ModifiedUnitedHealthcarePolicy CSOH2025D0095.E

Viltepso (viltolarsen) — Ohio Medical Benefit Drug Policy

This policy governs medical benefit coverage of Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy (DMD) in Ohio members; it specifies initial and continuation authorization criteria, coding, and limitations.

Policy Summary

PayerUnitedHealthcare

PolicyViltepso (viltolarsen) — Ohio Medical Benefit Drug Policy

Policy CodePolicy CSOH2025D0095.E

Change TypeRevised coverage criteria (initial therapy)

Effective DateAug 1, 2025

Next Review Date

Key ActionSubmit prior authorization with medical records confirming exon 53–amenable DMD mutation and ambulatory status; if prior gene therapy, include records showing clinically meaningful functional decline.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria for initial therapy; added criterion requiring one of the following: the patient has not previously received gene therapy (e.g., Elevidys) 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.

Archived previous policy version CSOH2025D0095.D was identified as supporting information.

12 monthsmaximum initial authorization period

12 monthsmaximum reauthorization period

J1427HCPCS code

G71.01

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Coverage Criteria for Viltepso (viltolarsen)

Initial Therapy

Covered when ALL of the following are met

Initial authorization criteria: Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD; and submission of medical records (e.g., chart notes, laboratory values) confirming the mutation of the DMD gene is amenable to exon 53 skipping; and submission of medical records (e.g., chart notes, laboratory values) confirming that the patient is ambulatory prior to beginning Viltepso therapy; and one of the following prior gene therapy status conditions must be met (see prior gene therapy requirement block): patient has not previously received gene therapy for DMD; OR patient has previously received gene therapy and submission of medical records documenting a clinically meaningful functional decline since gene replacement therapy; and Viltepso is not used concomitantly with other exon skipping therapies for DMD; and prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; and dosing is in accordance with the U.S. Food and Drug Administration approved labeling; and initial authorization will be for no more than 12 months.initial authorization <= 12 months

Includes exception path for prior gene therapy with documented decline

Continuation Therapy

Covered when ALL of the following are met

Continuation authorization criteria: Submission of medical records (e.g., chart notes) demonstrating that the patient continues to remain ambulatory; and Viltepso is not used concomitantly with other exon skipping therapies for DMD; and prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; and dosing is in accordance with the U.S. Food and Drug Administration approved labeling; and reauthorization will be for no more than 12 months.reauthorization <= 12 months

Ongoing ambulatory status required

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) and has not been studied in DMD that is not amenable to exon 53 skipping.

Initial therapy — prior gene therapy requirement (revised 08/01/2025)

Revised initial therapy requirement (08/01/2025):

Prior gene therapy status: One of the following must be met: (1) the patient has not previously received gene therapy (e.g., Elevidys [delandistrogene moxparvovec-rokl]) for the treatment of DMD; OR (2) both: the patient has previously received gene therapy for DMD, AND submission of medical records 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.

This is an added gate for initial therapy coverage as of 08/01/2025.

Viltepso (viltolarsen) is unproven and not medically necessary for the treatment of other forms of muscular dystrophy such as Becker muscular dystrophy. Clinical trial evidence and the sponsor’s ongoing studies focus on ambulatory DMD patients with mutations amenable to exon 53 skipping; there are no studies supporting use in non‑exon‑53‑amenable DMD or other muscular dystrophies.

The RACER53 pivotal program is an ongoing phase 3, randomized, double‑blind, placebo‑controlled trial enrolling ambulatory boys with genotypically confirmed DMD and deletions amenable to exon 53 skipping; therefore efficacy and safety data do not extend to Becker muscular dystrophy or DMD genotypes not amenable to exon 53 skipping.

FDA approval is informational and does not by itself determine coverage. Although Viltepso is indicated for DMD patients with a confirmed mutation amenable to exon 53 skipping and was granted accelerated approval based on increased dystrophin, coverage decisions must follow this policy and applicable benefit terms rather than approval status alone.

Use of Viltepso for other forms of muscular dystrophy (for example, Becker muscular dystrophy) is considered unproven and not medically necessary. The available clinical evidence and ongoing trials are limited to DMD patients with mutations amenable to exon 53 skipping; there are no efficacy or safety data supporting treatment of Becker or DMD not amenable to exon 53 skipping.

Coding

HCPCS codesHCPCSCovered

J1427

Injection, viltolarsen, 10 mg

Diagnosis codes (listed)ICD-10

G71.01

Duchenne or Becker muscular dystrophy

Provider Actions, Authorization & Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required. Providers must obtain prior authorization before initiating Viltepso (viltolarsen) therapy and must submit supporting documentation as described below. Initial authorizations are limited to a maximum of 12 months.

Prescribed by, or in consultation with, a neurologist with expertise in the diagnosis and treatment of Duchenne muscular dystrophy (DMD).
Initial authorization duration: up to 12 months; reauthorization (continuation) up to 12 months.

Prior Authorization

Prior Authorization Requirements for Initial Therapy

For initial therapy, submit medical records that include documentation of: a diagnosis of Duchenne muscular dystrophy by (or in consultation with) a neurologist with expertise in DMD; genetic testing results confirming a DMD gene mutation amenable to exon 53 skipping; and evidence that the patient is ambulatory at the time therapy is initiated. Also document prior gene therapy status as follows: either the patient has not previously received gene therapy (e.g., Elevidys), or if the patient previously received gene therapy, submit records documenting a clinically meaningful functional decline (for example, loss of muscle strength/motor ability or loss of a motor milestone) since receiving gene replacement therapy. Initial authorization will be for no more than 12 months.

Definitions and Context

Exon 53 skipping — molecular approach and clinical aim

DefinitionAntisense oligonucleotide that binds exon 53 of dystrophin pre-mRNA to exclude exon 53 during mRNA processing, producing an internally truncated dystrophin protein.

Target populationPatients with DMD who have confirmed DMD gene mutations amenable to exon 53 skipping (approximately 8–10% of DMD patients).

Molecular aimRestore reading frame to permit production of a truncated but functional dystrophin protein.

Accelerated approval — surrogate endpoint and confirmatory trial requirement

Regulatory basisFDA granted accelerated approval for Viltepso based on an increase in dystrophin in skeletal muscle as a surrogate endpoint.

ImplicationAccelerated approval is contingent on the surrogate endpoint; continued approval may require confirmatory trials demonstrating clinical benefit.

Background

Duchenne muscular dystrophy (DMD) is an X‑linked progressive myopathy caused by absent or nonfunctional dystrophin, leading to progressive muscle weakness and eventual respiratory and cardiac failure. Viltolarsen is an antisense oligonucleotide designed to induce exon 53 skipping, producing an internally truncated dystrophin protein in patients whose DMD gene mutation is amenable to skipping exon 53; approximately 8–10% of DMD patients have such mutations. Clinical trials to date (including the ongoing RACER53 phase 3 study) have enrolled ambulatory boys with genotypically confirmed DMD amenable to exon 53 skipping, and Viltepso has not been studied in DMD not amenable to exon 53 skipping.

Revision History

2025-08-01revisedLatest

Revised coverage criteria for initial therapy to add a prior gene therapy requirement: either the patient has not previously received gene therapy (e.g., Elevidys) or, if previously received gene therapy, submission of medical records documenting a clinically meaningful functional decline since gene replacement therapy is required.