ModifiedColorado Rocky Mountain Health PlansPolicy CSOH2026D0095.F

Viltepso (viltolarsen) — Clinical Coverage Criteria (Ohio)

Clinical coverage policy for Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy in Ohio; defines initial and continuation authorization criteria, dosing guidance per FDA label, and exclusions.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyViltepso (viltolarsen) — Clinical Coverage Criteria (Ohio)

Policy CodePolicy CSOH2026D0095.F

Change TypeCoverage criteria revised — new concomitant-use exclusion

Effective DateJun 1, 2026

Next Review Date

Key ActionObtain prior authorization with records confirming DMD diagnosis amenable to exon 53 skipping and attest that Viltepso will not be used concomitantly with Duvyzat (givinostat).

SourceLink

POLICY UPDATE CHANGES

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

12 monthsMax approval period

J1427HCPCS code

Exon 53Mutation requirement

1New exclusion added

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Coverage Criteria for Viltepso (viltolarsen)

Initial Therapy Criteria

Covered when ALL of the following criteria are met:

Initial criteria: 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 confirming the patient is ambulatory prior to beginning Viltepso therapy; dosing is in accordance with FDA-approved labeling; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; initial authorization up to 12 months.

Prior gene therapy status: Either patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) OR both: patient previously received gene therapy 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.

See documentation requirement for prior gene therapy.

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

Concomitant use is an exclusion for coverage.

Continuation Therapy Criteria

Covered when ALL of the following are met for reauthorization:

Continuation criteria: Submission of medical records (e.g., chart notes) demonstrating the patient continues to remain ambulatory; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing is in accordance with FDA-approved labeling; reauthorization for up to 12 months.

Concomitant use is an exclusion for coverage.

Unproven / Not Medically Necessary

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

For coverage determinations, Viltepso must not be used for other forms of muscular dystrophy such as Becker muscular dystrophy; use in these indications is considered not medically necessary. This exclusion aligns with the policy statement that Viltepso is specifically for Duchenne muscular dystrophy with an exon 53–amenable mutation and is not supported for other dystrophies.

Additionally, concomitant use of Viltepso with other therapies intended to modify dystrophin production is excluded. The policy explicitly prohibits simultaneous use with givinostat (Duvyzat) and with other exon‑skipping agents (for example, Amondys, Exondys 51, Vyondys 53), and such concomitant prescribing may result in denial of coverage.

An operational exclusion has been added specifying that Viltepso will not be used concomitantly with Duvyzat (givinostat). This change is reflected in the policy revision effective 06/01/2026 and is an explicit contraindication for combined use of these products for covered members.

Providers requesting authorization should document that Viltepso will be prescribed alone (i.e., not in combination with Duvyzat); requests indicating concomitant use may be denied per the revised coverage criteria.

Viltepso 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 supported by the policy (Duchenne muscular dystrophy with an exon 53–amenable mutation).

Billing and Diagnosis Codes

Applicable HCPCS CodesHCPCS

J1427

Injection, viltolarsen, 10 mg.

Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

Provider Requirements and Authorization Workflow

Prior Authorization

Prior Authorization Required

Prior authorization required. Initial authorization will be for up to 12 months. Review applicable federal, state (OAC), and contractual benefit plan requirements as they may govern coverage decisions.

Initial authorization duration: up to 12 months
Check federal, state (OAC), or contractual requirements for plan-specific coverage rules

Documentation Required

Required Supporting Documentation

Submit supporting medical records with the prior authorization request. Documentation must confirm diagnosis of Duchenne muscular dystrophy and that the DMD gene mutation is amenable to exon 53 skipping. Include chart notes demonstrating the patient is ambulatory prior to initiating Viltepso.

Documentation: chart notes, laboratory values, genetic test results confirming exon 53 amenable mutation
Confirmation of ambulatory status prior to therapy start

Background on Duchenne Muscular Dystrophy and Therapy

Duchenne muscular dystrophy (DMD) is an X‑linked genetic disorder characterized by progressive muscle weakness resulting from absent or nonfunctional dystrophin. It most commonly affects males, with an estimated incidence of approximately 1 in 3,500 to 5,000 live male births. Progressive loss of ambulation and declining respiratory and cardiac function are typical features of the disease course.

Approximately 8–10% of patients with DMD have mutations amenable to exon 53 skipping; viltolarsen (Viltepso) is an antisense oligonucleotide designed to skip exon 53 and produce an internally truncated dystrophin protein. The agent is approved under accelerated pathways based on increases in dystrophin; clinical use is therefore limited to the genetically defined subgroup described above.

Key Definitions

Exon 53 skipping–amenable DMD

DefinitionDuchenne muscular dystrophy caused by out-of-frame deletion mutations where exclusion of exon 53 from dystrophin pre-mRNA can restore the reading frame to allow production of an internally truncated dystrophin protein.

Clinical mechanismViltolarsen is an antisense oligonucleotide that binds to exon 53 of dystrophin pre-mRNA, resulting in skipping of exon 53 during mRNA processing to increase dystrophin production.

Prevalence among DMD patientsApproximately 8%–10% of DMD patients have mutations amenable to exon 53 skipping.

Viltepso

Brand nameViltepso

Active ingredientViltolarsen (per package insert)