CurrentUnitedHealthcarePolicy CSNJ2026D0086M

Vyondys 53 (Golodirsen) — Medical Benefit Drug Policy (New Jersey)

This UnitedHealthcare Medical Benefit Drug Policy governs coverage of Vyondys 53 (golodirsen) for treatment of Duchenne muscular dystrophy in New Jersey members and outlines initial and continuation authorization criteria, coding, and evidence considerations.

Policy Summary

PayerUnitedHealthcare

PolicyVyondys 53 (Golodirsen) — Medical Benefit Drug Policy (New Jersey)

Policy CodePolicy CSNJ2026D0086M

Change TypeRevised coverage criteriaclarified references

Effective DateMay 1, 2026

Next Review Date

Key ActionObtain prior authorization with medical records confirming DMD amenable to exon 53 skipping and that Vyondys 53 will not be used concomitantly with Duvyzat or other exon-skipping therapies.

SourceLink

POLICY UPDATE CHANGES

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

Updated References section to reflect the most current information.

12 monthsmax initial auth

12 monthsmax reauth

J1429HCPCS

≈8%amenable proportion

May 1, 2026

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Coverage Criteria for Vyondys 53 (golodirsen)

Initial Therapy - Covered when ALL of the following are met.

Covered when ALL of the following are met:

Initial Therapy - main requirements: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD; submission of medical records confirming the DMD gene mutation is amenable to exon 53 skipping; submission of medical records providing baseline status prior to starting therapy (e.g., patient is walking without needing an assistive device); prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with the United States Food and Drug Administration approved labeling; initial authorization will be for no more than 12 months; Vyondys 53 will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys, Exondys 51, Viltepso).

Prior gene therapy status (nested): Patient has not previously received gene therapy (e.g., Elevidys) OR both: patient has previously received gene therapy (e.g., Elevidys) AND submission of medical records documents a clinically meaningful functional decline (loss of muscle strength/motor ability or loss of a motor milestone) since receiving gene replacement therapy.

This reflects the policy's nested OR/AND logic regarding prior gene therapy.

Continuation Therapy - Covered for continuation when ALL of the following are met.

Covered for continuation when ALL of the following are met:

Continuation Therapy requirements: Submission of medical records confirming the patient is receiving clinical benefit (e.g., walking without needing an assistive device); Vyondys 53 will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., Amondys, Exondys 51, Viltepso); prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with the United States Food and Drug Administration approved labeling; reauthorization will be for no more than 12 months.

Unproven / Not Medically Necessary

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

Treatment of other forms of muscular dystrophy is excluded. Specifically, use of Vyondys 53 for muscular dystrophies other than Duchenne muscular dystrophy (for example, Becker muscular dystrophy) is considered unproven and not medically necessary and therefore is not covered under this policy.

Vyondys 53 is not indicated for Becker muscular dystrophy. Use of golodirsen in patients with Becker muscular dystrophy is unproven and not medically necessary, and claims for this indication will be denied.

Coding and Functional Measures

HCPCS CodesHCPCSCovered

J1429

Injection, golodirsen, 10 mg

Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

6‑Minute Walk Test (6MWT) baseline thresholds and trial eligibility notes

ESSENCE trial baseline 6MWT eligibility≥ 300 m and ≤ 450 m (ambulatory, corticosteroid-treated males aged 6–13 years)

SKIP‑NMD Part 2 inclusion>250 m baseline 6MWT for enrolled patients (male ages 6–15 years with additional functional criteria)

Purpose of 6MWT thresholdsUsed as an eligibility and primary efficacy endpoint (change from baseline) in clinical trials of golodirsen

Provider Actions, Authorization, and Documentation Requirements

Prior Authorization

Prior authorization required — initial auth ≤ 12 months; medical record verification

Prior authorization is required before initiating Vyondys 53. Initial authorizations are limited to no more than 12 months and medical records must confirm diagnosis and eligibility criteria (see documentation requirements).

Initial authorization will be for no more than 12 months.
Prior authorization must include medical records confirming diagnosis by or in consultation with a neurologist with expertise in DMD and mutation amenable to exon 53 skipping.

Step Therapy

Concomitant therapy restriction — prohibited concurrent agents

Vyondys 53 must not be administered concomitantly with specified therapies; concurrent use with these agents is prohibited and will affect coverage.

Do not use concomitantly with Duvyzat (givinostat).
Do not use concomitantly with other exon-skipping therapies for DMD (e.g., Amondys [casimersen], Exondys 51 [eteplirsen], Viltepso [viltolarsen]).

Background

Duchenne muscular dystrophy (DMD) is the specific disorder addressed by this policy; therapies directed at DMD‑specific molecular defects are evaluated separately from treatments for other muscular dystrophies. As noted throughout this policy, Vyondys 53 is indicated only for DMD with a confirmed mutation amenable to exon 53 skipping. Use of Vyondys 53 outside of that context — including for other muscular dystrophies such as Becker muscular dystrophy — is considered unproven and not medically necessary.

Definitions and Mechanism

Exon 53 skipping / golodirsen mechanism and definition

Mechanism — exon 53 skippingAntisense oligonucleotide binds to exon 53 of dystrophin pre‑mRNA resulting in exclusion of exon 53 during mRNA processing to allow production of an internally truncated dystrophin protein.

Golodirsen descriptionGolodirsen (Vyondys 53) is an antisense oligonucleotide designed to induce exon 53 skipping in patients with DMD mutations amenable to exon 53 skipping (~8% of DMD patients).

FDA indication noteIndicated for DMD patients with a confirmed DMD gene mutation amenable to exon 53 skipping; approval granted under accelerated approval based on dystrophin increase.