CurrentCentenePolicy CP.PHAR.453

Golodirsen (Vyondys 53) for Duchenne muscular dystrophy

Defines medical necessity and prior authorization criteria for use of golodirsen (Vyondys 53) to treat Duchenne muscular dystrophy in patients with DMD gene mutations amenable to exon 53 skipping for Centene-affiliated health plans.

Policy Summary

PayerCentene

PolicyGolodirsen (Vyondys 53) for Duchenne muscular dystrophy

Policy CodePolicy CP.PHAR.453

Change TypeNo material change

Effective DateMar 1, 2020

Next Review Date

Key ActionSubmit prior authorization with documentation showing genetic confirmation of exon 53 amenable DMD and required clinical assessments (6MWT, LVEF, FVC).

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsapproval duration

30 mg/kg/weekmax dose

≥250 m6MWT threshold

≥50%LVEF threshold

≥50%FVC threshold

Coverage Criteria for Golodirsen (Vyondys 53)

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inv-01: Initial Therapy

Covered when ALL of the following are met:

Initial Approval - DMD: 1. Diagnosis of Duchenne muscular dystrophy (DMD) with a mutation amenable to exon 53 skipping confirmed by genetic testing; 2. Age ≤ 15 years at therapy initiation; 3. Prescribed by or in consultation with a neurologist; 4. Member has all of the following assessed within the last 30 days: a) ambulatory function with 6-minute walk test (6MWT) distance ≥ 250 m (able to walk with or without assistive devices; not wheelchair dependent); b) stable cardiac function with left ventricular ejection fraction (LVEF) ≥ 50%; c) stable pulmonary function with predicted forced vital capacity (FVC) ≥ 50%; 5. Inadequate response (e.g., significant decline in 6MWT, LVEF, or FVC) despite adherent use of an oral corticosteroid (for example, prednisone or deflazacort) for ≥ 6 months, unless contraindicated or clinically significant adverse effects are experienced; 6. Vyondys 53 (golodirsen) is prescribed concurrently with an oral corticosteroid unless contraindicated; 7. Vyondys 53 is not prescribed concurrently with other exon-skipping therapies; 8. Member has not previously received gene replacement therapy for DMD (e.g., Elevidys); 9. Dose does not exceed 30 mg/kg per week.see node text

Approval duration: 6 months

inv-02: Continuation Therapy

Covered for continuation when ALL of the following are met:

Continued Therapy - DMD: 1. Currently receiving medication for DMD with mutation amenable to exon 53 skipping or member previously met initial approval criteria; 2. Member is responding to therapy as evidenced by one of the following: a) all of the following assessed within the last 6 months: i) ambulatory function with 6MWT ≥ 250 m; ii) stable cardiac function with LVEF ≥ 50%; iii) stable pulmonary function with predicted FVC ≥ 50%; OR b) member received the medication via a healthcare insurer without meeting 2a and medical records show improved or stable LVEF and FVC assessed within the last 6 months; 3. Member has been assessed by a neurologist within the last 6 months; 4. Vyondys 53 is prescribed concurrently with an oral corticosteroid unless contraindicated; 5. Vyondys 53 is not prescribed concurrently with other exon-skipping therapies; 6. Member has not previously received gene replacement therapy for DMD (e.g., Elevidys); 7. If request is for a dose increase, the new dose does not exceed 30 mg/kg per week.see node text

Approval duration: 6 months

Coverage is not authorized for indications that are not FDA‑approved and are not addressed by this policy unless there is sufficient documentation of efficacy and safety in accordance with applicable off‑label use policies (e.g., CP.CPA.09 for commercial, HIM.PA.154 for Health Insurance Marketplace, and CP.PMN.53 for Medicaid) or unless supported by the member's evidence of coverage documents.

Coverage determinations and benefit administration for golodirsen are governed by the member's plan documents. This clinical policy is intended as a guide to medical necessity and does not constitute a contract or guarantee of payment. All decisions remain subject to the terms, conditions, exclusions, and limitations of the applicable coverage documents (for example, evidence of coverage, certificate of coverage, policy, or contract of insurance), as well as applicable state and federal requirements and Health Plan‑level administrative policies and procedures.

Use of golodirsen outside the FDA‑approved indication for Duchenne muscular dystrophy with mutations amenable to exon 53 skipping, or use that does not meet the clinical criteria listed in this policy, may be considered not medically necessary. The FDA approval for Vyondys 53 is an accelerated approval based on increased dystrophin production, and continued approval may depend on confirmatory trials to verify clinical benefit.

Provider Actions, Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required for Vyondys 53. Providers must submit a prior authorization request and include supporting documentation (e.g., office chart notes, genetic test results, 6MWT, echocardiogram reports, pulmonary function test results, medication history) demonstrating that the member meets all approval criteria in this policy. All requests may be subject to medical director review.

Prior authorization required
Include documentation supporting all approval criteria (genetic testing, 6MWT, LVEF, FVC, corticosteroid history)
HCPCS J1429 referenced for billing (Injection, golodirsen, 10 mg)

Documentation Required

Provider must submit documentation (such as office chart notes, laboratory and testing results, and specialist consultation notes) supporting that the member has met all approval criteria. Documentation should include timing of assessments (see assessment timing callout) and records of corticosteroid use or contraindication. Failure to provide adequate documentation may result in denial.

Submit office notes, genetic test reports, 6MWT, echocardiogram, PFT results, and medication history
All requests may require medical director review

Denial Risk

Incomplete or Non‑Covered Requests May Be Denied

Requests that do not meet the clinical criteria in this policy, or that are for non‑FDA approved indications not addressed by this policy, may be denied. For off‑label requests, coverage may be considered only with sufficient documentation of efficacy and safety per applicable off‑label use policies (see policy references).

Requests not meeting policy clinical criteria may be denied
Non‑FDA approved indications require sufficient documentation per off‑label use policies (CP.CPA.09, HIM.PA.154, CP.PMN.53)

Note

Coverage Determined by Plan Documents

Coverage decisions are subject to the terms, conditions, exclusions, and limitations of the member's coverage documents (e.g., evidence of coverage, certificate/policy/contract). Providers must follow applicable state and federal requirements and Health Plan‑level administrative policies and procedures. This clinical policy is a guide to medical necessity and does not guarantee payment.

Coverage governed by member's evidence of coverage and contract terms
Providers must follow state and federal requirements and plan administrative policies

Documentation Required

Timing of Assessments

Assessments used to determine initial and continued eligibility must be recent: for initial approval, 6MWT, LVEF, and FVC must be assessed within the last 30 days; for continued therapy, these assessments must have been performed within the last 6 months. Documentation must include dates and results of the 6MWT, echocardiogram (LVEF), and pulmonary function testing (FVC).

Initial approval: 6MWT, LVEF, and FVC assessed within 30 days of therapy initiation
Continued therapy: 6MWT, LVEF, and FVC assessed within the last 6 months
Include dates and numeric results in documentation

Definitions and Abbreviations

Product identification and indication summary

DrugGolodirsen (Vyondys 53)

Mechanism / classAntisense oligonucleotide

FDA‑approved indicationTreatment of Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation amenable to exon 53 skipping (accelerated approval)

Typical dosing constraintDose does not exceed 30 mg/kg per week

Billing code referencedHCPCS J1429 (Injection, golodirsen, 10 mg)

Abbreviations

6MWT6-minute walk test

FVCForced vital capacity

LVEFLeft ventricular ejection fraction

Health Plan definition and scope

Health Plan (definition)A health plan that has adopted this clinical policy and that is operated or administered, in whole or in part, by Centene Management Company, LLC, or any of such health plan's affiliates.

Policy purposeProvides a guide to medical necessity to assist in making coverage decisions and administering benefits; does not guarantee payment.

Administrative scopeCoverage decisions are subject to all terms, conditions, exclusions, and limitations of the member's coverage documents and applicable state/federal requirements.

OpenPayer

Golodirsen (Vyondys 53) for Duchenne muscular dystrophy

Coverage Criteria for Golodirsen (Vyondys 53)

Coding and Clinical Thresholds

Provider Actions, Authorization, and Documentation

Background

Definitions and Abbreviations