Clinical Policy: Golodirsen (Vyondys 53)
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Defines medical necessity and prior authorization criteria for golodirsen (Vyondys 53) for treatment of Duchenne muscular dystrophy (DMD) in Medicaid members whose DMD mutations are amenable to exon 53 skipping.
Changed six-minute walk test (6MWT) criterion from 250 meters to 180 meters to align with Department of Community Health request.
Changed left ventricular ejection fraction (LVEF) criterion from ≥50% to ≥40% to align with Department of Community Health request.
Added criterion that member has not previously received gene replacement therapy for DMD (e.g., Elevidys).
Added Agamree (vamorolone) to list of corticosteroid options in Appendix B and to corticosteroid requirements.
Updated Appendix C to add contraindication of serious hypersensitivity to golodirsen or inactive ingredients per prescribing information.
Coverage Criteria
inv-01: Initial Therapy
Covered when ALL of the following are met:
inv-02: Continuation Therapy
Covered when ALL of the following are met:
inv-03: Modified Coverage Criteria
Policy was updated with state-requested numeric thresholds and exclusions
Numeric thresholds and exclusions were revised to align with Department of Community Health requests; see policy history for revision dates.
Diagnoses or indications that are not specifically listed under the authorized coverage section are not covered. For diagnoses not listed, refer to the appropriate off-label use policy for the member's line of business (for example, CP.CPA.09 for commercial, HIM.PA.154 for marketplace, and CP.PMN.53 for Medicaid).
Coverage is excluded for members who have previously received gene replacement therapy for Duchenne muscular dystrophy (for example, Elevidys).
Use of Vyondys 53 (golodirsen) for indications other than Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 53 skipping, or use when the policy's clinical criteria are not met, is not authorized. The indication for Vyondys 53 is limited and was approved under accelerated approval based on increased dystrophin production; continued approval depends on confirmatory trial results.
Use that is inconsistent with the numeric thresholds adopted per the Department of Community Health (for example, a six-minute walk test < 180 meters or an ejection fraction <= 40%) does not meet the updated Georgia-specific coverage criteria and would not be authorized.
Initial Therapy Criteria
inv-26: Initial Therapy — genetic confirmation, age limit, neurologist involvement, recent objective assessments, corticosteroid requirements
Covered when ALL of the following are met:
inv-27: Initial Therapy — state-aligned functional thresholds and therapy history exclusions
Covered when ALL of the following are met:
These state-aligned thresholds and exclusions were added or revised per Department of Community Health requests.
Continuation Criteria
inv-28: Continued Therapy — requires demonstration of stability or improvement and regular neurologist follow-up
Continued use requires demonstration of stability or improvement and regular neurologist follow-up.
inv-29: Continuation Therapy — annual reviews noted; Appendix updates
Covered when ALL of the following are met:
Coding
| J1429 | Injection, golodirsen, 10 mg |
| J1429 | Injection, golodirsen, 10 mg |
Provider Actions & Requirements
Prior authorization required for golodirsen (J1429)
Prior authorization is required for golodirsen (J1429).
PA must document Georgia-specific clinical criteria with J1429
Submit prior authorization requests using HCPCS J1429 for golodirsen and ensure the member meets Georgia-specific clinical criteria (including 6MWT ≥ 180 m and LVEF ≥ 40%) and other policy requirements before approval.
Corticosteroid trial required before and during golodirsen
Members must have had adherent oral corticosteroid therapy for ≥ 6 months (e.g., prednisone, Emflaza, Agamree/vamorolone) unless contraindicated; Vyondys 53 must be prescribed concurrently with an oral corticosteroid unless contraindicated.
- Trial duration: ≥ 6 months of adherent use unless contraindicated or intolerant
- Examples of corticosteroids listed: prednisone, Emflaza (deflazacort), Agamree (vamorolone)
- Vyondys 53 concurrent prescribing with oral corticosteroid required unless contraindicated
Verify clinical entry criteria and prior therapy exclusions; no multi-step algorithm
Confirm the member meets the initial clinical entry criteria (genetic confirmation of exon 53-amenable DMD, age ≤15, neurologist involvement, recent 6MWT ≥ 180 m, LVEF ≥ 40%, FVC ≥ 50% predicted) and note exclusions such as prior gene replacement therapy; no multi-step sequencing beyond the corticosteroid requirement is specified.
- Do not approve if member previously received gene replacement therapy (e.g., Elevidys)
- Ensure prescribing neurologist involvement and recent objective assessments within 30 days
Submit supporting clinical documentation with PA request
Provider must submit supporting documentation (office chart notes, lab results, or other clinical information) showing the member meets all approval criteria as part of the authorization request.
- Include recent 6MWT, LVEF, and FVC results when applicable
- Include genetic testing confirming exon 53–amenable mutation
Submit medical necessity and adherence evidence
Provide documentation demonstrating medical necessity per the clinical policy and applicable coverage documents, including evidence of adherence to required corticosteroid therapy and objective measures of response or stability.
- Document adherent corticosteroid use for ≥ 6 months or contraindication
- Document objective assessments (6MWT, LVEF, FVC) and neurologist follow-up
Incomplete documentation or absent medical director review may cause denial
Failure to submit required documentation showing the member meets all approval criteria, or lack of medical director review when requested, may result in denial of the request.
- All requests may require medical director review
- Missing office notes, labs, or genetic confirmation can trigger denial
Coding (J1429) on claims does not ensure coverage — use correct, current codes
Inclusion of HCPCS J1429 or other codes in a claim does not guarantee coverage; providers must use appropriate, up-to-date coding guidance and follow the policy criteria when submitting claims.
- J1429 = Injection, golodirsen, 10 mg
- Reference current professional coding guidance prior to claim submission
Step Therapy
| Step | Requirement | Duration / Notes |
|---|---|---|
| 1 | Trial of adherent oral corticosteroid therapy (examples listed: prednisone, deflazacort/Emflaza, vamorolone/Agamree) | ≥ 6 months unless contraindicated or clinically significant adverse effects; Vyondys 53 must be prescribed concurrently with an oral corticosteroid unless contraindicated |
| Step | Policy statement | Notes |
|---|---|---|
| 1 | No multi-step therapy algorithm is described in this policy; requirements focus on corticosteroid trial and specified exclusions (e.g., prior gene replacement therapy such as Elevidys) | Appendix B lists corticosteroid options (prednisone, Emflaza (deflazacort), Agamree (vamorolone)); member must not have previously received gene replacement therapy |
Quantity Limits
Definitions
Background
Golodirsen (Vyondys 53) is an antisense oligonucleotide indicated under accelerated approval for the treatment of Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation amenable to exon 53 skipping. The approval was based on increased dystrophin production in skeletal muscle; verification of clinical benefit is pending confirmatory trials.
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