CurrentAnthemPolicy CC-0152

Vyondys 53 (golodirsen) — Clinical Coverage Criteria

Clinical coverage criteria for the medical benefit use of Vyondys 53 (golodirsen) to treat Duchenne muscular dystrophy (DMD) with mutations amenable to exon 53 skipping; applies to prior authorization and continuation determinations.

Policy Summary

PayerAnthem

PolicyVyondys 53 (golodirsen) — Clinical Coverage Criteria

Policy CodePolicy CC-0152

Change TypeReviewed — no material changes

Effective DateN/A

Next Review DateAug 15, 2025

Key ActionObtain prior authorization with documentation of diagnosis, CLIA-confirmed genetic test showing mutation amenable to exon 53 skipping, age 6–15 years, corticosteroid use, and baseline functional measures (6MWT, NSAA or Gowers time).

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

30 mg/kg weeklyrecommended dosing

6 monthsapproval duration

6–15 yearsage range

J1429HCPCS

Coverage Criteria for Vyondys 53 (golodirsen)

Initial Therapy

Covered when ALL of the following are met:

Initial Approval Criteria: Diagnosis of Duchenne muscular dystrophy (DMD)

Documented diagnosis required.

Initial Approval Criteria: Genetic mutation amenable to exon 53 skipping documented by a CLIA‑accredited laboratory

Genetic test result must confirm amenability to exon 53 skipping.

Initial Approval Criteria: Age 6-15 years

Matches study inclusion (NCT02310906, Study 4053-101).

Initial Approval Criteria:

Policy Summary

PayerAnthem

PolicyVyondys 53 (golodirsen) — Clinical Coverage Criteria

Policy CodePolicy CC-0152

Change TypeReviewed — no material changes

Effective DateN/A

Next Review DateAug 15, 2025

SourceLink

On This Page

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Continuation Therapy

Covered when ALL of the following are met:

Continuation Criteria: Initial criteria were met at therapy initiation

Documentation of meeting all initial approval elements at treatment start is required.

Continuation Criteria: Patient remains ambulatory (with or without assistive device)

Ambulatory status must be documented (may include cane or walker).

Approval duration: 6 months

Reauthorization requests should include updated documentation of ambulatory status and ongoing benefit.

Coding and Clinical Thresholds

HCPCSHCPCS

J1429

Injection, golodirsen 10 mg [Vyondys 53]

ICD-10 DiagnosisICD-10

G71.01

Duchenne or Becker muscular dystrophy

inv-07: 6-Minute Walk Test (6MWT)

6MWT threshold6-minute walk test (6MWT) ≥ 250 m

ContextRequired at baseline for initial approval per Study 4053-101 (NCT02310906)

Use for continuationBaseline 6MWT documented; ambulatory status must be maintained for continuation

inv-08: NorthStar Ambulatory Assessment (NSAA)

NSAA thresholdNorthStar Ambulatory Assessment (NSAA) total > 17

ContextOne of two functional criteria required for initial approval (per Study 4053-101)

DocumentationNSAA total must be provided in prior authorization request

inv-09: Rise (Gowers) time

Gowers (rise) time thresholdRise (Gowers) time < 7 seconds

ContextAlternative functional criterion to NSAA for initial approval (per Study 4053-101)

DocumentationGowers time must be provided in prior authorization request

Prior Authorization, Documentation, and Monitoring Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Vyondys 53 (golodirsen). Submit clinical documentation demonstrating that all coverage criteria are met. Prior authorization approvals are time-limited (see policy) and may require renewal with supporting records.

Submit genetic testing results from a CLIA-certified laboratory confirming a mutation amenable to exon 53 skipping (methodology that evaluates all exons: e.g., MLPA, comparative genomic hybridization, single-condition amplification/internal primer analysis).
Provide documentation of diagnosis of Duchenne muscular dystrophy (DMD) and patient age (must be 6–15 years at initiation).
Provide evidence of corticosteroid use.
Provide baseline and recent functional assessments: 6MWT ≥ 250 m, and either NSAA total > 17 or Gowers (rise) time < 7 seconds.
For continuation requests, document prior approval criteria were met at initiation and that the patient remains ambulatory (with or without assistive device).
Include baseline renal monitoring: 24-hour urine collection to measure GFR prior to initiation; monthly proteinuria monitoring and serum cystatin C every 3 months per label. If proteinuria ≥ 2+ or elevated cystatin C, submit 24-hour urine quantification and GFR assessment.

Denial Risk

Concurrent Exon-Skipping Agents Contraindicated

Concurrent use of other exon-skipping agents for DMD is not allowed and is a basis for denial of the request.

Do not combine Vyondys 53 with other exon-skipping therapies (e.g., eteplirsen/Exondys 51, casimersen/Amondys 45).
Requests showing concurrent or recent use of another exon-skipping agent will be denied.

Documentation Required

Required Documentation (Genetic, Diagnosis, Monitoring Records)

Provide required documentation to support the medical necessity of Vyondys 53. Complete records reduce delays and help avoid denials.

Genetic testing report from a CLIA-certified laboratory confirming an exon 53–amenable mutation (report must show methodology and exon-level analysis).
Clinical documentation confirming DMD diagnosis, patient age, and documentation of corticosteroid therapy.
Baseline and recent functional testing results: 6-minute walk test (6MWT) with distance (must be ≥ 250 m), NSAA total score, and Gowers (rise) time as applicable.
Renal monitoring: baseline 24-hour urine GFR collection prior to initiation; monthly urine protein checks; serum cystatin C every 3 months; if proteinuria 2+ or greater or elevated cystatin C, submit 24-hour urine protein quantification and GFR reassessment.
Records of prior authorization approvals (for continuation requests) showing initial criteria were met and documentation of continued ambulatory status.

Denial Risk

Criteria-Based Denials — Common Reasons

Common reasons for denial include failure to meet age or diagnostic criteria, missing genetic confirmation, lack of required functional thresholds, not being on corticosteroid therapy, or concurrent use of other exon-skipping agents.

Wrong or unconfirmed diagnosis (not DMD).
Genetic testing absent or not from a CLIA-certified laboratory, or does not demonstrate exon 53–amenable mutation.
Patient age outside the required 6–15 years range at initiation.
Not currently using a corticosteroid as required by criteria.
Functional measures below thresholds (6MWT < 250 m; NSAA ≤ 17; Gowers time ≥ 7 seconds).
Concurrent use of another exon-skipping agent (e.g., Exondys 51, Amondys 45).
Missing baseline renal monitoring (no pre-initiation 24-hour urine GFR) or absent ongoing renal surveillance per label.

OpenPayer

Vyondys 53 (golodirsen) — Clinical Coverage Criteria

Coverage Criteria for Vyondys 53 (golodirsen)

Coding and Clinical Thresholds

Prior Authorization, Documentation, and Monitoring Requirements

Background on Duchenne Muscular Dystrophy and Therapy

Key Definitions