Modifiedsierra health and lifePolicy 2026D0088I

Vyondys 53 (golodirsen) — coverage criteria for Duchenne muscular dystrophy (exon 53 skipping)

Defines medical necessity criteria, coding, and coverage stance for Vyondys 53 (golodirsen) for treatment of Duchenne muscular dystrophy in members whose DMD gene mutation is amenable to exon 53 skipping; applies to UnitedHealthcare Commercial and Individual Exchange plans noted in the document.

Policy Summary

Payersierra health and life

PolicyVyondys 53 (golodirsen) — coverage criteria for Duchenne muscular dystrophy (exon 53 skipping)

Policy CodePolicy 2026D0088I

Change TypeRevised — added concomitant therapy restrictiontemplate/application updates

Effective DateMay 1, 2026

Next Review Date

Key ActionObtain prior authorization with medical records confirming DMD diagnosis, exon 53 amenable mutation, and required baseline functional measures (e.g., 6MWT, NSAA, or TTR).

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 Application section.

Updated References section to reflect the most current information and archived previous policy versions.

05/01/2026policy revision date

12max initial auth (months)

≈8%patients amenable

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Coverage Criteria for Vyondys 53 (golodirsen)

inv-01: Initial Therapy

Covered when ALL of the following are met:

Initial therapy - core requirements: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in DMD; documentation (chart notes, laboratory values) confirming the DMD gene mutation is amenable to exon 53 skipping; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with U.S. FDA–approved labeling; initial authorization for no more than 12 months.

Baseline ambulatory function: Either: (A) medical records showing 6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently (without side-by-side assist, cane, walker, wheelchair, etc.) prior to starting Vyondys 53; OR (B) both: medical records confirming the patient is ambulatory without an assistive device AND one of the following: NSAA score > 17 OR time to rise (TTR) < 7 seconds.6MWT ≥ 300 m OR NSAA >17 OR TTR <7s

Ambulatory without assistive device means no side-by-side assist, cane, walker, wheelchair, etc.

Prior gene therapy history: Either: patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) for DMD; OR both: patient previously received gene therapy and submission of medical records documenting a clinically meaningful functional decline (e.g., loss of a motor milestone) since receiving gene replacement therapy.

Concomitant therapy restrictions: Vyondys 53 must not be used concomitantly with Duvyzat (givinostat) or other exon-skipping therapies for DMD (e.g., Amondys [casimersen], Exondys 51 [eteplirsen], Viltepso [viltolarsen]).

Concomitant use is a disqualifying condition for coverage.

inv-02: Continuation Therapy

Reauthorization covered when ALL of the following are met:

Continuation therapy - core requirements: Submission of medical records confirming the patient remains ambulatory without needing an assistive device (no side-by-side assist, cane, walker, wheelchair, etc.); prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with U.S. FDA–approved labeling; reauthorization for no more than 12 months.

Concomitant use is a disqualifying condition for continuation of therapy.

inv-03: Unproven

Unproven indications: Use of Vyondys 53 for other forms of muscular dystrophy (for example, Becker muscular dystrophy) is considered unproven and is not medically necessary.

inv-04: Coverage Rationale (summary)

Covered when ALL of the following are met (as summarized from document changes and FDA indication):

FDA indication: Patient has Duchenne muscular dystrophy with a confirmed DMD gene mutation that is amenable to exon 53 skipping.

Indication approved under accelerated approval based on increased dystrophin; continued approval may be contingent on confirmatory trials.

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

This criterion was added in the 05/01/2026 policy revision.

Benefit plan verification: Coverage is subject to the member-specific benefit plan terms; verify the applicable UnitedHealthcare Commercial or Individual Exchange plan document and applicable federal/state mandates.

UnitedHealthcare policy and member plan govern coverage determinations.

Treatment of other forms of muscular dystrophy (for example, Becker muscular dystrophy) with Vyondys 53 is explicitly excluded as unproven and not medically necessary.

FDA approval alone does not establish coverage. This policy applies to specified UnitedHealthcare plans and member-specific documents; coverage determinations should follow the applicable UnitedHealthcare Medical Benefit Drug Policy and the member’s benefit plan document.

Vyondys 53 is considered not medically necessary for muscular dystrophies other than Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 53 skipping (for example, treatment of Becker muscular dystrophy).

No additional explicit not medically necessary conditions are stated in this segment beyond the exclusion of non‑DMD indications; the document notes FDA approval is informational and not by itself a basis for coverage.

Coding and Diagnostic Codes

Applicable HCPCS CodesHCPCS

J1429

Injection, golodirsen, 10 mg

Relevant Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

6-Minute Walk Test (6MWT) — baseline thresholds referenced for inclusion/coverage

Policy initial threshold6MWT ≥ 300 meters while walking independently prior to beginning Vyondys 53 therapy

Alternate baseline criteriaAmbulatory without assistive device AND either NSAA score > 17 OR time to rise (TTR) < 7 seconds

Trial inclusion reference rangesClinical trials referenced baseline 6MWT inclusion of >250 m (SKIP‑NMD) and 300–450 m (ESSENCE) depending on study

Provider Requirements and Operational Notes

Prior Authorization

Prior Authorization Required

Prior authorization required. Submission of medical records (e.g., chart notes, laboratory values) demonstrating a diagnosis of Duchenne muscular dystrophy and that the patient's DMD gene mutation is amenable to exon 53 skipping is required for initial and continuation therapy. Initial authorization will be for no more than 12 months.

Submit documentation confirming DMD diagnosis and exon 53 amenable mutation (e.g., genetic test reports, chart notes).
Provide baseline functional measures prior to initiation: 6-Minute Walk Test (6MWT) ≥ 300 meters OR documentation of ambulatory status plus NSAA >17 or time to rise <7 seconds.
For patients previously treated with gene therapy (e.g., Elevidys), submit medical records documenting clinically meaningful functional decline since gene replacement therapy.

Documentation Required

Concomitant Therapy Restriction

Vyondys 53 must not be used concomitantly with Duvyzat (givinostat) or other exon-skipping therapies (e.g., Amondys, Exondys 51, Viltepso). Prescriptions must be by, or in consultation with, a neurologist experienced in DMD and dosed per FDA labeling.

Background

Duchenne muscular dystrophy (DMD) is an X‑linked disorder caused by mutations in the dystrophin gene that lead to progressive muscle degeneration, loss of ambulation in childhood or adolescence, and cardiopulmonary complications. Golodirsen (Vyondys 53) is an antisense oligonucleotide designed to induce exon 53 skipping, allowing production of internally truncated dystrophin in patients whose mutations are amenable to exon 53 skipping; approximately 8% of patients with DMD have such mutations.

Definitions and Key Terms

Exon 53 skipping — molecular strategy definition

Molecular strategy definitionGolodirsen is an antisense oligonucleotide that binds to exon 53 of dystrophin pre‑mRNA to induce exclusion (skipping) of exon 53 during mRNA processing, aiming to produce an internally truncated dystrophin protein

Therapeutic intentExon 53 skipping is intended to restore the dystrophin reading frame in patients with mutations amenable to exon 53 skipping (~8% of DMD patients)

Indication scopeApplicable only to patients with confirmed DMD gene mutations amenable to exon 53 skipping; not studied in non‑amenable DMD or other muscular dystrophies (e.g., Becker)

6-Minute Walk Test (6MWT) — definition and thresholds

DefinitionThe 6‑Minute Walk Test (6MWT) is a functional measure of ambulatory capacity used to assess baseline and change in walking distance over 6 minutes

Policy baseline requirement

References and Revision History

2026-05-01policy_revisionLatest

Revised coverage criteria to add a prohibition on concomitant use of Vyondys 53 with Duvyzat (givinostat) and transferred content to a shared policy template applicable to UnitedHealthcare Commercial and Individual Exchange plans (Application section added).

2026-05-01supporting_update

Updated References section to reflect current information and archived prior policy versions (2025D0088H and IEXD0088.09).

References listed in this policy include the Vyondys 53 package insert, clinical trial publications, long‑term safety/efficacy reports, the ESSENCE trial registry entry, and an ICER evidence report. The policy history was updated 05/01/2026 to revise coverage criteria, update the References section, and archive prior policy versions.