CurrentColorado Rocky Mountain Health PlansPolicy 2025 P 2217-7

Evrysdi (risdiplam) prior authorization / medical necessity

Prior authorization and medical necessity criteria for coverage of Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in pediatric and adult patients, including initial authorization, reauthorization, clinical documentation requirements, prescriber requirement, and authorization duration.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyEvrysdi (risdiplam) prior authorization / medical necessity

Policy CodePolicy 2025 P 2217-7

Change TypeCriteria added/revised for post-gene therapy decline

Effective DateJun 1, 2025

Next Review Date

Key ActionEvrysdi requires prior authorization under this medical necessity program; submit medical records confirming genetic diagnosis, baseline motor assessment, documentation of functional status and any decline after gene therapy when applicable; approvals issued for 12 months.

POLICY UPDATE CHANGES

Added criteria for patients that have documented decline from pretreatment baseline status following administration of gene replacement therapy.

Revised criteria for patients that have documented decline from pretreatment baseline status following administration of gene replacement therapy.

Revised prescriber requirement and updated Upper Limb Module to Revised Upper Limb Module test.

12Authorization duration

5Accepted baseline motor assessments

>=1

Genetic confirmation requirement (SMN1)

NoVentilation exclusion for eligibility

Coverage Summary

Scope: Prior authorization and medical necessity criteria for coverage of Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Coverage stance: Covered with criteria. Product/indication: Evrysdi (risdiplam), an SMN2 splicing modifier indicated for treatment of SMA in pediatric and adult patients. Authorization duration: 12 months. Prescriber requirement: prescribed by or in consultation with a neurologist with expertise in the treatment of SMA.

Key Authorization Parameters

Authorization duration12 months

Accepted baseline motor assessments5 (CHOP INTEND, HINE-2, HFMSE, RULM, MFM-32)

Genetic confirmation requirement (SMN1)>= 1 (homozygous deletion/mutation or compound heterozygous mutation of SMN1)

Ventilation exclusionNot dependent on ventilation (no invasive ventilation; no NIV beyond naps/night sleep)

Initial Authorization Criteria

Initial Authorization

Evrysdi will be approved based on all of the following criteria:

ALL of the following

Diagnosis of spinal muscular atrophy (SMA)
ALL of the following
- ONE of
  - Homozygous gene deletion or mutation of SMN1 gene (e.g., homozygous deletion of exon 7 at locus 5q13)
  - Compound heterozygous mutation of SMN1 gene (e.g., deletion of SMN1 exon 7 [allele 1] and mutation of SMN1 [allele 2])
ALL of the following
- NONE of

Reauthorization / Continuation Criteria

Reauthorization

Evrysdi will be approved for reauthorization based on all of the following criteria:

ALL of the following

ALL of the following
- ONE of the following assessment criteria
  - CHOP INTEND: Improvement or maintenance of previous improvement of at least a 4 point increase in score from pretreatment baseline OR patient has achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so>= 4 point increase from pretreatment baseline
  - HINE-2: One of the following: Improvement or maintenance of previous improvement of at least 2 point (or maximal score) increase in ability to kick; OR improvement or maintenance of previous improvement of at least 1 point increase in any other HINE-2 milestone (excluding voluntary grasp); OR the patient exhibited improvement, or maintenance of previous improvement in more HINE motor milestones than worsening (net positive improvement) from pretreatment baseline; OR patient has achieved and maintained any new motor milestones when they would otherwise be unexpected to do so

Unproven / Exclusions

Exclusions

ALL of the following

ALL of the following
- NONE of
  - Invasive ventilation
  - Use of non-invasive ventilation beyond use for naps and nighttime sleep
Patient is not receiving concomitant chronic survival motor neuron (SMN) modifying therapy (e.g., Spinraza (nusinersen))

Special rule for prior gene replacement therapy: the standard initial-authorization rule excludes patients who have previously received gene replacement therapy for SMA. However, an alternative pathway allows approval when the patient has previously received gene replacement therapy and the submission includes medical records documenting a clinically meaningful functional decline (e.g., loss of motor milestone) since receiving gene replacement therapy.

Applicable Codes

Diagnosis codes referencedICD-10

No codes listed

Provider Actions & Documentation Requirements

Prior Authorization

Prior authorization required

Evrysdi requires prior authorization under this medical necessity program; approval issued for 12 months. Submission must include medical records confirming genetic diagnosis, baseline assessment by specified motor scales, and documentation of functional status and any decline after gene therapy if applicable.

Genetic diagnosis (SMN1 homozygous deletion/mutation or compound heterozygous mutation)
Baseline motor assessment (CHOP INTEND, HINE-2, HFMSE, RULM, or MFM-32)
Documentation of functional status and any clinically meaningful decline after gene replacement therapy, if applicable

Documentation Required

Genetic confirmation required

Submit medical records confirming SMN1 homozygous deletion/mutation OR compound heterozygous mutation of SMN1 (chromosome 5q) as part of initial authorization.

Clinical Evidence & References

Evidence: P&T approval dates: 9/2020, 9/2021, 7/2022, 8/2023, 7/2024, 12/2024, 4/2025. Key reference: Evrysdi [package insert], Genentech, Inc; September 2024.

Background

Background: Evrysdi is an SMN2 splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. The policy establishes prior authorization and medical necessity criteria that require genetic confirmation of SMN1 mutation/deletion, baseline motor function assessment using specified scales, exclusion of chronic concomitant SMN-modifying therapies, and special rules regarding patients previously treated with gene replacement therapy (including documentation of decline to qualify). The program issues authorizations for 12 months and requires prescribing by or in consultation with a neurologist experienced in SMA.

Definitions: Baseline assessments <2 months: Baseline assessments for patients less than 2 months of age requesting Evrysdi are not necessary; initial assessments shortly post-therapy can serve as baseline.

Revision History

2025-04addedLatest

Added criteria for patients that have documented decline from pretreatment baseline status following administration of gene replacement therapy.

2025-04revised

Revised criteria for patients that have documented decline from pretreatment baseline status following administration of gene replacement therapy.

2024-12revised

Revised prescriber requirement and updated Upper Limb Module to Revised Upper Limb Module test.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyEvrysdi (risdiplam) prior authorization / medical necessity

Policy CodePolicy 2025 P 2217-7

Change TypeCriteria added/revised for post-gene therapy decline

Effective DateJun 1, 2025

Next Review Date

On This Page

Invasive ventilation
Use of non-invasive ventilation beyond use for naps and nighttime sleep

Patient is not receiving concomitant chronic survival motor neuron (SMN) modifying therapy (e.g., Spinraza (nusinersen))

ALL of the following

ONE of
- Patient has not previously received gene replacement therapy for the treatment of SMA (e.g., Zolgensma (onasemnogene abeparvovec-xioi))
- OR (if previously received gene replacement therapy)
  - Submission of medical records documenting a clinically meaningful functional decline (e.g., loss of motor milestone) since receiving gene replacement therapy

ALL of the following

CHOP INTEND (Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders)
HINE-2 (Hammersmith Infant Neurological Exam Part 2)
HFMSE (Hammersmith Functional Motor Scale Expanded)
RULM (Revised Upper Limb Module Test)
MFM-32 (Motor Function Measure 32 Scale)

Prescribed by or in consultation with a neurologist with expertise in the treatment of SMA

HINE-2: kick >= 2 point (or maximal), other milestones >= 1 point

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

RULM: Improvement or maintenance of previous improvement of at least a 2 point increase in score from pretreatment baseline OR patient has achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so>= 2 point increase from pretreatment baseline

MFM-32: Improvement or maintenance of previous improvement of at least a 3 point increase in score from pretreatment baseline OR patient has achieved and maintained any new motor milestone from pretreatment baseline when they would otherwise be unexpected to do so>= 3 point increase from pretreatment baseline

ALL of the following

NONE of
- Invasive ventilation
- Use of non-invasive ventilation beyond use for naps and nighttime sleep

Patient is not receiving concomitant chronic survival motor neuron (SMN) modifying therapy (e.g., Spinraza (nusinersen))

Prescribed by or in consultation with a neurologist with expertise in the treatment of SMA

Reauthorization Numeric Thresholds

CHOP INTEND improvement>= 4 point increase from pretreatment baseline

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

HINE-2 other milestone improvement>= 1 point increase

HFMSE improvement>= 3 point increase from pretreatment baseline

RULM improvement>= 2 point increase from pretreatment baseline

MFM-32 improvement>= 3 point increase from pretreatment baseline

Documentation Required

Baseline and follow-up motor assessments

For initial authorization submit at least one baseline motor exam (CHOP INTEND, HINE-2, HFMSE, RULM, or MFM-32). For reauthorization submit most recent results documenting improvement versus pretreatment baseline per the specific thresholds.

Accepted baseline/follow-up scales: CHOP INTEND
HINE-2
HFMSE
RULM (Revised Upper Limb Module)
MFM-32

Documentation Required

Gene therapy history and decline documentation

If patient previously received gene replacement therapy (e.g., Zolgensma), submit records documenting clinically meaningful functional decline (e.g., loss of motor milestone) since receiving gene replacement therapy to qualify for Evrysdi.

Billing Rule

Authorization duration

Authorizations will be issued for 12 months.