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    v1.0.28
    OpenPayerPolicy UpdatesPharmacy & Specialty DrugsConnectiCare Vyondys 53 Coverage Update | OpenPaye…
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    CurrentConnectiCarePolicy C17926-A

    Vyondys 53 (golodirsen) Coverage Criteria

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    This policy governs coverage determinations for Vyondys 53 (golodirsen) for members with Duchenne muscular dystrophy. The drug is considered not medically necessary for all indications because clinical benefit has not been established.

    Policy Summary
    PayerConnectiCare
    PolicyVyondys 53 (golodirsen) Coverage Criteria
    Policy CodePolicy C17926-A
    Change TypeAnnual reviewno material clinical or coverage changes
    Effective DateJune 6, 2026
    Next Review DateApril 1, 2027
    Key ActionSubmit complete medical rationale and supporting patient records, test results, and provider credentials when requested.
    SourceLink
    POLICY UPDATE CHANGES

    No material clinical or coverage changes in this revision.

    All indicationsCoverage stance
    DMDCondition addressed
    IVRoute of administration
    Not establishedClinical benefit

    OpenPayer is powered by Trek Health's payer performance platform. Trek continuously ingests, validates, and normalizes Transparency in Coverage data alongside payer policies and other commercial payer data to create a structured payer intelligence foundation. OpenPayer uses this foundation to deliver personalized search results, dynamically generated policy pages, and tailored policy monitoring based on each user's payers, specialties, billing codes, and areas of interest. The same intelligence powers broader payer performance workflows, including reimbursement benchmarking, contract evaluation, payer negotiations, and financial decision-making.

    Experimental
    Other uses

    Coverage Criteria

    Not medically necessary

    Vyondys 53 is considered not medically necessary for all indications.

    Not medically necessary: Vyondys 53 (golodirsen) is not medically necessary for all indications, including Duchenne muscular dystrophy, because insufficient evidence of therapeutic value is available and clinical benefit has not been established.

    Coverage may be reconsidered as relevant clinical evidence becomes available or if confirmatory trials verify clinical benefit.

    All other uses of Vyondys 53 (golodirsen) are considered experimental/investigational and follow the payer’s Off-Label policy.

    Vyondys 53 (golodirsen) is considered not medically necessary for all indications, including Duchenne muscular dystrophy, because there is insufficient evidence of therapeutic value and clinical benefit has not been established.

    Coding Information

    HCPCS CodeHCPCS
    NANA

    Provider and Billing Guidance

    Documentation Required

    Medical Rationale and Supporting Records

    When requesting an exception to this guideline, providers must submit complete medical rationale and supporting documentation for the request. The plan may require additional records to evaluate coverage, quality, and fraud, waste, and abuse concerns.

    • Medical rationale: Explain the clinical basis for the requested exception and the appropriateness of the drug or service.
    • Supporting records: Include complete patient records, relevant test results, and credentials or identifying information for the provider ordering or performing the drug or service, as applicable.
    Denial Risk

    Clinical Benefit Not Established

    Vyondys 53 (golodirsen) is considered not medically necessary for all indications, including Duchenne muscular dystrophy (DMD), because insufficient evidence demonstrates therapeutic value or established clinical benefit. Clinical studies showed a consistent safety and tolerability profile in a small number of people with DMD, but pivotal trials were not designed to evaluate long-term safety or establish clinical benefit; coverage may be reconsidered if confirmatory trials verify benefit.

    Clinical Background

    Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder caused by mutations in the dystrophin gene and characterized by progressive muscle weakness and loss of function. Vyondys 53 is intended for patients with a confirmed DMD-gene mutation amenable to exon 53 skipping; however, the policy states that clinical benefit, including improved motor function, has not been established.

    Definitions

    Accelerated approval
    PathwayAccelerated approval provides for approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments.
    Basis for approvalApproval can be based on adequate and well-controlled studies showing an effect on a surrogate endpoint reasonably likely to predict clinical benefit.
    Confirmatory evidenceThe pathway provides earlier patient access while the company conducts clinical trials to verify the predicted clinical benefit.
    Surrogate marker
    DefinitionA surrogate marker is a laboratory measurement or physical sign used in therapeutic trials as a substitute for a clinically meaningful endpoint.
    Clinical endpointA clinically meaningful endpoint directly measures how a patient feels, functions, or survives.
    Policy Summary
    PayerConnectiCare
    PolicyVyondys 53 (golodirsen) Coverage Criteria
    Policy CodePolicy C17926-A
    Change TypeAnnual reviewno material clinical or coverage changes
    Effective DateJune 6, 2026
    Next Review DateApril 1, 2027
    Key ActionSubmit complete medical rationale and supporting patient records, test results, and provider credentials when requested.
    SourceLink
    On This Page
    Coverage CriteriaCoding InformationProvider and Billing GuidanceClinical BackgroundDefinitions
    Denial Risk

    Insufficient Supporting Documentation

    Reimbursement may be denied, or other appropriate action may be taken, when submitted documentation does not support that the drug or service is medically necessary, is not investigational or experimental, and is within the member's benefit scope. Action may also be taken when documentation demonstrates a pattern of inappropriate or excessive billing or other practice.

    Note

    Non-Labeled Use

    All uses of Vyondys 53 (golodirsen) outside the applicable labeled use are considered experimental or investigational and follow the plan's Off-Label policy. Vyondys 53 has no labeled contraindications.

    Billing Rule

    Coding and Benefit Limitations

    Codes in this policy are for reference only, may not be all-inclusive or applicable to every state or line of business, and deleted or otherwise ineffective codes may not be reimbursable. Listing a code does not guarantee coverage; coverage is determined by the member's benefit document, and providers and facilities must use industry-standard coding practices. Claims may be rejected or denied and payments recovered when services are billed incorrectly or are not covered.

    • The policy lists no HCPCS code (NA).
    • CPT codes and descriptions are subject to applicable CPT® and AMA copyright requirements.
    • The plan may revise coding and billing guidance as needed.
    Expected purposeThe surrogate marker is expected to predict the effect of the therapy.
    Accelerated approval
    Diseases addressedAccelerated approval applies to drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments.
    Evidence requiredApproval may be based on adequate and well-controlled studies showing an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients.
    Post-approval requirementConfirmatory clinical trials are conducted to verify the predicted clinical benefit; if benefit is not verified, the FDA may initiate proceedings to withdraw approval.