CurrentFidelis CarePolicy NY.CP.PHAR.453

Golodirsen (Vyondys 53) Coverage

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Governs prior authorization and medical necessity criteria for golodirsen (Vyondys 53) for treatment of Duchenne muscular dystrophy patients with DMD gene mutations amenable to exon 53 skipping for Fidelis Care (NY Medicaid and HIM lines of business). Applies to providers requesting coverage.

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CurrentFidelis CarePolicy NY.CP.PHAR.453

Golodirsen (Vyondys 53) Coverage

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Policy Summary

PayerFidelis Care

PolicyGolodirsen (Vyondys 53) Coverage

Policy CodePolicy NY.CP.PHAR.453

Change TypeNo material change

Effective DateMar 1, 2020

Next Review Date

Key ActionProvider must submit clinical chart notes, lab results and claims demonstrating diagnosis of DMD amenable to exon 53, baseline kidney function testing, and stable corticosteroid dosing unless contraindicated.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

J1429HCPCS code for golodirsen (10 mg)

6 monthsRenewal period

Kidney functionBaseline lab

30 mg/kg wklyFDA dosing for DMD

No concurrent exon-skippingConcurrent therapy exclusion

Coverage Criteria for Golodirsen (Vyondys 53) and Related Therapies

Initial Approval — Duchenne Muscular Dystrophy

Covered when ALL of the following are met

Initial DMD criteria: 1. Diagnosis of Duchenne muscular dystrophy (DMD) with confirmed mutation of the DMD gene that is amenable to exon 53; 2. Documentation indicates kidney function testing prior to starting therapy; 3. Documentation stating the member is on a stable dose of corticosteroids prior to starting therapy unless contraindicated or intolerant; 4. Vyondys 53 is not prescribed concurrently with other exon‑skipping therapies (e.g., Exondys 51, Viltepso); 5. Dosing does not exceed FDA labeling.

All five elements must be documented in clinical chart notes, laboratory reports, medical claims and/or pharmacy claims.

Continuation Therapy — Duchenne Muscular Dystrophy

Covered for continuation when ALL of the following are met

Continued therapy criteria: a. Member will continue to receive concurrent corticosteroids unless contraindicated or intolerant; b. Vyondys 53 is not prescribed concurrently with other exon‑skipping therapies (e.g., Exondys 51, Viltepso); c. Dosing does not exceed FDA labeling.

Approval duration: 6 months.

Initial Approval Criteria

Covered when ALL of the following are met:

Initial Approval - DMD: 1. Diagnosis of Duchenne muscular dystrophy with confirmed DMD gene mutation amenable to exon 51; 2. Member is on a stable dose of corticosteroids prior to starting therapy unless contraindicated or intolerant; 3. Exondys 51 is not prescribed concurrently with other exon‑skipping therapies (e.g., Vyondys 53, Viltepso); 4. Dosing does not exceed FDA labeling.

Approval duration: 6 months.

Continuation Criteria

Covered when ALL of the following are met:

Continuation Therapy - DMD: a. Member will continue to receive concurrent corticosteroids unless contraindicated or intolerant; b. Exondys 51 is not prescribed concurrently with other exon‑skipping therapies (e.g., Vyondys 53, Viltepso); c. Dosing does not exceed FDA labeling.

Approval duration: 6 months.

Initial Approval

Covered when ALL of the following are met:

Initial Approval for DMD: 1) Documentation of Duchenne muscular dystrophy with confirmed DMD gene mutation amenable to exon 45; 2) Documentation indicates kidney function testing prior to starting therapy; 3) Documentation stating the member is on a stable dose of corticosteroids prior to starting therapy unless contraindicated or intolerant; 4) Amondys 45 is not prescribed concurrently with other exon‑skipping therapies (e.g., Vyondys 53, Exondys 51, Viltepso); 5) Dosing does not exceed FDA labeling.

All listed elements must be present and documented in clinical chart notes, laboratory reports, medical claims and/or pharmacy claims.

Continuation Criteria

Covered for continuation when ALL of the following are documented:

Continued Therapy for DMD: a) Member will continue to receive concurrent corticosteroids unless contraindicated or intolerant; b) Amondys 45 is not prescribed concurrently with other exon‑skipping therapies (e.g., Vyondys 53, Exondys 51, Viltepso); c) Dosing does not exceed FDA labeling.

Approval duration: 6 months.

Initial Approval — Viltepso

Covered when ALL of the following are met

Viltepso Initial Approval (DMD amenable to exon 53): 1. Diagnosis of Duchenne muscular dystrophy (DMD) with confirmed DMD gene mutation amenable to exon 53; 2. Documentation of kidney function testing prior to starting therapy; 3. Documentation member is on a stable dose of corticosteroids prior to starting therapy unless contraindicated or intolerant; 4. Viltepso is not prescribed concurrently with other exon‑skipping therapies (e.g., Exondys 51, Vyondys 53); 5. Dosing does not exceed FDA labeling.

All items must be documented in clinical chart notes, laboratory reports, medical or pharmacy claims. Continued FDA approval may be contingent on confirmatory trials.

Initial Therapy

Covered when ALL of the following are met for Duchenne Muscular Dystrophy:

DMD Initial Criteria: 1) Diagnosis of DMD with confirmed DMD gene mutation amenable to exon 53; 2) Documentation of kidney function testing prior to starting therapy; 3) Member on stable dose of corticosteroids prior to starting therapy unless contraindicated or intolerant; 4) Viltepso not prescribed concurrently with other exon‑skipping therapies (e.g., Exondys 51, Vyondys 53); 5) Dosing does not exceed FDA labeling.

All five conditions required for initial approval and must be documented in chart notes, labs, medical or pharmacy claims.

Continuation Therapy

Continued coverage when ALL of the following are documented:

DMD Continued Therapy: 1) Member will continue to receive concurrent corticosteroids unless contraindicated or intolerant; 2) Viltepso not prescribed concurrently with other exon‑skipping therapies (e.g., Exondys 51, Vyondys 53); 3) Dosing does not exceed FDA labeling.

All three conditions required for continued approval.

Coverage is not authorized for indications that are not FDA‑approved and are not specifically addressed in this policy, unless the request is supported by sufficient documentation of efficacy and safety consistent with the applicable off‑label use policies (HIM.PA.154 for health insurance marketplace, CP.PMN.53 for Medicaid) or by the subscriber contract. Requests for non‑FDA indications lacking that documentation risk denial.

Non‑FDA approved indications that are not addressed in this policy are not authorized unless the provider supplies sufficient clinical evidence of efficacy and safety in accordance with the off‑label use policies (HIM.PA.154; CP.PMN.53) or applicable contract terms. Such documentation should be submitted with the prior authorization request.

Requests for coverage of non‑FDA approved indications without adequate documentation of efficacy and safety are not authorized. Providers must follow the off‑label use policies (HIM.PA.154, CP.PMN.53) and submit supporting clinical information if seeking coverage for an off‑label use.

Viltepso must not be prescribed concurrently with other exon‑skipping therapies (for example, Exondys 51™ or Vyondys 53™). Concurrent exon‑skipping therapy is a trigger for denial and must be avoided; documentation showing no concurrent exon‑skipping agents is required with the request.

Non‑FDA approved indications that are not specifically addressed by this policy are not authorized unless the provider presents sufficient documentation of efficacy and safety per the applicable off‑label use policies or subscriber contract provisions.

Vyondys 53 was approved under the FDA’s accelerated approval pathway based on an increase in dystrophin production observed in treated patients. Because clinical benefit has not yet been verified, continued approval may be contingent upon confirmatory trials; this uncertainty should be considered when evaluating requests and when advising members.

Although eteplirsen and similar exon‑skipping therapies increase dystrophin levels, the clinical significance is unknown. The Institute for Clinical and Economic Review (ICER) concluded current evidence is insufficient to demonstrate net clinical benefit when these agents are added to corticosteroids and supportive care; therefore, evidence for dystrophin increase alone is considered insufficient to establish clinical effectiveness.

Requests for therapy where the patient’s mutation is not amenable to the specified exon skipping (for example, exon 45 for Amondys 45) or where proposed dosing would exceed FDA‑labeled recommendations do not meet medical necessity. Documentation must confirm the mutation is appropriate for the exon targeted and dosing must remain within FDA labeling.

Viltepso was approved based on an observed increase in dystrophin in skeletal muscle, but it is unknown whether that increase translates to a clinical benefit. Continued FDA approval may depend on confirmatory trials; prior authorization decisions should reflect the current uncertainty and require documentation described in the policy (diagnosis, kidney function testing, corticosteroid stability, absence of concurrent exon‑skipping therapy, and dosing within FDA labeling).

Coverage for non‑FDA approved indications that lack sufficient documentation of efficacy and safety, per the off‑label use policies (HIM.PA.154, CP.PMN.53) or subscriber contract, will be considered not authorized. Providers seeking coverage for off‑label uses must submit the required supporting evidence.

Coding and Product Information

Prior Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Vyondys 53, Exondys 51, Amondys 45, and Viltepso. Provider must submit documentation (such as office chart notes, laboratory reports, medical and/or pharmacy claims) supporting that the member meets all approval criteria for initial and continued therapy. All requests reviewed under this policy may require medical director review; lack of required documentation (for example, documentation confirming DMD mutation amenable to the specified exon, baseline kidney function testing, or stable corticosteroid use) may result in denial.

Submit clinical chart notes, lab results, and medical/pharmacy claims to demonstrate all approval criteria are met.
All requests may be escalated for medical director review.
Approval durations where specified: typically 6 months for continuation of exon‑skipping therapies.

Prior Authorization

Viltepso Prior Authorization Requirements

For Viltepso specifically, initial approval requires documentation of: Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 53; kidney function testing prior to starting therapy; documentation that the member is on a stable dose of corticosteroids prior to starting therapy unless contraindicated or intolerant; Viltepso is not prescribed concurrently with other exon‑skipping therapies (e.g., Exondys 51™, Vyondys 53™); and dosing that does not exceed FDA labeling. Continued therapy requires documentation that the member will continue concurrent corticosteroids unless contraindicated or intolerant, Viltepso is not prescribed concurrently with other exon‑skipping therapies, and dosing does not exceed FDA labeling.

Background and Context

Background: Golodirsen (Vyondys 53) is an antisense oligonucleotide indicated for treatment of Duchenne muscular dystrophy in patients with confirmed DMD gene mutations amenable to exon 53 skipping. The approval is under the FDA’s accelerated approval pathway based on increased dystrophin production; continued approval may be contingent upon verification of clinical benefit in confirmatory trials. Standard DMD management includes corticosteroids, and the policy requires documentation of kidney function testing and stable corticosteroid dosing (unless contraindicated) when evaluating prior authorization requests.

Definitions and Clinical Measures

Golodirsen (Vyondys 53)

DefinitionGolodirsen (Vyondys 53) is an antisense oligonucleotide therapeutic indicated for DMD patients with mutations amenable to exon 53 skipping.

Regulatory contextApproved under accelerated approval based on dystrophin increase; clinical benefit verification pending (document notes).

Therapeutic classAntisense oligonucleotide

Clinical measures referenced (6MWT, FVC, LVEF)

Clinical measures referenced6-minute walk test (6MWT), forced vital capacity (FVC), left ventricular ejection fraction (LVEF)

Additional measure sometimes referencedTime to stand (TTSTAND) noted in appendices for some studies

Policy Summary

PayerFidelis Care

PolicyGolodirsen (Vyondys 53) Coverage

Policy CodePolicy NY.CP.PHAR.453

Change TypeNo material change

Effective DateMar 1, 2020

Next Review Date

SourceLink

On This Page

Initial and continued therapy documentation must be present in clinical chart notes, laboratory reports, medical claims and/or pharmacy claims.
Baseline kidney function testing is required prior to initiation.
Stable corticosteroid dose must be documented prior to initiation unless contraindicated or intolerant.

Documentation Required

Medical Director Review May Be Required

All requests for exon‑skipping therapies (Vyondys 53, Exondys 51, Amondys 45, Viltepso) may require medical director review. Providers should ensure complete documentation is submitted to avoid delays or denials.

Medical director review may be requested for any submission.
Missing documentation (e.g., lack of confirmed exon‑amenable mutation, absent baseline kidney testing, or absent corticosteroid status) may trigger denial or request for additional information.

Note

Provider Actions (Not Otherwise Specified)

Provider actions not specified in this excerpt should follow standard prior authorization submission processes and any additional instructions on the authorization request form. When in doubt, contact Fidelis Care for guidance or to confirm required supporting documentation.

Follow usual PA submission workflows and include all supporting clinical documentation.
Contact the plan for questions or if policy excerpts do not address a specific provider action.

Use in criteriaThese measures are used as study inclusion thresholds and baseline clinical assessments

Eteplirsen (Exondys 51)

DefinitionEteplirsen (Exondys 51) is an antisense oligonucleotide indicated for DMD with mutations amenable to exon 51 skipping.

Regulatory noteApproved under accelerated approval based on increased dystrophin; continued approval may require confirmatory trials.

Context in policyMentioned as a comparable exon‑skipping therapy with enrollment criteria and prior authorization references.

Mutations amenable to exon 51 skipping

Examples of exon 51-amenable mutationsCommon mutations listed include deletions such as 3-50, 4-50, 5-50, ... 52-76

Proportion noteDeletions make up >97% of mutations amenable to exon 51 skipping per DMD registration database (document context).

Reference locationAppendix D: common mutations enumerated

Casimersen (Amondys 45)

DefinitionCasimersen (Amondys 45) is an antisense oligonucleotide indicated for DMD patients with mutations amenable to exon 45 skipping.

Regulatory noteApproved under accelerated approval based on increased dystrophin; confirmatory clinical benefit may be required.

Context in policyReferenced as another exon‑skipping therapy with its own enrollment and documentation requirements.

6-minute walk test

Definition of 6MWT6-minute walk test (6MWT): a standardized measure of ambulatory function used in DMD clinical studies and included in appendices.

PurposeUsed as an inclusion threshold and outcome measure in pivotal DMD trials referenced in the document.

Abbreviation6MWT = 6-minute walk test

left ventricular ejection fraction

Definition of LVEFLeft ventricular ejection fraction (LVEF): a measure of cardiac systolic function referenced for baseline cardiac assessment in DMD trials.

Clinical threshold examplesStudies referenced use thresholds such as LVEF ≥ 50% or >40% for enrollment; LVEF < 40% may indicate cardiomyopathy/heart failure.

AbbreviationLVEF = left ventricular ejection fraction

Institute for Clinical and Economic Review

ICER definition/contextInstitute for Clinical and Economic Review (ICER): referenced report concluded evidence insufficient to show net clinical benefit for some exon‑skipping therapies added to corticosteroids and supportive care.

Document useICER evidence report cited in appendices regarding clinical benefit assessment for Vyondys 53 and other therapies.

ImplicationUsed to contextualize uncertainty of clinical benefit despite dystrophin increases.

Exon 53 amenable mutations

Examples of exon 53-amenable mutationsCommon mutations include deletions/variants such as 3-52, 4-52, 5-52, 6-52, 9-52, 10-52, 11-52, 13-52, ... 54-77

Reference locationAppendix D enumerates common mutations amenable to exon 53 skipping.

Use in criteriaMutation must be confirmed as amenable to exon 53 skipping for coverage eligibility.

Clinical measures referenced: 6MWT, FVC, LVEF, TTSTAND

Clinical measures list6-minute walk test (6MWT), forced vital capacity (FVC), left ventricular ejection fraction (LVEF), time to stand (TTSTAND)

ContextThese measures are referenced across appendices and study inclusion criteria for DMD therapies in the policy document.

ApplicationUsed for baseline assessment and enrollment thresholds in cited studies.