1 tablet/day; 60 mg oral solution: 2 bottles per 24 days; weight/age-based dosing per FDA labeling)

Effective date2022-09-09

Last review2025-04

Initial Therapy Criteria

Initial Approval / Medically Necessary

Evrysdi (risdiplam) may be considered medically necessary when ALL of the following criteria are met:

ALL of the following

Genetic confirmation of SMA: deletion or mutation at the SMN1 allele (examples: homozygous deletion of exon 7 at locus 5q13 or compound heterozygous mutation such as deletion of SMN1 exon 7 and mutation of SMN1)
Member has a diagnosis of SMA type 1, type 2, or type 3
Member is not dependent on invasive ventilation or tracheostomy
Use in patients who require permanent invasive ventilation is considered investigational (not medically necessary)
Respiratory support exclusion
- Not dependent on use of non-invasive ventilation beyond use for naps and nighttime sleep
Prior gene therapy status
- Patient has not previously received gene replacement therapy for SMA (e.g., Zolgensma)
- ALL of the following
  - HFMSE: Decline of at least 3 points
  - HINE-2: Decline of at least 2 points on kicking or 1 point on any other milestone (excluding voluntary grasp)
  - CHOP-INTEND: Decline of at least 4 points
  - MFM32: Decline from baseline
  - BSID-III: Inability to sit without support for more than 5 seconds (item 22)
Member will not use Evrysdi and Spinraza (nusinersen) concomitantly
Submission of documentation for prior authorization including: genetic testing confirming deletion or mutation at SMN1 allele; baseline motor assessment using at least one accepted tool (HINE-2, CHOP-INTEND, HFMSE, MFM32, or BSID-III); medical records documenting respiratory status and need for respiratory support

Not Medically Necessary / Investigational

Evrysdi is considered investigational or not medically necessary for the following uses:

ANY of the following

Use in patients with SMA type 1, 2, or 3 who require permanent invasive ventilation
Concomitant use of Evrysdi and gene replacement therapy
Concomitant use of Evrysdi and Spinraza (nusinersen)

Provider Actions & Administrative Requirements

Prior Authorization

Prior authorization submission requirements

Submit genetic testing confirming SMN1 deletion or mutation, baseline motor assessments using at least one accepted tool (HINE-2, CHOP-INTEND, HFMSE, MFM32, or BSID-III), and medical records documenting respiratory status and need for respiratory support. For continuation requests, submit the most recent assessments within 90 days demonstrating clinical benefit (e.g., improvement or maintenance on the accepted motor assessments).

Documentation Required

Prescriber specialty requirement

This medication must be prescribed by or in consultation with a neurologist or neuromuscular specialist with expertise in the treatment of spinal muscular atrophy (SMA).

Billing Rule

Approval duration

Approvals will be for 12 months.

Billing Rule

Quantity limits and dosing

Quantity limits and dosing follow FDA-recommended, weight/age-based dosing specified in the policy. Tablet: quantity limit = 1 tablet per day (5 mg tablet dosing for patients 2 years and older and ≥20 kg: 5 mg once daily). Oral solution 60 mg bottle: quantity limit = 2 bottles (120 mg) per 24 days; oral solution FDA-recommended dosing provided by weight/age in the policy.

Clinical Evidence & References

Primary reference: Evrysdi [package insert], Genentech, Inc; October 2022. Coverage in this policy aligns with FDA labeling for Evrysdi as an oral treatment for SMA in pediatric and adult patients and is based on clinical guidelines and studies for SMA; the policy uses FDA-approved indications and evidence-based guidance to define initiation, continuation, dosing, prescriber, and documentation requirements.

Background & Definitions

This policy is based on the intent to ensure appropriate selection of patients for Evrysdi therapy using product labeling, clinical guidelines, and clinical studies in SMA. Patients are selected according to FDA labeling and documented genetic confirmation of SMN1 deletion or mutation, clinical diagnosis of SMA type 1–3, baseline and follow-up motor assessments, and respiratory status. The policy covers FDA-approved indications for pediatric and adult patients when the specified approval criteria are met and lists excluded uses such as treatment of patients requiring permanent invasive ventilation and concomitant use with certain other SMA therapies.

Definitions

TermEvrysdi

DefinitionRisdiplam, an oral therapy indicated for treatment of spinal muscular atrophy (SMA).

Clinical Thresholds

Clinical thresholds

HFMSE decline (prior gene therapy loss)>= 3-point decline

HINE-2 decline (prior gene therapy loss)>= 2 points on kicking or >=1 point on any other milestone (excluding voluntary grasp)

CHOP-INTEND decline (prior gene therapy loss)>= 4-point decline

BSID-III loss criterionInability to sit without support for more than 5 seconds on item 22

HFMSE improvement for continuation>= 3-point increase

CHOP-INTEND improvement for continuation>= 4-point increase

HINE-2 improvement (kicking) for continuation>= 2-point increase

BSID-III continuation milestoneAbility to sit without support for >= 5 seconds after 12 months

Revision History

2025-04reviewedLatest

Policy reviewed April 2025; no specific clinical policy changes flagged (material change = false).

2025-04revisedLatest

Policy revised April 2025; no specific clinical policy changes flagged (material change = false).

2022-09-09effective_date

Current effective date September 9, 2022 (review April 2025).