ModifiedUnitedHealthcarePolicy 2025D0095I

Viltepso (viltolarsen) — Coverage Criteria for Duchenne muscular dystrophy (exon 53 skipping)

This policy governs coverage criteria for Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping for UnitedHealthcare Community Plan members in NC.

Policy Summary

PayerUnitedHealthcare

PolicyViltepso (viltolarsen) — Coverage Criteria for DMD (exon 53 skipping)

Policy CodePolicy 2025D0095I

Change TypeRevised coverage criteria (added prior gene therapy requirement)

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionSubmit medical records documenting DMD diagnosis, exon 53–amenable mutation, functional test results, prior gene therapy history and, if applicable, documentation of clinically meaningful functional decline since gene therapy when requesting authorization.

SourceLink

POLICY UPDATE CHANGES

Coverage criteria for initial therapy were revised to require either no prior gene therapy for DMD or, if prior gene therapy was received, documentation of clinically meaningful functional decline since gene replacement therapy.

Revised coverage criteria for initial therapy to require one of the following: the patient has not previously received gene therapy for DMD, or both that the patient previously received gene therapy and submission of medical records documenting a clinically meaningful functional decline since receiving gene replacement therapy.

Benefit Considerations section was updated to reflect the most current information.

Updated Benefit Considerations section to reflect the most current information.

≤12 moauthorization duration

5references

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1new prior-gene-therapy requirement

J1427HCPCS (viltolarsen)

G71.01Diagnosis code

Coverage Criteria

Initial Therapy

Covered when ALL of the following are met for Initial Therapy

Initial therapy overall

Functional eligibility option A: Submission of medical records confirming 6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently (without assistive device) prior to beginning Viltepso therapy.6MWT ≥ 300 m
Functional eligibility option B
- Ambulatory requirement: Ambulatory without needing an assistive device (no side-by-side assist, cane, walker, wheelchair).
- Motor function tests: NSAA > 17 OR TTR < 7 seconds.NSAA >17 or TTR <7s

Prior gene therapy branch

No prior gene therapy: Patient has not previously received gene therapy (e.g., Elevidys).
Prior gene therapy with decline: Patient previously received gene therapy AND submission of medical records documenting a clinically meaningful functional decline since gene therapy (e.g., loss of a motor milestone).

Initial Therapy — prior gene therapy condition

Covered when ALL of the following are met (includes new condition addressing prior gene therapy):

Initial therapy additional requirement: One of the following must be true: (1) The patient has not previously received gene therapy (e.g., Elevidys) for DMD; OR (2) BOTH: the patient has previously received gene therapy for DMD, AND submission of medical records documenting a clinically meaningful functional decline (e.g., loss of a motor milestone) since receiving gene replacement therapy.

This is an added requirement to initial therapy coverage criteria.

Continuation Therapy

Covered when ALL of the following are met for Continuation Therapy

Continuation therapy: Submission of medical records confirming patient remains ambulatory without needing an assistive device; not used concomitantly with other exon skipping therapies; prescribed by or in consultation with a neurologist with expertise in the treatment of DMD; dosing per FDA labeling; reauthorization ≤ 12 months.

Unproven/Not Medically Necessary

Not covered / Unproven: Viltepso is unproven and not medically necessary for the treatment of other forms of muscular dystrophy (e.g., Becker muscular dystrophy) and has not been studied in DMD not amenable to exon 53 skipping.

Viltepso (viltolarsen) is not covered for other forms of muscular dystrophy such as Becker muscular dystrophy and has not been studied in DMD that is not amenable to exon 53 skipping. Use in these diagnoses is considered outside the scope of this policy and is classified as unproven and not medically necessary.

Coverage determinations are governed by the member’s specific benefit plan document; when plan language conflicts with this policy, the member specific benefit plan controls. Providers must also check any applicable federal or state mandates prior to relying on this policy for coverage decisions.

Use of Viltepso for other forms of muscular dystrophy (for example, Becker muscular dystrophy) is considered unproven and not medically necessary. The agent has not been studied in non–exon 53–amenable DMD or in other muscular dystrophies, and therefore such uses do not meet criteria for coverage.

Coding

Applicable HCPCS CodesHCPCS

J1427

Injection, viltolarsen, 10 mg.

Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

Referenced therapies and productsNDC | mixed

Elevidys (delandistrogene moxparvovec-rokl)

Example gene therapy referenced in coverage criteria

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

6-Minute Walk Test (6MWT) threshold>= 300 meters while walking independently (without assistive device) prior to starting Viltepso

Context for useMust be documented in medical records (e.g., chart notes, laboratory values) submitted with prior authorization

Related denial riskFailure to document 6MWT ≥ 300 m may lead to denial of coverage

North Star Ambulatory Assessment (NSAA) score — numeric threshold/value

NSAA score threshold> 17 (North Star Ambulatory Assessment) documented prior to initiating Viltepso

Ambulatory requirementPatient must be ambulatory without needing an assistive device when NSAA is used as the functional criterion

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Viltepso (viltolarsen). Initial authorization will be for no more than 12 months; reauthorization (continuation therapy) will be granted for no more than 12 months per approval period.

Initial and reauthorization: up to 12 months
Prior authorization required

Documentation Required

Required Documentation

Submit medical records confirming diagnosis and treatment details. Required documentation includes confirmation of Duchenne muscular dystrophy diagnosis by or in consultation with a neurologist with expertise in DMD; documentation that the patient’s DMD mutation is amenable to exon 53 skipping; baseline and follow-up functional measures as applicable (e.g., 6-Minute Walk Test, North Star Ambulatory Assessment score, time to rise); documentation of ambulatory status; and records of prior gene therapy status and outcomes if applicable.

Definitions

Exon 53 skipping — definition and brief mechanism

DefinitionExon 53 skipping: a molecular approach where an agent binds to exon 53 of dystrophin pre-mRNA causing exclusion of exon 53 during mRNA processing to enable production of an internally truncated dystrophin protein in patients with mutations amenable to exon 53 skipping

Mechanism summarySkipping exon 53 permits translation of a shorter, partially functional dystrophin by restoring the mRNA reading frame

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

Viltolarsen — Viltepso definition (exon-skipping antisense oligonucleotide)

AgentViltolarsen (Viltepso) — an exon-skipping antisense oligonucleotide targeting exon 53 of dystrophin pre-mRNA

Intended effect

Background

Duchenne muscular dystrophy (DMD) is an X‑linked disorder caused by loss‑of‑function variants in the DMD gene that result in dystrophin deficiency and progressive muscle degeneration. Viltolarsen is an exon‑skipping antisense oligonucleotide designed to bind exon 53 of dystrophin pre‑mRNA to enable production of an internally truncated dystrophin protein and is applicable to approximately 8–10% of patients with DMD whose mutations are amenable to exon 53 skipping.

Revision History

2025-07-01coverage_criteria_revisionLatest

Revised coverage criteria for initial Viltepso therapy to add an explicit prior gene therapy branch: either no prior gene therapy (e.g., Elevidys) OR prior gene therapy plus submission of medical records documenting a clinically meaningful functional decline since gene replacement therapy; updated Benefit Considerations.

Policy Summary

PayerUnitedHealthcare

PolicyViltepso (viltolarsen) — Coverage Criteria for DMD (exon 53 skipping)

Policy CodePolicy 2025D0095I

Change TypeRevised coverage criteria (added prior gene therapy requirement)

Effective DateJul 1, 2025

Next Review DateN/A

SourceLink

On This Page

DocumentationNSAA score must be included in submitted medical records for prior authorization

Time to rise (TTR) — numeric threshold/value

Time to rise (TTR) threshold< 7 seconds measured prior to initiating Viltepso

Ambulatory requirementTTR criterion applies when patient is ambulatory without an assistive device

DocumentationRecorded TTR must be submitted in medical records for prior authorization review

Documentation of clinically meaningful functional decline — example/descriptor

Definition/exampleClinically meaningful functional decline is not numerically specified; example provided: loss of a motor milestone (e.g., loss of ability to perform a previously achieved motor task)

When requiredRequired when the patient previously received gene replacement therapy (e.g., Elevidys) to establish eligibility for initial Viltepso therapy

Documentation to submitMedical records (chart notes, laboratory values) documenting the functional decline since receiving gene therapy must be included with the prior authorization request

Diagnosis confirmed by or in consultation with a neurologist experienced in DMD

Medical records confirming DMD gene mutation amenable to exon 53 skipping

Baseline functional data (e.g., 6MWT ≥ 300 m OR ambulatory without assistive device plus NSAA >17 or TTR <7 sec) for initial therapy

For continuation: medical records confirming patient remains ambulatory without assistive device

Documentation of prior gene therapy (e.g., Elevidys) status and, if received, medical records documenting a clinically meaningful functional decline since gene replacement therapy

Denial Risk

Triggers for Denial

Therapy will be denied if required documentation or eligibility criteria are not met. Triggers for denial include failure to document DMD diagnosis by or in consultation with a qualified neurologist, lack of documentation that the mutation is amenable to exon 53 skipping, missing baseline functional measures or ambulatory status as required for initial or continuation therapy, use of Viltepso concomitantly with other exon‑skipping therapies, or absence of documentation regarding prior gene therapy status when that is required by the initial therapy criteria.

Failure to document DMD diagnosis by/with a neurologist expert in DMD
No documentation that DMD mutation is amenable to exon 53 skipping
Missing required baseline functional data (e.g., 6MWT, NSAA, TTR) for initial authorization
Missing documentation confirming ambulatory status for continuation therapy
Viltepso used concomitantly with other exon skipping therapies (e.g., casimersen, eteplirsen, golodirsen)
Missing documentation of prior gene therapy status or lacking records showing clinically meaningful decline since gene therapy when applicable

Note

Concomitant Therapy and Prescribing

Viltepso must not be used concomitantly with other exon skipping therapies for DMD (e.g., Amondys, Exondys 51, Vyondys 53). Prescriptions should follow FDA‑approved dosing and be prescribed by or in consultation with a neurologist experienced in DMD.

Do not administer concurrently with other exon skipping therapies for DMD
Prescribe per FDA labeling and in consultation with a DMD‑experienced neurologist

Binds to exon 53 of dystrophin pre-mRNA to cause exclusion of exon 53 during mRNA processing, enabling production of internally truncated dystrophin

Indicated populationFor treatment of Duchenne muscular dystrophy in patients with DMD gene mutations amenable to exon 53 skipping