ModifiedUnitedHealthcarePolicy CS2026D0095P

Viltepso (viltolarsen) for Duchenne muscular dystrophy — Medical Benefit Drug Policy (coverage criteria)

Clinical coverage policy for Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy (DMD) in patients with DMD gene mutations amenable to exon 53 skipping; governs medical benefit drug coverage and prior authorization requirements for UnitedHealthcare members (Commercial/Community Plan) except where state-specific exceptions apply.

Policy Summary

PayerUnitedHealthcare

PolicyViltepso (viltolarsen) for Duchenne muscular dystrophy — Medical Benefit Drug Policy

Policy CodePolicy CS2026D0095P

Change TypeRevised — added concomitant-use exclusion

Effective DateMay 1, 2026

Next Review Date

Key ActionObtain prior authorization and submit documentation confirming DMD mutation amenable to exon 53 skipping and that Viltepso will not be used concomitantly with Duvyzat.

SourceLink

POLICY UPDATE CHANGES

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

8-10%Proportion amenable to exon 53 skipping

J1427HCPCS code

≤12 moInitial auth duration

≤12 moReauth duration

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Coverage Criteria for Viltepso (viltolarsen)

Initial Therapy

Covered when ALL of the following are met

Initial Therapy 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; dosing per FDA-approved labeling; initial authorization will be for no more than 12 months; Viltepso will not be used concomitantly with givinostat or other exon skipping therapies; prescribed by or in consultation with a neurologist experienced in DMD

Functional eligibility is required as described in the nested node

Functional eligibility

6MWT pathway: Submission of medical records confirming 6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently (no assistive devices) prior to beginning Viltepso therapy6MWT >= 300 m
Ambulatory pathway: Submission of records confirming patient is ambulatory without needing an assistive device AND one of: NSAA > 17 OR time to rise (TTR) < 7 secondsNSAA > 17 OR TTR < 7s

Gene therapy history

Covered when ONE of the following is met regarding prior gene therapy

Gene therapy status: Either patient has not previously received gene therapy for DMD (e.g., Elevidys) OR patient previously received gene therapy and submission of medical records documents a clinically meaningful functional decline (loss of muscle strength/motor ability, e.g., loss of a motor milestone) since receiving gene replacement therapy

Records must document decline if prior gene therapy was given

Continuation Therapy

Reauthorization covered when ALL of the following are met

Continuation criteria: Submission of medical records confirming the patient is ambulatory without needing an assistive device; Viltepso not used concomitantly with givinostat or other exon skipping therapies; prescribed by, or in consultation with, a neurologist experienced in DMD; dosing in accordance with FDA-approved labeling; reauthorization will be for no more than 12 months

Unproven / Not Medically Necessary

Unproven uses: Viltepso is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (e.g., Becker muscular dystrophy) or DMD not amenable to exon 53 skipping

Coverage criteria (summary)

Covered when ALL of the following are met

FDA-indication-and-exclusion: Patient has Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 53 skipping AND Viltepso will not be used concomitantly with Duvyzat (givinostat).

Indication approved under accelerated approval based on increased dystrophin; continued approval may be contingent on verification of clinical benefit in confirmatory trials.

Use of Viltepso for other forms of muscular dystrophy (for example, Becker muscular dystrophy) is considered unproven and not medically necessary. Clinical documentation must demonstrate that the patient has Duchenne muscular dystrophy with a DMD gene mutation amenable to exon 53 skipping; treatments outside that indication (other dystrophies or non‑amenable DMD mutations) are not covered.

Per the revised coverage criteria effective 05/01/2026, Viltepso must not be used concomitantly with Duvyzat (givinostat). Concomitant use with Duvyzat is excluded from coverage and may result in denial of authorization.

Treatment of other forms of muscular dystrophy (for example, Becker muscular dystrophy) or Duchenne muscular dystrophy that is not amenable to exon 53 skipping is considered not medically necessary and is excluded from coverage.

Coding — HCPCS and Diagnosis Codes; Functional Thresholds

HCPCS CodesHCPCS

J1427

Injection, viltolarsen, 10 mg

Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

6-Minute Walk Test (6MWT) — threshold value

6MWT threshold≥ 300 meters (measured while walking independently, without assistive device)

Context for useRequired prior to initiating Viltepso as one pathway to meet functional eligibility

DocumentationSubmission of medical records (chart notes, test results) confirming the 6MWT value is required

North Star Ambulatory Assessment (NSAA) — threshold value

NSAA threshold> 17 (score greater than 17)

Context for useUsed as part of the ambulatory pathway when patient is ambulatory without assistive device

Provider Actions, Authorization, and Documentation

Prior Authorization

Prior Authorization and Authorization Length

Prior authorization is required for Viltepso (viltolarsen). Initial approvals are limited to a maximum of 12 months. Reauthorization (continuation) approvals are also limited to no more than 12 months per approval period.

Prior authorization required for initiation and continuation
Initial and reauthorization approvals capped at 12 months

Prior Authorization

Prior Authorization Requirement

Prior authorization is required when Viltepso is prescribed for Duchenne muscular dystrophy (DMD) with a DMD gene mutation amenable to exon 53 skipping. Coverage determination relies on medical benefit drug policy criteria and submission of supporting clinical documentation as described below.

PA must document indication: DMD with mutation amenable to exon 53 skipping
Refer to Medical Benefit Drug Policy for full coverage criteria

Background on Duchenne Muscular Dystrophy and Treatment Context

Duchenne muscular dystrophy (DMD) is an X‑linked disorder characterized by absent functional dystrophin and progressive muscle degeneration leading to respiratory and cardiac failure. Viltolarsen (Viltepso) is an antisense oligonucleotide designed to induce exon 53 skipping in dystrophin pre‑mRNA to produce an internally truncated, functional dystrophin protein in patients with mutations amenable to this approach. Approximately 8–10% of DMD patients have mutations amenable to exon 53 skipping.

Definitions

Exon 53 skipping — definition (key value)

DefinitionMolecular approach in which an antisense oligonucleotide binds exon 53 of dystrophin pre-mRNA to exclude that exon during mRNA processing, allowing production of an internally truncated but functional dystrophin protein

Treatment modalityMechanism targeted by viltolarsen (Viltepso) to restore dystrophin reading frame in eligible DMD mutations

Population proportionApproximately 8% to 10% of DMD patients have mutations amenable to exon 53 skipping

Amenable to exon 53 skipping — definition (key value)

DefinitionA genetic mutation in the DMD gene for which excluding exon 53 during mRNA processing can restore the dystrophin reading frame and enable production of a truncated functional dystrophin protein

Clinical relevance

Policy Summary

PayerUnitedHealthcare

PolicyViltepso (viltolarsen) for Duchenne muscular dystrophy — Medical Benefit Drug Policy

Policy CodePolicy CS2026D0095P

Change TypeRevised — added concomitant-use exclusion

Effective DateMay 1, 2026

Next Review Date

Key ActionObtain prior authorization and submit documentation confirming DMD mutation amenable to exon 53 skipping and that Viltepso will not be used concomitantly with Duvyzat.

SourceLink

On This Page

DocumentationSubmission of medical records (chart notes) confirming NSAA score is required

Time to Rise (TTR) — threshold value

TTR threshold< 7 seconds (time to rise less than 7 seconds)

Context for useUsed as an alternative functional criterion in the ambulatory pathway when patient is ambulatory without assistive device

DocumentationInstructions for use require documentation of TTR in medical records prior to therapy initiation

Denial Risk

Documentation and Clinical Criteria Absent or Contraindicated

If submitted medical records do not document a DMD gene mutation amenable to exon 53 skipping, required baseline functional or ambulatory data, or other required clinical information, coverage may be denied. Denials may also occur if dosing is not consistent with FDA‑approved labeling or if other contraindications in the benefit plan apply.

Lack of documentation of an exon 53 amenable mutation may result in denial
Missing baseline 6MWT, NSAA, or ambulatory status documentation may result in denial
Non‑FDA‑aligned dosing or plan exclusions may result in denial

Denial Risk

Concomitant Therapy Exclusion

Viltepso must not be used concomitantly with Duvyzat (givinostat) or with other exon‑skipping therapies for DMD. Coverage may be denied if Viltepso is prescribed concurrently with these excluded therapies.

Concomitant use with Duvyzat (givinostat) is prohibited
Concomitant use with other exon‑skipping therapies for DMD (e.g., casimersen, eteplirsen, golodirsen) is prohibited
Denial risk if concomitant therapy is documented

Documentation Required

Required Supporting Documentation

Required supporting documentation must be submitted with the prior authorization request, including confirmation of diagnosis, genetic testing showing a DMD gene mutation amenable to exon 53 skipping, and baseline functional assessments.

Genetic test results confirming DMD mutation amenable to exon 53 skipping
Baseline ambulatory status documentation (ambulatory without assistive device)
Baseline functional measures: 6MWT result (if applicable) and/or NSAA score
Medical records/chart notes and relevant laboratory values

Billing Rule

Concurrent Therapy Restrictions

Concurrent use of Viltepso with other exon‑skipping therapies or with givinostat is not allowed. Ensure medication lists and prior therapies are reviewed and documented in the medical record prior to authorization.

Verify patient is not currently receiving or planned to receive Duvyzat (givinostat)
Verify patient is not concurrently receiving other exon‑skipping therapies (casimersen, eteplirsen, golodirsen)
Document prior gene therapy and any subsequent clinically meaningful functional decline if applicable

Note

Provider-Facing Highlight

Provider note: This policy interprets UnitedHealthcare standard benefit plans. Always verify federal, state, and contractual plan terms which may supersede this guidance. Use specialist consultation and provide complete records to support the prior authorization request.

Prescribed by or in consultation with a neurologist experienced in DMD is required
Dosing must follow FDA‑approved labeling

Patients with such mutations are eligible for Viltepso per FDA indication when mutation is confirmed

Documentation requirementMedical records must confirm the DMD gene mutation is amenable to exon 53 skipping prior to coverage