ModifiedMass General Brigham Health PlanPolicy N/A

Evrysdi (risdiplam)

Defines prior authorization, coverage criteria, continuation criteria, dosing limits, specialty pharmacy requirement, and quantity limits for Evrysdi (risdiplam) under the commercial/pharmacy benefit.

Policy Summary

PayerMass General Brigham Health Plan

PolicyEvrysdi (risdiplam)

Policy CodePolicy N/A

Change TypeMaterial modification: criteria revised and quantity limits added

Effective DateJul 1, 2025

Next Review Date

Key ActionObtain prior authorization and ensure prescription is by or in consultation with a neurologist specializing in SMA, with required genetic testing and documentation of ventilation status.

SourceLink

POLICY UPDATE CHANGES

Updated initial criteria to remove requirement that member is at least 2 years and less than 25 years of age at initiation; required prescription by or in consultation with an SMA-specialist neurologist; included dosing for members less than 2 months of age.

Added quantity limit for Evrysdi tablet (30 tablets per 30 days).

Also added quantity limit for Evrysdi tablet.

3Covered SMA types

12 moApproval length (initial/reauth)

120 mL/30dSolution quantity limit

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Tablet quantity limit

Coverage Summary

Coverage stance: covered_with_criteria for Evrysdi (risdiplam). Scope: Defines prior authorization, coverage criteria, continuation criteria, dosing limits, specialty pharmacy requirement, and quantity limits for Evrysdi (risdiplam) under the commercial/pharmacy benefit. Initial/Reauthorization approval length: 12 months. Effective date: 2025-07-01. Last review date: 2025-04-09.

Key policy facts

Covered SMA typesType 1, Type 2, Type 3

Approval length12 months

Solution quantity limit120 mL per 30 days (0.75 mg/mL oral solution)

Tablet quantity limit30 tablets per 30 days

Prior authorizationRequired under the pharmacy benefit; specialty pharmacy dispensing

Specialist requirementPrescribed by or in consultation with a neurologist specializing in SMA

Initial Authorization Criteria

Initial Authorization - Covered when ALL criteria are met

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

ALL of the following

Documented diagnosis of type 1, type 2, or type 3 spinal muscular atrophy (SMA)
Documentation of genetic testing confirming 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote
Specialist prescriber: Prescribed by or in consultation with a neurologist specializing in the treatment of spinal muscular atrophy
Not ventilator-dependent

Continuation of Therapy / Reauthorization Criteria

Continuation of Therapy / Reauthorization - Covered when ALL criteria are met

Requests for reauthorization will be approved when the member meets ALL initial criteria AND the following criteria are met:

ALL of the following

Submission requirement: Submission of medical records of the most recent (less than 1 month prior to continuation request) assessment documenting a positive clinical response from pretreatment baseline to Evrysdi therapy
Examples: chart notes, laboratory values
Positive clinical response demonstrated by at least ONE of the following assessments
- HINE-2
  - HINE-2 kick: Improvement or maintenance of previous improvement of at least a 2 point (or maximal score) increase in ability to kick

Provider Actions & Billing Requirements

Prior Authorization

Prior authorization required

Prior authorization is required for Evrysdi under the pharmacy benefit; approvals may be reviewed for members new to the plan within the past 90 days who are currently receiving treatment.

Documentation Required

Genetic testing documentation required

Provide documentation of genetic testing confirming 5q SMA homozygous gene mutation, homozygous gene deletion, or compound heterozygote.

Documentation Required

Specialist involvement required

Prescription must be by or in consultation with a neurologist specializing in SMA.

Applicable Codes & Quantity Limits

Benefit / Billingmixed

No codes listed

Quantity LimitsNDC|mixedCovered

Evrysdi 0.75mg/mL (80mL) oral solution	Quantity limit: 120 mL per 30 days
Evrysdi tablet	Quantity limit: 30 tablets per 30 days

This medication is designated specialty and must be filled at a contracted specialty pharmacy. Quantity limits: Evrysdi 0.75 mg/mL oral solution — 120 mL per 30 days; Evrysdi tablet — 30 tablets per 30 days. Ensure the member's daily dose does not exceed the specified age/weight-based maximums; claims exceeding dose limits may be denied.

Clinical Evidence & References

Evidence: Prescribing information: Evrysdi (risdiplam) [prescribing information], Genentech, February 2025.

Background & Definitions

Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SMA is a genetic disorder caused by loss or dysfunction of the SMN1 gene resulting in loss of motor neurons and progressive muscle weakness and wasting.

Term	Definition
5q SMA
SMA caused by mutation, deletion, or compound heterozygote in the SMN1 gene located on chromosome 5q.
HINE-2
Hammersmith Infant Neurological Examination, Section 2 (motor milestones) used to assess motor function in infants.
HFMSE
Hammersmith Functional Motor Scale, Expanded - motor function assessment for SMA.
CHOP-INTEND
Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders - motor function assessment for infants with SMA.
MFM32
Motor Function Measure 32-item scale for neuromuscular disease.

Revision History

04/09/2025policy_revisionLatest

Updated initial criteria to remove the prior age requirement (at least 2 years and less than 25 years at initiation), required prescription by or in consultation with a neurologist specializing in SMA, and included dosing for members less than 2 months of age. Added quantity limit for Evrysdi tablet (30 tablets per 30 days). Effective 07/01/2025.

07/01/2025quantity_limit_addedLatest

Specified quantity limits: Evrysdi 0.75 mg/mL (80 mL) oral solution — 120 mL per 30 days; Evrysdi tablet — 30 tablets per 30 days.

Policy Summary

PayerMass General Brigham Health Plan

PolicyEvrysdi (risdiplam)

Policy CodePolicy N/A

Change TypeMaterial modification: criteria revised and quantity limits added

Effective DateJul 1, 2025

Next Review Date

Key ActionObtain prior authorization and ensure prescription is by or in consultation with a neurologist specializing in SMA, with required genetic testing and documentation of ventilation status.

SourceLink

On This Page

Member is not dependent on invasive ventilation or tracheostomy

Member is not dependent on non-invasive ventilation beyond naps and nighttime sleep

Member meets ONE of the following

Member has not previously received gene therapy for SMA
Post-gene therapy worsening
- HINE-2 decline: HINE-2: Decline of at least 2 points on kicking and 1 point on any other milestone (excluding voluntary grasp)Decline of at least 2 points on kicking and 1 point on any other milestone (excluding voluntary grasp)
- HFMSE decline: HFMSE: Decline of at least 3 pointsDecline of at least 3 points
- CHOP-INTEND decline: CHOP-INTEND: Decline of at least 4 pointsDecline of at least 4 points

Concomitant therapy: Member will not use Evrysdi and Spinraza concomitantly

Dose limits

<2 months: Member less than 2 months of age: 0.15 mg/kg (daily)0.15 mg/kg (daily)
2 months to <2 years: Members 2 months to less than 2 years of age: 0.2 mg/kg (daily)0.2 mg/kg (daily)
>=2 years <20 kg: Members 2 years of age and older weighing less than 20 kg: 0.25 mg/kg (daily)0.25 mg/kg (daily)
>=2 years >=20 kg: Members 2 years of age and older weighing 20 kg or more: 5 mg (daily)5 mg (daily)

>= 2-point increase (or maximal score)

HINE-2 other: Improvement or maintenance of previous improvement of at least a 1 point (or maximal score) increase in any other HINE-2 milestone (excluding voluntary grasp)>= 1-point increase (or maximal score)

HINE-2 net: Improvement or maintenance of previous improvement in more HINE-2 motor milestones than worsening (net positive improvement)

HINE-2 new milestones: Achieved and maintained any new motor milestones when they would otherwise be unexpected to do so (e.g., sit or stand unassisted, walk)

HFMSE

HFMSE increase: Improvement or maintenance of previous improvement of at least a 3-point increase in score>= 3-point increase
HFMSE new milestones: Achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so

CHOP-INTEND

CHOP-INTEND increase: Improvement or maintenance of previous improvement of at least a 4-point increase in score>= 4-point increase
CHOP-INTEND new milestones: Achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so

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

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

Continuation & post-gene-therapy thresholds

HINE-2 continuation (kick)>= 2-point increase (or maximal score)

HINE-2 continuation (other milestones)>= 1-point increase (or maximal score) excluding voluntary grasp

HFMSE continuation>= 3-point increase

CHOP-INTEND continuation>= 4-point increase

Post-gene-therapy decline (HINE-2)Decline of at least 2 points on kicking and 1 point on any other milestone (excluding voluntary grasp)

Post-gene-therapy decline (HFMSE)Decline of at least 3 points

Post-gene-therapy decline (CHOP-INTEND)Decline of at least 4 points

Documentation Required

Ventilation status documentation

Document that member is not dependent on invasive ventilation/tracheostomy and not dependent on non-invasive ventilation beyond naps and nighttime sleep.

Documentation Required

Clinical assessments for continuation

For reauthorization submit medical records within 1 month documenting positive clinical response using validated scales (HINE-2, HFMSE, CHOP-INTEND, MFM32) or stabilization/improvement post-gene therapy.

Billing Rule

Specialty pharmacy dispensing

Medication designated specialty and must be filled at a contracted specialty pharmacy.

Billing Rule

Dose limit enforcement

Ensure daily dose does not exceed age/weight-based maximums specified in the policy; claims exceeding limits may be denied.

03/17/2021policy_creation

Policy created and reviewed at March P&T. Effective 05/01/2021.