ModifiedUnitedHealthcarePolicy 2026D0088I

Vyondys 53 (golodirsen) for Duchenne muscular dystrophy — Medical Benefit Drug Policy

Coverage policy for Vyondys 53 (golodirsen) for treatment of Duchenne muscular dystrophy in members of UnitedHealthcare Commercial and Individual Exchange plans; defines initial and continuation authorization criteria, dosing/label adherence, and exclusions.

Policy Summary

PayerUnitedHealthcare

PolicyVyondys 53 (golodirsen) — Medical Benefit Drug Policy

Policy CodePolicy 2026D0088I

Change TypeRevised coverage criteriaconcomitant-use prohibition added

Effective DateMay 1, 2026

Next Review Date

Key ActionObtain prior authorization with medical records confirming DMD mutation amenable to exon 53 skipping, required baseline ambulatory measures, and ensure Vyondys 53 is not used concomitantly with givinostat.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria; added criterion requiring Vyondys 53 will not be used concomitantly with Duvyzat (givinostat).

Transferred content to shared policy template that applies to both UnitedHealthcare Commercial and Individual Exchange benefit plans and added an Application section indicating applicability.

Updated References section to reflect the most current information and archived previous policy versions 2025D0088H and IEXD0088.09.

≤12 moInitial authorization duration

≤12 moReauthorization duration

J1429HCPCS

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

≈8%Amenable proportion

1Concomitant-use prohibition

Coverage Criteria for Vyondys 53 (golodirsen)

Unproven / Not Medically Necessary

Unproven indications: Vyondys 53 is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (e.g., Becker muscular dystrophy)

explicit exclusion

Revised Coverage Criteria (excerpt)

Policy coverage is contingent on the FDA‑indicated population and meeting member‑specific plan criteria; the following revised criteria are excerpted from the 05/01/2026 update:

Eligibility per FDA indication: Patient has Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 53 skipping

Indication approved under accelerated approval; continued approval may be contingent on confirmatory trials

Concomitant therapy restriction: Vyondys 53 will not be used concomitantly with Duvyzat (givinostat)

Explicitly added to coverage criteria in 05/01/2026 revision

The use of Vyondys 53 (golodirsen) for other forms of muscular dystrophy, such as Becker muscular dystrophy, is considered unproven and not medically necessary for treatment of those conditions.

The policy revision dated 05/01/2026 explicitly adds a coverage criterion prohibiting concomitant use of Vyondys 53 with Duvyzat (givinostat); use of Vyondys 53 together with givinostat is therefore excluded from coverage.

Vyondys 53 is not medically necessary for patients with DMD who do not have a DMD gene mutation amenable to exon 53 skipping, and is not medically necessary for other muscular dystrophies (for example, Becker muscular dystrophy).

FDA approval of Vyondys 53 alone does not establish coverage. Coverage requires that the patient and request meet the plan’s clinical criteria and member-specific benefit terms; the FDA indication is informational but not sufficient by itself to guarantee coverage.

Coding — HCPCS / Diagnosis / Functional Baseline

Applicable HCPCS CodesHCPCS

J1429

Injection, golodirsen, 10 mg

Relevant Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

6-Minute Walk Test (6MWT) thresholds and alternative functional criteria

Initial 6MWT threshold6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently (no side‑by‑side assist, cane, walker, wheelchair, etc.) prior to starting Vyondys 53

Alternative baseline functional criteriaAmbulatory without assistive device PLUS one of: NSAA score > 17 OR Time to Rise (TTR) < 7 seconds

ESSENCE trial eligibility rangeESSENCE trial required baseline 6MWT ≥ 300 and ≤ 450 meters (ambulatory, corticosteroid‑treated males aged 6–13)

Documentation requiredSubmission of medical records (chart notes, laboratory values) confirming baseline 6MWT or NSAA/TTR results

Continuation/reauthorization implicationOngoing coverage requires documentation of ambulatory status without assistive device and adherence to continuation criteria per policy (prescriber, dosing, and no concomitant excluded therapies)

Provider Actions, Documentation, and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required for Vyondys 53. Submit complete medical records with the prior authorization request, including documentation that the member has a DMD gene mutation confirmed to be amenable to exon 53 skipping. Prior authorization decisions are made after review of member-specific benefits and applicable federal or state mandates.

Required: medical records confirming mutation amenable to exon 53 skipping
Required: baseline ambulation documentation (see below for examples)
Providers must verify member benefits and submit prior authorization per the member's plan

Note

Verify Member Benefits and Prior Authorization

Providers must verify the member's benefits and prior authorization requirements before initiating therapy. Coverage is determined by the member-specific benefit plan; in the event of a conflict, the member's plan document governs. Check for any applicable federal or state mandates that may affect coverage.

Verify eligibility, formulary status, and prior authorization requirements in the payer portal or by calling provider services

Background on Duchenne Muscular Dystrophy and Therapy

Duchenne muscular dystrophy (DMD) is an X-linked disorder caused by mutations in the dystrophin gene leading to progressive muscle degeneration, loss of ambulation during childhood, and cardiopulmonary complications that shorten life expectancy. Golodirsen (Vyondys 53) is an antisense oligonucleotide designed to induce exon 53 skipping, allowing production of an internally truncated dystrophin protein in patients with DMD gene mutations amenable to exon 53 skipping (approximately 8% of DMD patients).

Definitions and Regulatory Notes

Exon 53 skipping — mechanism and golodirsen's role

Molecular mechanismExon 53 skipping uses an antisense oligonucleotide (golodirsen) that binds exon 53 of dystrophin pre‑mRNA to cause exclusion of that exon during mRNA processing, enabling production of an internally truncated dystrophin protein

Therapeutic role of golodirsenGolodirsen (Vyondys 53) is the antisense oligonucleotide drug designed to induce exon 53 skipping in patients with DMD whose mutations are amenable to exon 53 skipping

Intended effectExon skipping is intended to restore the mRNA reading frame and allow synthesis of a shorter, functional dystrophin protein

Amenable to exon 53 skipping — eligible patient definition

Definition of eligible populationPatients with Duchenne muscular dystrophy who have a confirmed DMD gene mutation that is amenable to exon 53 skipping

Prevalence among DMD patients

Policy Summary

PayerUnitedHealthcare

PolicyVyondys 53 (golodirsen) — Medical Benefit Drug Policy

Policy CodePolicy 2026D0088I

Change TypeRevised coverage criteriaconcomitant-use prohibition added

Effective DateMay 1, 2026

Next Review Date

SourceLink

On This Page

Reference the member-specific benefit plan and applicable mandates when submitting requests

Documentation Required

Concomitant Therapy Prohibition

Vyondys 53 must not be used concomitantly with givinostat (Duvyzat) or with other exon-skipping therapies for DMD (for example, casimersen, eteplirsen, viltolarsen). Authorization may be denied or returned if documentation suggests concomitant use.

Do not prescribe Vyondys 53 together with Duvyzat (givinostat)
Do not prescribe Vyondys 53 together with other exon-skipping therapies (e.g., Amondys/casimersen, Exondys 51/eteplirsen, Viltepso/viltolarsen)

Documentation Required

Required Clinical Records

Submit the following clinical records with the prior authorization request to avoid delay or denial: mutation confirmation, baseline functional assessments, and treating neurologist documentation.

Genetic testing report confirming DMD mutation amenable to exon 53 skipping
Baseline ambulation documentation: 6-Minute Walk Test (6MWT) result, North Star Ambulatory Assessment (NSAA) score, time to rise (TTR), or clinician notes documenting independent ambulation
Relevant chart notes, laboratory values, and prior therapies (including any gene therapy history and documentation of functional decline if applicable)
Documentation of prescribing clinician specialty/consultation (neurologist with DMD expertise)

Denial Risk

Denial Risk — Incomplete Documentation

Lack of documentation confirming a DMD mutation amenable to exon 53 skipping or missing baseline ambulation documentation increases the risk of denial. Provide clear, legible copies of genetic reports and clinical assessments with the request.

Missing genetic confirmation or baseline ambulation data may result in denial of the request
Ensure genetic test reports specifically state exon 53 amenability

Note

Coverage Basis and Plan Reference

Coverage is not based solely on FDA approval; UnitedHealthcare evaluates medical necessity against the policy criteria. When deciding coverage, always reference the member-specific benefit plan and any applicable mandates.

FDA approval is informational and not sufficient alone to guarantee coverage
Reference member plan documents and applicable federal/state mandates when submitting requests

Approximately 8% of DMD patients have deletion mutations amenable to exon 53 skipping

Clinical limitationGolodirsen has not been studied in DMD not amenable to exon 53 skipping or in other muscular dystrophies (e.g., Becker muscular dystrophy)

Evidence requirementGenetic confirmation (laboratory/genetic report) documenting the mutation amenable to exon 53 skipping must be submitted for coverage