CurrentUnitedHealthcarePolicy CSOH2026D0086.E

Vyondys 53 (Golodirsen) (for Ohio Only)

State-specific UnitedHealthcare Medical Benefit Drug Policy that governs coverage of Vyondys 53 for members in Ohio with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

Policy Summary

PayerUnitedHealthcare

PolicyVyondys 53 (Golodirsen) (for Ohio Only)

Policy CodePolicy CSOH2026D0086.E

Change TypeRevised coverage criteria

Effective DateJun 1, 2026

Next Review Date

Key ActionObtain prior authorization and submit medical records confirming DMD diagnosis amenable to exon 53 skipping and ambulatory status; initial and reauthorization limited to ≤12 months.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria to add a requirement that Vyondys 53 will not be used concomitantly with Duvyzat (givinostat).

Ohio-onlyapplicability

J1429listed drug code

≤12 moinitial auth

≤12 moreauth

~8%eligible subgroup

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Coverage Criteria for Vyondys 53 (golodirsen)

Initial Therapy

Covered when ALL of the following are met

Initial coverage criteria

Diagnosis and prescriber: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD.
Genetic confirmation: Submission of medical records (e.g., chart notes, laboratory values) confirming the mutation of the DMD gene is amenable to exon 53 skipping.
Ambulatory status: Submission of medical records (e.g., chart notes, laboratory values) confirming that the patient is ambulatory prior to beginning Vyondys 53 therapy.
Prior gene therapy: Either the patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]), OR both: the patient previously received gene therapy for DMD and submission of medical records documents a clinically meaningful functional decline (e.g., loss of a motor milestone) since receiving gene replacement therapy.
Concomitant therapy exclusions: Vyondys 53 will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys, Exondys 51, Viltepso).
Dosing and authorization length: Dosing is in accordance with the United States Food and Drug Administration approved labeling; initial authorization will be for no more than 12 months.

Prescriber and dosing (duplicate node for clarity): Prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing per FDA label.

Continuation Therapy

Covered when ALL of the following are met

Continuation coverage criteria

Ongoing ambulatory status: Submission of medical records (e.g., chart notes) demonstrating that the patient continues to remain ambulatory.
Concomitant therapy exclusions: Vyondys 53 will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys, Exondys 51, Viltepso).
Prescriber and dosing: Prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing is in accordance with the United States Food and Drug Administration approved labeling.
Reauthorization length: Reauthorization will be for no more than 12 months.

Unproven / Not Medically Necessary

Not covered indications: Vyondys 53 is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (e.g., Becker muscular dystrophy) or for DMD that is not amenable to exon 53 skipping.

Vyondys 53 (golodirsen) is not medically necessary for the treatment of other forms of muscular dystrophy, including Becker muscular dystrophy. Coverage is limited to indications and patient populations specifically described elsewhere in this policy; therapies for non-DMD muscular dystrophies are excluded from coverage as unproven.

Use of Vyondys 53 for muscular dystrophies that are not amenable to exon 53 skipping is considered unproven and not medically necessary. The policy only contemplates coverage when a confirmed DMD gene mutation is demonstrated to be amenable to exon 53 skipping; absent that molecular eligibility, Vyondys 53 will not be covered.

Coding

Applicable HCPCS CodesHCPCS

J1429

Injection, golodirsen, 10 mg

Diagnosis Codes (reference)ICD-10

G71.01

Duchenne or Becker muscular dystrophy

Provider Actions, Authorization & Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial authorizations are limited to no more than 12 months; reauthorization (continuation) is limited to no more than 12 months.

Initial authorization: <= 12 months
Reauthorization/continuation: <= 12 months

Documentation Required

Submit medical records (e.g., chart notes, laboratory values) confirming the diagnosis of Duchenne muscular dystrophy (DMD) with documentation that the patient has a DMD gene mutation amenable to exon 53 skipping. For initial requests, include documentation the patient is ambulatory prior to beginning therapy. For continuation requests, submit chart notes demonstrating the patient continues to remain ambulatory. If the patient previously received DMD gene replacement therapy (e.g., Elevidys), include records documenting a clinically meaningful functional decline (loss of muscle strength/motor ability or loss of a motor milestone) since receiving gene therapy.

Background

Duchenne muscular dystrophy (DMD) is an X-linked disorder caused by mutations in the dystrophin gene that result in progressive muscle degeneration, loss of ambulation during childhood, and life‑threatening cardiopulmonary complications. Golodirsen (Vyondys 53) is an antisense oligonucleotide designed to skip exon 53 during dystrophin pre‑mRNA processing, which can restore the reading frame in patients with mutations amenable to exon 53 skipping and permit production of internally truncated dystrophin protein. Approximately 8% of DMD patients have mutations potentially amenable to exon 53 skipping.

Definitions

Amenable to exon 53 skipping — definition and key details

DefinitionA confirmed DMD gene mutation (typically an out-of-frame deletion) that allows exclusion of exon 53 to restore the reading frame and permit production of truncated dystrophin protein.

MechanismGolodirsen is an antisense oligonucleotide designed to bind exon 53 of dystrophin pre-mRNA, resulting in exclusion (skipping) of exon 53 during mRNA processing.

Clinical scopeAmenable mutations are typically out-of-frame deletions; approximately 8% of DMD patients have mutations amenable to exon 53 skipping.

Intended effectExon 53 skipping is intended to allow production of an internally truncated dystrophin protein, potentially restoring the reading frame.

Regulatory contextVyondys 53 is FDA‑approved for DMD patients with a confirmed mutation amenable to exon 53 skipping under accelerated approval based on increased dystrophin in skeletal muscle.