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    v1.0.28
    OpenPayerPolicy UpdatesPharmacy & Specialty DrugsBlue Cross Blue Shield SD Vyondys 53 Coverage | Op…
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    Currentblue cross blue shield - south dakotaPolicy 05.02.90

    Vyondys 53 (golodirsen) — Coverage Criteria

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    This policy governs coverage determinations for Vyondys 53 (golodirsen) for members of Wellmark Blue Cross and Blue Shield plans in South Dakota, addressing its use for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 53 skipping.

    Policy Summary
    Payerblue cross blue shield - south dakota
    PolicyVyondys 53 (golodirsen) — Coverage Criteria
    Policy CodePolicy 05.02.90
    Change TypeNo material clinical or coverage changes
    Effective DateJan 30, 2020
    Last ReviewJun 1, 2025
    Next Review DateJun 30, 2025
    Key ActionDocument member benefit verification and confirm that DMD diagnosis includes a mutation amenable to exon 53 skipping.
    SourceLink
    POLICY UPDATE CHANGES

    No material clinical or coverage changes in this revision.

    Not medically necessarycoverage stance
    FDA accelerated approval

    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.

    regulatory status
    8%amenable population
    J1429billing code
    ESSENCE 2025confirmatory trial

    Coverage Determination

    Coverage stance

    Policy determination

    Policy coverage determination: Vyondys 53 (golodirsen) is considered not medically necessary for all indications, including treatment of Duchenne muscular dystrophy (DMD), due to insufficient evidence to demonstrate clinical efficacy.

    Despite FDA accelerated approval based on increased dystrophin as a surrogate endpoint, a clinical benefit (including improved motor or pulmonary function) has not been demonstrated; a post-marketing confirmatory trial (ESSENCE) is ongoing and expected to conclude in 2025.

    Policy determination: Vyondys 53 (golodirsen) is considered not medically necessary for all indications. This includes use for Duchenne muscular dystrophy (DMD); the policy cites insufficient evidence to demonstrate clinical efficacy as the basis for the not medically necessary determination.

    Rationale: The policy states that Vyondys 53 (golodirsen) is considered not medically necessary for all indications, including the treatment for DMD, because there is insufficient evidence to demonstrate clinical efficacy. Although regulatory actions (such as accelerated approval) and post‑marketing studies may exist, this policy’s coverage determination is based on the current assessment that clinical benefit has not been established and therefore treatment is not covered.

    Billing and Coding

    Covered HCPCS codes listed in policy documentHCPCS
    J1429Injection, golodirsen, 10 mg (effective 7/1/20)

    Provider Requirements and Actions

    Prior Authorization

    Prior authorization and billing: use HCPCS J1429 but PA expected to be denied

    Report golodirsen using HCPCS code J1429 (Injection, golodirsen, 10 mg); however, the policy designates Vyondys 53 (golodirsen) as not medically necessary for all indications, so prior authorization requests are expected to be denied on medical necessity grounds.

    • Use HCPCS J1429 to bill for golodirsen.
    • Prior authorization requests will be evaluated but are expected to be denied because the drug is considered not medically necessary for all indications.
    Note

    Submit PA with supporting clinical documentation

    Confirm and submit any required prior authorization requests per standard pharmacy/specialty drug processes; include clinical documentation to support the request even though policy states the drug is not medically necessary for all indications.

    • Initiate prior authorization through the plan’s established channel.

    Clinical Background

    Background: Vyondys 53 (golodirsen) is an antisense oligonucleotide therapy developed for patients with Duchenne muscular dystrophy whose genetic mutation is amenable to exon 53 skipping. The drug is administered intravenously on a recurring schedule; however, per this policy the agent is not medically necessary for all indications because available evidence does not demonstrate clinical efficacy.

    Key Definitions

    Exon 53 skipping — definition and mechanism
    DefinitionExon 53 skipping is a targeted exon‑skipping therapy that induces skipping of mutated exons of the DMD gene to restore production of truncated dystrophin protein.
    Mechanism of actionVyondys 53 (golodirsen) targets dystrophin pre‑messenger RNA (mRNA) and induces skipping of mutated exons that disrupt downstream protein synthesis, allowing restoration of a small amount of dystrophin.
    Target populationPatients with Duchenne muscular dystrophy (DMD) who have a confirmed DMD gene mutation that is amenable to exon 53 skipping (approximately 8% of the DMD population).
    Route and frequencyAdministered once weekly via intravenous (IV) infusion; may be given as monotherapy or with corticosteroids.
    Regulatory contextFDA granted accelerated approval in December 2019 based on increased dystrophin as a surrogate endpoint; clinical benefit has not been established and confirmatory trials are ongoing.

    Post-Marketing Commitments / Step Therapy

    StepRequirementNotes
    1Post-marketing confirmatory study (ESSENCE) is ongoing to verify clinical benefitESSENCE is a placebo-controlled trial assessing whether golodirsen improves motor function in DMD patients with mutations amenable to exon 53 skipping; expected to conclude in 2025 and continued FDA approval may be contingent on verification of clinical benefit (withdrawal possible if no benefit found).

    Administration Site

    Note

    Infusion center guidance: once‑weekly IV; may be given with corticosteroids

    Administer Vyondys 53 (golodirsen) as a once‑weekly intravenous infusion; it may be given as monotherapy or concurrently with corticosteroids when delivered in an infusion center setting.

    • Schedule administrations once weekly via IV infusion.
    • May be administered alone or with corticosteroids per treating clinician.
    Policy Summary
    Payerblue cross blue shield - south dakota
    PolicyVyondys 53 (golodirsen) — Coverage Criteria
    Policy CodePolicy 05.02.90
    Change TypeNo material clinical or coverage changes
    Effective DateJan 30, 2020
    Last ReviewJun 1, 2025
    Next Review DateJun 30, 2025
    Key ActionDocument member benefit verification and confirm that DMD diagnosis includes a mutation amenable to exon 53 skipping.
    SourceLink
    On This Page
    Coverage DeterminationBilling and CodingProvider Requirements and ActionsClinical BackgroundKey DefinitionsPost-Marketing Commitments / Step TherapyAdministration Site
  1. Attach clinical records demonstrating diagnosis and mutation status if available.
    2. Documentation Required

    Verify benefits and document indication

    Verify member benefits and document the clinical indication — specifically Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 53 skipping — before submitting a request, as benefit determinations depend on applicable contract language and member coverage.

    • Check the member’s contract for exclusions, limitations, and whether benefits exist for this service.
    • Document confirmation of the DMD gene mutation amenable to exon 53 skipping in the medical record and attach to the request.
    Denial Risk

    Denial risk: not medically necessary for all indications

    Requests for Vyondys 53 (golodirsen) will be denied as not medically necessary for all indications due to insufficient evidence of clinical efficacy; expect denials even for the FDA‑approved indication.

    • Denials are based on the policy statement that Vyondys 53 is not medically necessary for all indications.
    • The policy notes that FDA accelerated approval was based on a surrogate endpoint and that clinical benefit has not been established.