ModifiedUnitedHealthcarePolicy CSNJ2026D0086M

Vyondys 53 Nj Cs

State-specific UnitedHealthcare Medical Benefit Drug Policy for Vyondys 53 (golodirsen) applicable only to New Jersey members, defining initial and continuation coverage criteria, excluded uses, applicable HCPCS and diagnosis codes, and authorization durations.

Policy Summary

PayerUnitedHealthcare

PolicyVyondys 53 Nj Cs

Policy CodePolicy CSNJ2026D0086M

Change TypeRevised coverage criteriaadded concomitant therapy exclusion

Effective DateMay 1, 2026

Next Review Date

Key ActionProviders must submit medical records confirming DMD diagnosis amenable to exon 53 skipping, baseline functional status, and documentation regarding prior gene therapy status; initial authorization limited to up to 12 months.

SourceLink

POLICY UPDATE CHANGES

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

12Initial authorization months max

12Reauthorization months max

~8%DMD patients amenable to exon 53 skipping (approx.)

Coverage Summary

This is the UnitedHealthcare Medical Benefit Drug Policy specific to New Jersey for Vyondys 53 (golodirsen). The policy defines initial and continuation coverage criteria, excluded uses, applicable HCPCS and diagnosis codes, and authorization durations for New Jersey members. Coverage stance: covered_with_criteria. Effective date: May 1, 2026; Last review: May 1, 2026. Status: MODIFIED.

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Coverage Rationale - Initial Therapy (Medically Necessary)

Vyondys 53 may be covered when ALL of the following are met:

ALL of the following

Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD
Submission of medical records (e.g., chart notes, laboratory values) confirming the mutation of the DMD gene is amenable to exon 53 skipping
Submission of medical records (e.g., chart notes) that provides baseline status of the patient prior to starting therapy (e.g., confirming that the patient is walking without needing an assistive device such as a cane, walker, wheelchair, etc.)
ONE of the following
- Patient has not previously received gene therapy (e.g., Elevidys (delandistrogene moxparvovec-rokl)) for the treatment of DMD
- If previously received gene therapy
  - Patient has previously received gene therapy (e.g., Elevidys (delandistrogene moxparvovec-rokl)) for the treatment of DMD
  - Submission of medical records (e.g., chart notes, laboratory values) documenting a clinically meaningful functional decline resulting from loss of muscle strength/motor ability (e.g., loss of a motor milestone) since receiving gene replacement therapy (e.g., Elevidys)
Vyondys 53 will not be used concomitantly with any of the following: Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen))
Explicit contraindication to concomitant use with listed agents
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
Initial authorization will be for no more than 12 months

Coverage Rationale - Continuation Therapy (Reauthorization)

Reauthorization may be approved when ALL of the following are met:

ALL of the following

Submission of medical records (e.g., chart notes) confirming that the patient is receiving clinical benefit (e.g., walking without needing an assistive device such as a cane, walker, wheelchair, etc.)
Vyondys 53 will not be used concomitantly with any of the following: Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen))
Explicit contraindication to concomitant use with listed agents
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 will be for no more than 12 months
Note: reauthorization duration limit

Unproven Uses and Exclusions

Unproven / Not Medically Necessary

ALL of the following

Vyondys 53 is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (e.g., Becker muscular dystrophy)
Concomitant therapy contraindication (explicit): Vyondys 53 will not be used concomitantly with any of the following: Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen))
This explicit contraindication is surfaced here as an exclusion/denial risk

Provider Actions & Authorization Requirements

Prior Authorization

Initial prior authorization required

Providers must submit medical records confirming the patient has DMD amenable to exon 53 skipping, documentation of baseline functional status (for example, walking without an assistive device), and information on prior gene therapy status. Initial authorization will be for no more than 12 months.

J1429

Prior Authorization

Reauthorization requirements

For continuation, submit medical records documenting that the patient is receiving clinical benefit (for example, ambulatory status without an assistive device). Reauthorization will be for no more than 12 months.

J1429

Documentation Required

Documentation of mutation amenable to exon 53 skipping

Medical records or laboratory values confirming that the DMD gene mutation is amenable to exon 53 skipping must be submitted with the authorization request.

Denial Risk

Concomitant therapy exclusion

Claims may be denied if Vyondys 53 is used concomitantly with Duvyzat (givinostat) or other exon‑skipping therapies for DMD (examples: Amondys (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen)).

J1429

Documentation Required

Specialist prescribing requirement

Vyondys 53 must be prescribed by, or in consultation with, a neurologist with expertise in the diagnosis and treatment of Duchenne muscular dystrophy.

Quick thresholds and limits

Initial authorization months max12

Reauthorization months max12

DMD patients amenable to exon 53 skipping (approx.)~8%

Baseline walking statuswalking without needing an assistive device (e.g., cane, walker, wheelchair)

Clinical Evidence

-99.0 mMean 6MWT decline at 144 weeks for golodirsen-treated patients (vs -181.4 m in controls; p = 0.067)

Remaining evidence: The ESSENCE study is an ongoing Phase 3, randomized, double-blind, placebo-controlled trial with a primary endpoint of change in the 6-minute walk test (6MWT) at week 96. The Institute for Clinical and Economic Review (ICER) concluded that data for eteplirsen and golodirsen are insufficient and that the clinical significance of observed dystrophin increases is uncertain. Vyondys 53 received accelerated FDA approval based on increases in dystrophin in skeletal muscle, and continued approval may be contingent upon verification of clinical benefit in confirmatory trials.

Background & Definitions

Golodirsen is an antisense oligonucleotide that binds exon 53 of dystrophin pre-mRNA to induce skipping of exon 53 during mRNA processing, enabling production of an internally truncated dystrophin protein. Approximately 8% of DMD patients have mutations amenable to exon 53 skipping. Vyondys 53 received accelerated FDA approval based on increases in dystrophin production; verification of clinical benefit in confirmatory trials is pending.

Exon 53 skipping amenable mutation: A DMD gene mutation (usually deletion) that permits exon 53 exclusion during mRNA processing to restore reading frame and allow truncated dystrophin production.

Revision History

2026-05-01policy_effectiveLatest

Policy CSNJ2026D0086M became effective; revised coverage criteria added explicit prohibition on concomitant use of Vyondys 53 with Duvyzat (givinostat) and other exon-skipping therapies; supporting information references updated and prior policy CSNJ2025D0086L archived.

2026-05-01material_changeLatest

Supporting Information updated references; previous policy version CSNJ2025D0086L archived.

Policy Summary

PayerUnitedHealthcare

PolicyVyondys 53 Nj Cs

Policy CodePolicy CSNJ2026D0086M

Change TypeRevised coverage criteriaadded concomitant therapy exclusion

Effective DateMay 1, 2026

Next Review Date

SourceLink

On This Page

OpenPayer

Vyondys 53 Nj Cs

Coverage Summary

Unproven Uses and Exclusions

Applicable Codes

Provider Actions & Authorization Requirements

Clinical Evidence

Background & Definitions

Revision History