ModifiedUnitedHealthcarePolicy CSPA2026D0095I

Viltepso (viltolarsen) (for Pennsylvania Only)

Medical benefit drug coverage of Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy in patients with DMD gene mutations amenable to exon 53 skipping; applies to Pennsylvania only.

Policy Summary

PayerUnitedHealthcare

PolicyViltepso (viltolarsen) (for Pennsylvania Only)

Policy CodePolicy CSPA2026D0095I

Change TypeRevised coverage criteriaadded concomitant therapy exclusion

Effective DateJun 1, 2026

Next Review Date

Key ActionObtain prior authorization with neurologist documentation confirming the DMD mutation is amenable to exon 53 skipping and ensure Viltepso is not used concomitantly with Duvyzat (givinostat).

SourceLink

POLICY UPDATE CHANGES

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

Archived previous policy version CSPA2025D0095H.

J1427HCPCS code

G71.01Diagnosis code

12 monthsInitial auth duration

8-10%Amenable proportion

1Material changes

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Coverage Criteria for Viltepso (viltolarsen)

Initial Therapy

Covered when ALL of the following are met:

Initial Therapy for Viltepso: 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 documenting a baseline evaluation including a standardized assessment of motor function by a neurologist experienced in treating DMD; Viltepso will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., casimersen, eteplirsen, golodirsen); prescribed by, or in consultation with, a neurologist experienced in the treatment of DMD; dosing in accordance with U.S. FDA‑approved labeling; initial authorization for no more than 12 months.

One of the following must also be met: patient has not previously received gene therapy (e.g., Elevidys), OR patient previously received gene therapy and there is submission of medical records documenting a clinically meaningful functional decline since gene replacement therapy.

Continuation Therapy

Covered when ALL of the following are met:

Continuation Therapy for Viltepso: Documentation of continued clinical benefit based on the prescriber's assessment, including a standardized motor function reassessment by a neurologist experienced in treating DMD; Viltepso will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (e.g., casimersen, eteplirsen, golodirsen); prescribed by, or in consultation with, a neurologist with expertise in DMD; dosing in accordance with U.S. FDA‑approved labeling; reauthorization for no more than 12 months.

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).

Requests outside of the specified criteria will be reviewed for medical necessity on a case-by-case basis.

Initial coverage criteria

Covered when ALL of the following are met

FDA-indicated population: Patient has Duchenne muscular dystrophy with a confirmed DMD gene mutation that is amenable to exon 53 skipping.

Based on FDA indication under accelerated approval (increase in dystrophin observed); continued approval may be contingent on verification of clinical benefit in confirmatory trials.

Concomitant therapy exclusion: Viltepso will not be used concomitantly with Duvyzat (givinostat).

Newly added coverage criterion per policy revision.

Viltepso must not be administered at the same time as other specific investigational or approved therapies that employ exon‑skipping or histone‑deacetylase inhibition. Specifically, concomitant use with Duvyzat (givinostat) or other exon skipping therapies (for example, Amondys/casimersen, Exondys 51/eteplirsen, Vyondys 53/golodirsen) is not allowed and is an exclusion to coverage. This prohibition applies to both initiation and continuation of Viltepso therapy and must be verified in the documentation submitted for prior authorization.

Per the June 1, 2026 revision to the coverage criteria, Viltepso will not be used concomitantly with Duvyzat (givinostat). This revised criterion is a required condition of coverage and must be confirmed when evaluating prior authorization requests; lack of adherence may result in denial.

Requests for Viltepso to treat forms of muscular dystrophy other than Duchenne muscular dystrophy (for example, Becker muscular dystrophy) are considered not medically necessary. Such uses are unproven and will be reviewed only on a case‑by‑case basis under the policy’s medical necessity process.

Relevant Billing and Diagnosis Codes

Applicable HCPCS CodesHCPCS

J1427

Injection, viltolarsen, 10 mg.

Applicable Diagnosis Codes (listed)ICD-10

G71.01

Duchenne or Becker muscular dystrophy

Provider Requirements and Authorization Actions

Prior Authorization

Prior Authorization Required

Prior authorization is required. Prescriptions must be by, or in consultation with, a neurologist with expertise in the diagnosis and treatment of Duchenne muscular dystrophy (DMD). Medical records must be submitted confirming the DMD gene mutation is amenable to exon 53 skipping and documenting baseline standardized motor function assessments prior to initiating therapy. Initial authorization will be for no more than 12 months.

Prescriber: neurologist with DMD expertise required (or consult).
Documentation: medical records confirming amenable exon 53 mutation.
Documentation: baseline standardized motor function assessment by neurologist prior to initiation.
Initial authorization duration: up to 12 months.

Documentation Required

Submit medical records (chart notes, laboratory values, and standardized motor assessments) to support that the patient meets coverage criteria. Failure to provide requested documentation may result in denial or delay of coverage.

Background on Duchenne Muscular Dystrophy and Viltepso

Duchenne muscular dystrophy (DMD) is an X‑linked disorder characterized by progressive muscle weakness due to absent or nearly absent functional dystrophin. Viltolarsen (Viltepso) is an antisense oligonucleotide indicated for treatment of DMD in patients with a confirmed DMD gene mutation amenable to exon 53 skipping. The indication was approved under accelerated approval on the basis of increased dystrophin observed in skeletal muscle; continued approval may depend on confirmatory trials demonstrating clinical benefit. Coverage under this policy is limited to the FDA‑indicated population and requires documentation of the mutation’s amenability to exon 53 skipping, baseline and follow‑up standardized motor assessments by a neurologist experienced in DMD, dosing consistent with FDA labeling, and avoidance of prohibited concomitant agents such as Duvyzat or other exon skipping therapies.

Definitions and Regulatory Context

Amenable to exon 53 skipping

DefinitionDMD gene mutations amenable to exon 53 skipping are out‑of‑frame deletion mutations that can be corrected by excluding exon 53 during mRNA processing, enabling production of an internally truncated dystrophin protein.

MechanismViltolarsen (Viltepso) is an antisense oligonucleotide that binds exon 53 of dystrophin pre‑mRNA to induce exclusion of exon 53 and restore the dystrophin reading frame.

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

Clinical impactExon 53 skipping is intended to allow production of internally truncated dystrophin protein in patients with qualifying mutations, potentially increasing dystrophin levels above the typically <3% seen in DMD.

Accelerated approval

Regulatory pathway